Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

HE MGA — Multi & Mono: v1.0

EUROPEAN HEALTH AND DIGITAL EXECUTIVE AGENCY (HADEA)

HADEA.A – Health and Food A.3 – Health research

GRANT AGREEMENT

Project 101156595 — ERDERA

PREAMBLE

This Agreement (‘the Agreement’) is between the following parties:

on the one part,

the European Health and Digital Executive Agency (HADEA) (‘EU executive agency’ or ‘granting authority’), under the powers delegated by the European Commission (‘European Commission’),

and

on the other part,

1. ‘the coordinator’:

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM), PIC 999997833, established in RUE DE TOLBIAC 101, PARIS 75654, France,

and the following other beneficiaries, if they sign their ‘accession form’ (see Annex 3 and Article 40):

2. TEAMIT RESEARCH SL (TEAMIT), PIC 896104237, established in PASSE DE GRACIA 58 PLANTA 4 PUERTA 2, BARCELONA 08007, Spain,

3. DEUTSCHES ZENTRUM FUR LUFT - UND RAUMFAHRT EV (DLR), PIC 999981731, established in LINDER HOHE, KOLN 51147, Germany,

4. AGENCE NATIONALE DE LA RECHERCHE (ANR), PIC 998711613, established in 86 RUE REGNAULT, PARIS 75013, France,

5. FONDAZIONE TELETHON ETS (FTELE), PIC 999448716, established in VIA VARESE 16/ B, ROMA 00185, Italy,

6. Lietuvos mokslo taryba (LMT), PIC 997808446, established in Gedimino 3, Vilnius LT-01103, Lithuania,

7. ZORGONDERZOEK NEDERLAND ZON (ZonMw), PIC 999544164, established in Laan Van Nieuw Oost Indie 334, DEN HAAG 2593 CE, Netherlands,

8. EBERHARD KARLS UNIVERSITAET TUEBINGEN (UT), PIC 999991916, established in GESCHWISTER-SCHOLL-PLATZ, TUEBINGEN 72074, Germany,

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9. TARTU ULIKOOL (UTARTU), PIC 999895013, established in ULIKOOLI 18, TARTU 50090, Estonia,

10. CONSORCIO PARA LA EXPLOTACION DEL CENTRO NACIONAL DE ANALISIS GENOMICO (CNAG), PIC 882802433, established in C BALDIRI 1 REIXAC 4, BARCELONA 08028, Spain,

11. STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM (SRUMC), PIC 892057785, established in GEERT GROOTEPLEIN 10 ZUID, NIJMEGEN 6525 GA, Netherlands,

12. TECHNISCHE UNIVERSITAET MUENCHEN (TUM), PIC 999977463, established in Arcisstrasse 21, MUENCHEN 80333, Germany,

13. UNIVERSITATSKLINIKUM HEIDELBERG (UKHD), PIC 999841081, established in IM NEUENHEIMER FELD 672, HEIDELBERG 69120, Germany,

14. CRITICAL PATH INSTITUTE STICHTING (C-PATH), PIC 887895418, established in BARBARA STROZZILAAN 101, AMSTERDAM 1083 HN, Netherlands,

15. FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA (VHIR), PIC 999541642, established in PASSEIG VALL D HEBRON 119-129 EDIFICIO DE RECERCA, BARCELONA 08035, Spain,

16. ACADEMISCH ZIEKENHUIS LEIDEN (LUMC), PIC 999990849, established in ALBINUSDREEF 2, LEIDEN 2333 ZA, Netherlands,

17. UNIVERSITEIT TWENTE (UTWENTE), PIC 999900833, established in DRIENERLOLAAN 5, ENSCHEDE 7522 NB, Netherlands,

18. UNIVERSIDAD POLITECNICA DE MADRID (UPM), PIC 999974844, established in CALLE RAMIRO DE MAEZTU 7 EDIFICIO RECTORADO, MADRID 28040, Spain,

19. ACADEMISCH ZIEKENHUIS GRONINGEN (UMCG), PIC 999914801, established in HANZEPLEIN 1, GRONINGEN 9713 GZ, Netherlands,

20. STICHTING AMSTERDAM UMC (AUMC), PIC 919322739, established in DE BOELELAAN 1117, AMSTERDAM 1081 HV, Netherlands,

21. UNIVERSITEIT MAASTRICHT (UM), PIC 999975911, established in MINDERBROEDERSBERG 4, MAASTRICHT 6200 MD, Netherlands,

22. EATRIS ERIC (EATRIS), PIC 941506445, established in DE BOELELAAN 1118, AMSTERDAM 1081 HZ, Netherlands,

23. FONDAZIONE PER LA RICERCA FARMACOLOGICA GIANNI BENZI ONLUS (FGB), PIC 962478330, established in VIA ABATE EUSTASIO 30, VALENZANO BARI 70010, Italy,

24. ASSISTANCE PUBLIQUE HOPITAUX DE PARIS (APHP), PIC 999645432, established in 55 BOULEVARD DIDEROT, PARIS 75012, France,

25. UNIVERSITEIT HASSELT (UHASSELT), PIC 999874934, established in MARTELARENLAAN 42, HASSELT 3500, Belgium,

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26. EURORDIS - RARE DISEASES EUROPE (EURORDIS), PIC 965288323, established in RUE DIDOT 96, Paris 75014, France,

27. VIESOJI ISTAIGA VILNIAUS UNIVERSITETO LIGONINE SANTAROS KLINIKOS (VULSK), PIC 991636530, established in SANTARISKIU G 2, VILNIUS LT-08661, Lithuania,

28. UNIVERSIDADE DE COIMBRA (UC), PIC 997826391, established in PACO DAS ESCOLAS, COIMBRA 3004-531, Portugal,

29. FONDATION MALADIES RARES (FFRD), PIC 954438485, established in RUE DIDOT 96, PARIS 75014, France,

30. ASS FRANCAISE CONTRE LES MYOPATHIES (AFM), PIC 959828387, established in 47-83 47 BOULEVARD DE L'HOPITAL, INSTITUT DE MYOLO, PARIS 75013, France,

31. BERNU KLINISKA UNIVERSITATES SLIMNICA VALSTS SIA (CCUH), PIC 904374748, established in VIENIBAS GATVE 45, RIGA 1004, Latvia,

32. AICIB - AGENCIA DE INVESTIGACAO CLINICA E INOVACAO BIOMEDICA (AICIB), PIC 892687606, established in RUA DE SANTA CATARINA 1288, PORTO 4000-447, Portugal,

33. INSTITUT ZA MOLEKULARNU GENETIKU I GENETICKO INZENJERSTVO (IMGGE), PIC 986427921, established in VOJVODE STEPE 444A, BEOGRAD 11010, Serbia,

34. RARE DISEASES INTERNATIONAL (RDI), PIC 881348694, established in 96 RUE DIDOT, PARIS 75014, France,

35. ACIBADEM MEHMET ALI AYDINLAR UNIVERSITY (ACU), PIC 959704421, established in ICERENKOY MAH. KAYISDAGI CAD. NO/32 ATASEHIR, ISTANBUL 34752, Türkiye,

36. AIT AUSTRIAN INSTITUTE OF TECHNOLOGY GMBH (AIT), PIC 999584128, established in GIEFINGGASSE 4, WIEN 1210, Austria,

37. UNIVERSITE D'AIX MARSEILLE (AMU), PIC 955518483, established in BOULEVARD CHARLES LIVON 58 LE PHARO, MARSEILLE 13284, France,

38. AZIENDA OSPEDALIERA UNIVERSITARIA MEYER IRCCS (AOU Meyer IRCCS), PIC 998778543, established in VIALE PIERACCINI 24, FIRENZE 50139, Italy,

39. AZIENDA SANITARIA UNIVERSITARIA FRIULI CENTRALE (ASU-FC), PIC 894464355, established in VIA POZZUOLO 330, UDINE 33100, Italy,

40. BAR ILAN UNIVERSITY (BIU), PIC 999886574, established in BAR ILAN UNIVERSITY CAMPUS, RAMAT GAN 52900, Israel,

41. BUNDESMINISTERIUM FUER BILDUNG UND FORSCHUNG (BMBF), PIC 999835455, established in Heinemannstrasse 2, BONN 53175, Germany,

42. ST. ANNA KINDERKREBSFORSCHUNG GMBH (CCRI GmbH), PIC 891384896, established in ZIMMERMANNPLATZ 10, WIEN 1090, Austria,

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43. CENTOGENE GMBH (CENTOGENE), PIC 878333449, established in AM.STRANDE 7, ROSTOCK 18055, Germany,

44. CHARITE - UNIVERSITAETSMEDIZIN BERLIN (CHARITE), PIC 999992692, established in Chariteplatz 1, BERLIN 10117, Germany,

45. CHECKIMMUNE GMBH (CHI), PIC 889852781, established in CAMPUS VIRCHOW KLINIKUM, CRANACH HAUS AUGUSTENBURG PLATZ 1, BERLIN 13353, Germany,

46. CENTRE HOSPITALIER REG UNIVERSITAIRE DIJON (CHU Dijon), PIC 959994451, established in BOULEVARD MAL DE LATTRE DE TASSIGNY 2, DIJON 21000, France,

47. COPENHAGEN ECONOMICS AS (CE), PIC 938801309, established in LANGEBROGADE 1B, KOBENHAVN K 1411, Denmark,

48. REGION HOVEDSTADEN (REGIONH), PIC 999654744, established in KONGENS VAENGE 2, HILLEROD 3400, Denmark,

49. MINISTRY OF HEALTH (CSO-MOH), PIC 999596156, established in YIRMIYAHU 39, JERUSALEM 9101002, Israel,

50. CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE (CVBF), PIC 999717018, established in VIA NICOLO PUTIGNANI 178, BARI 70122, Italy,

51. STICHTING DUCHENNE DATA FOUNDATION (DDF), PIC 899914882, established in WARMOESDREEF 10, BERGEN OP ZOOM 4614 HC, Netherlands,

52. ELSEVIER BV (ELS), PIC 999937014, established in RADARWEG 29, AMSTERDAM 1043NX, Netherlands,

53. ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM (Erasmus MC), PIC 999988424, established in DR MOLEWATERPLEIN 40, ROTTERDAM 3015 GD, Netherlands,

54. FONDS DE LA RECHERCHE SCIENTIFIQUE- FNRS (F.R.S.-FNRS), PIC 999540381, established in RUE D'EGMONT 5, BRUXELLES 1000, Belgium,

55. FRAUNHOFER GESELLSCHAFT ZUR FORDERUNG DER ANGEWANDTEN FORSCHUNG EV (FHG), PIC 999984059, established in HANSASTRASSE 27C, MUNCHEN 80686, Germany,

56. FONDAZIONE REGIONALE PER LA RICERCA BIOMEDICA (FRRB), PIC 929009450, established in PIAZZA CITTA DI LOMBARDIA 1, MILANO 20124, Italy,

57. ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK (ECRIN), PIC 948646712, established in 30 BD SAINT-JACQUES BATIMENT B, PARIS 75014, France,

58. FONDS ZUR FÖRDERUNG DER WISSENSCHAFTLICHEN FORSCHUNG (FWF), PIC 998735960, established in GEORG-COCH-PLATZ 2, WIEN 1010, Austria,

59. INNOVATIONSFONDEN (IFD), PIC 939076692, established in OSTERGADE 26 A, KOBENHAVN K 1100, Denmark,

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60. FUNDACIO PRIVADA PER A LA RECERCA I LA DOCENCIA SANT JOAN DE DEU (FSJD-CERCA), PIC 999565601, established in SANTA ROSA 39-4, ESPLUGUES DE LLOBREGAT 08950, Spain,

61. FUNDACAO PARA A CIENCIA E A TECNOLOGIA (FCT), PIC 999543970, established in AVENIDA D CARLOS I 126, LISBOA 1249 074, Portugal,

62. MINISTERSTVO ZDRAVOTNICTVI CESKE REPUBLIKY (MZd), PIC 924144512, established in PALACKEHO NAMESTI 375/4, PRAHA 12801, Czechia,

63. ASSOCIATION GENETHON (GNT), PIC 998217883, established in RUE DE L INTERNATIONALE 1 BIS, EVRY 91002, France,

64. GEORGIAN FOUNDATION FOR GENETIC AND RARE DISEASES (GERAD), PIC 953089312, established in TARKHNISHVILI STREET 9 APT 5, TBIILISI 0179, Georgia,

65. REGION STOCKHOLM (RS), PIC 999522921, established in HANTVERKARGATAN 45, STOCKHOLM 104 22, Sweden,

66. JOHANN WOLFGANG GOETHE-UNIVERSITAET FRANKFURT AM MAIN (GUF), PIC 999978724, established in THEODOR W ADORNO PLATZ 1, FRANKFURT AM MAIN 60629, Germany,

67. THE HEALTH RESEARCH BOARD (HRB), PIC 999546686, established in 67 72 LOWER MOUNT STREET, DUBLIN D02 H638, Ireland,

68. IZMIR BIYOTIP VE GENOM MERKEZI (İBG), PIC 906758620, established in MITHATPASA CAD. NO:58/5 BALCOVA, IZMIR 35340, Türkiye,

69. RANNSOKNAMIDSTOD ISLANDS (Rannis), PIC 999547268, established in BORGARTUNI 30, REYKJAVIK 105, Iceland,

70. IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION (IMAGINE), PIC 955276177, established in 24 BD DU MONTPARNASSE, PARIS 15 75015, France,

71. INSTYTUT GENETYKI CZLOWIEKA POLSKIEJ AKADEMII NAUK (IGC PAN), PIC 982958425, established in STRZESZYNSKA 32, POZNAN 60-479, Poland,

72. INSTITUTO DE SALUD CARLOS III (ISCIII), PIC 999507886, established in MONFORTE DE LEMOS 5, MADRID 28029, Spain,

73. INSTITUTO NACIONAL DE SAUDE DR. RICARDO JORGE (INSA), PIC 998308190, established in AVENIDA PADRE CRUZ, LISBOA 1649-016, Portugal,

74. ISTITUTO ORTOPEDICO RIZZOLI (IOR), PIC 999445709, established in VIA DI BARBIANO 1/10, BOLOGNA 40136, Italy,

75. ISTITUTO SUPERIORE DI SANITA (ISS), PIC 999978821, established in Viale Regina Elena 299, ROMA 00161, Italy,

76. MINISTERO DELLA SALUTE (IT-MOH), PIC 999531942, established in Via Giorgio Ribotta 5, ROMA 00144, Italy,

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77. KATHOLIEKE UNIVERSITEIT LEUVEN (KU Leuven), PIC 999991334, established in OUDE MARKT 13, LEUVEN 3000, Belgium,

78. LATVIJAS ZINATNES PADOME (LZP), PIC 999546589, established in SMILSU IELA 8, RIGA LV-1050, Latvia,

79. MAPI RESEARCH TRUST (MRT), PIC 925695542, established in 27 RUE DE LA VILLETTE, LYON 69003, France,

80. MEDICAL UNIVERSITY SOFIA (MUS), PIC 999857571, established in ACAD IV EVSTATIEV GESHOV ST 15, SOFIA 1431, Bulgaria,

81. FAKULTNI NEMOCNICE V MOTOLE (MUH), PIC 964458391, established in V UVALU 84, PRAHA 5 150 06, Czechia,

82. Ministero dell'università e della ricerca (MUR), PIC 894763406, established in Via Michele Carcani 61, Roma 00153, Italy,

83. NARODOWE CENTRUM BADAN I ROZWOJU (NCBR), PIC 999519720, established in UL. CHMIELNA 69, WARSZAWA 00-801, Poland,

84. ETHNIKO KAI KAPODISTRIAKO PANEPISTIMIO ATHINON (NKUA), PIC 999643007, established in 6 CHRISTOU LADA STR, ATHINA 10561, Greece,

85. MINISTERSTVO ZDRAVOTNICTVA SLOVENSKEJ REPUBLIKY (MoH SR), PIC 999825173, established in LIMBOVA 2, BRATISLAVA 83752, Slovakia,

86. ENOSI SPANION ASTHENON ELLADOS (RDG), PIC 883276763, established in KAPNIKAREAS 19A, ATHINA 105 56, Greece,

87. REGION SYDDANMARK (REGIONSYD), PIC 999602073, established in DAMHAVEN 12, VEJLE 7100, Denmark,

88. OSPEDALE PEDIATRICO BAMBINO GESU (OPBG), PIC 998837810, established in PIAZZA SANT ONOFRIO 4, ROMA 00165, Italy,

89. OSPEDALE SAN RAFFAELE SRL (OSR), PIC 953176030, established in VIA OLGETTINA 60, MILANO 20132, Italy,

90. INSTITUT JOZEF STEFAN (JSI), PIC 999971837, established in Jamova 39, LJUBLJANA 1000, Slovenia,

91. NORGES FORSKNINGSRAD (RCN), PIC 999460453, established in DRAMMENSVEIEN 288, OSLO 0283, Norway,

92. FONDS VOOR WETENSCHAPPELIJK ONDERZOEK-VLAANDEREN (FWO), PIC 997949484, established in LEUVENSEWEG 38, BRUSSEL 1000, Belgium,

93. RIGAS AUSTRUMU KLINISKA UNIVERSITATES SLIMNICA SIA (REUH), PIC 973366483, established in HIPOKRATA IELA 2, RIGA LV-1079, Latvia,

94. IDRYMA EREVNAS KAI KAINOTOMIAS (RIF), PIC 999946035, established in ANDREA MICHALAKOPOULOU 29 A, NICOSIA 1075, Cyprus,

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95. ROYAL COLLEGE OF SURGEONS IN IRELAND (RCSI), PIC 999867368, established in ST STEPHEN'S GREEN 123, DUBLIN 2, Ireland,

96. RIGAS STRADINA UNIVERSITATE (RSU), PIC 999843118, established in Dzirciema street 16, RIGA 1007, Latvia,

97. SCIENSANO (Sciensano), PIC 906160809, established in JULIETTE WYTSMANSTRAAT 14, ELSENE 1050, Belgium,

98. SERVICE PUBLIC DE WALLONIE (SPW), PIC 999811884, established in Place de la Wallonie 1, Namur 5100, Belgium,

99. SIGMUND FREUD PRIVATUNIVERSITAT WIEN GMBH (SFU), PIC 996709824, established in FREUDPLATZ 1, WIEN 1020, Austria,

100. SIHTASUTUS EESTI TEADUSAGENTUUR (ETAg), PIC 998483760, established in SOOLA 8, TARTU 51004, Estonia,

101. SLOVENSKA AKADEMIA VIED (SAS), PIC 999530390, established in STEFANIKOVA 49, BRATISLAVA 814 38, Slovakia,

102. SONIO (Sonio), PIC 886915427, established in 24 RUE DU FAUBOURG SAINT-JACQUES, PARIS 75014, France,

103. SOTSIAALMINISTEERIUM (MOSAE), PIC 998429731, established in Suur-Ameerika 1, TALLINN 10122, Estonia,

104. VETENSKAPSRADET - SWEDISH RESEARCH COUNCIL (SRC), PIC 999586165, established in BOX 1035, STOCKHOLM 101 38, Sweden,

105. TEDDY - EUROPEAN NETWORK OF EXCELLENCE FOR PAEDIATRIC CLINICAL RESEARCH (TEDDY), PIC 910591963, established in VIA LUIGI PORTA 14, PAVIA 231261, Italy,

106. TEKKARE (TEKKARE), PIC 881967069, established in 11-19 RUE DE LA VANNE, SOPARQ, BAT C, MONTROUGE 92120, France,

107. THALASSAEMIA INTERNATIONAL FEDERATION (TIF), PIC 940669626, established in AKROPOLEOS 21 STROVOLOS, LEFKOSIA 2006, Cyprus,

108. TURKIYE BILIMSEL VE TEKNOLOJIK ARASTIRMA KURUMU (TUBITAK), PIC 999587135, established in Ataturk Bulvari 221, ANKARA 06100, Türkiye,

109. REGIONE TOSCANA (RT (TuscReg)), PIC 998823842, established in Palazzo Strozzi Sacrati - Piazza del Duomo 10, FIRENZE 50122, Italy,

110. UNIVERSITAT AUTONOMA DE BARCELONA (UAB), PIC 999986484, established in EDIF A CAMPUS DE LA UAB BELLATERRA CERDANYOLA V, CERDANYOLA DEL VALLES 08193, Spain,

111. UNIVERSITY COLLEGE DUBLIN, NATIONAL UNIVERSITY OF IRELAND, DUBLIN (UCD), PIC 999974359, established in BELFIELD, DUBLIN 4, Ireland,

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112. UNIVERSITA CATTOLICA DEL SACRO CUORE (UCSC), PIC 999915771, established in LARGO GEMELLI 1, MILANO 20123, Italy,

113. ITA-SUOMEN YLIOPISTO (UEF), PIC 991207984, established in YLIOPISTONRANTA 8, KUOPIO 70211, Finland,

114. UNIVERSITAETSKLINIKUM AACHEN (UKA), PIC 999897632, established in Pauwelsstrasse 30, AACHEN 52074, Germany,

115. UNIVERSITAETSKLINIKUM FREIBURG (UKLFR), PIC 999881918, established in HUGSTETTER STRASSE 49, FREIBURG 79106, Germany,

116. UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI (UNICAMPANIA), PIC 999848356, established in VIALE ABRAMO LINCOLN 5, CASERTA 81100, Italy,

117. PANEPISTIMIO KRITIS (UoC), PIC 999588978, established in UNIVERSITY CAMPUS GALLOS, RETHIMNO 74100, Greece,

118. UNIVERSITEIT GENT (UGENT), PIC 999986096, established in SINT PIETERSNIEUWSTRAAT 25, GENT 9000, Belgium,

119. UNIVERZITETNI KLINICNI CENTER LJUBLJANA (UKCL), PIC 999882306, established in ZALOSKA CESTA 002, LJUBLJANA 1000, Slovenia,

120. STICHTING WORLD DUCHENNE ORGANIZATION (WDO), PIC 930321860, established in KONINGINNELAAN 69, VEENENDAAL 3905 GG, Netherlands,

121. UPPSALA UNIVERSITET (UU), PIC 999985029, established in VON KRAEMERS ALLE 4, UPPSALA 751 05, Sweden,

122. CONECT4CHILDREN STICHTING (c4c-S), PIC 881618063, established in NICOLAAS BEETSSTRAAT 216, UTRECHT 3511 HG, Netherlands,

123. UNIVERSITE CADI AYYAD (CAU), PIC 998639930, established in BOULEVARD ABDELKARIM KHATTABI, MARRAKECH 40 000, Morocco,

124. CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS (CNRS), PIC 999997930, established in RUE MICHEL ANGE 3, PARIS 75794, France,

125. CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P. (CIBER), PIC 997154957, established in CALLE MONFORTE DE LEMOS 5, MADRID 28029, Spain,

126. UNITATEA EXECUTIVA PENTRU FINANTAREA INVATAMANTULUI SUPERIOR A CERCETARII DEZVOLTARII SI INOVARII (UEFISCDI), PIC 972130024, established in STR D I MENDELEEV 21-25, BUCURESTI 010362, Romania,

127. UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA (UNIROMA1), PIC 999987745, established in Piazzale Aldo Moro 5, ROMA 00185, Italy,

128. UNIVERSITEIT ANTWERPEN (UANTWERPEN), PIC 999902870, established in PRINSSTRAAT 13, ANTWERPEN 2000, Belgium,

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129. BULGARIAN NATIONAL SCIENCE FUND (BNSF), PIC 984222335, established in BLVD KNYAZ DONDUKOV 2 A, SOFIA 1000, Bulgaria,

130. REGION NORDJYLLAND (NORTH DENMARK REGION) (Aalborg UH), PIC 997381064, established in Niels Bohrs Vej 30, AALBORG 9220, Denmark,

131. REDKI BOLESTI BULGARIA (RDB), PIC 880694720, established in NISHAVA 121, SOFIA 1408, Bulgaria,

132. BIOBANKS AND BIOMOLECULAR RESOURCES RESEARCH INFRASTRUCTURE CONSORTIUM (BBMRI-ERIC) (BBMRI-ERIC), PIC 946597878, established in NEUE STIFTINGTALSTRASSE 2/B/6, GRAZ 8010, Austria,

133. AARHUS UNIVERSITETSHOSPITAL (AarhusUH), PIC 999643880, established in PALLE JUUL-JENSENS BOULEVARD 99, AARHUS 8200, Denmark,

134. ASSOCIATION INTERNATIONALE DE STANDARDISATION BIOLOGIQUE POUR L'EUROPE(IABS-EU) (IABS-EU), PIC 934774936, established in 24 RUE JEAN BALDASSINI, LYON 69393, France,

135. HEALTH RESEARCH CHARITIES IRELAND COMPANY LIMITED BY GUARANTEE (HRCI), PIC 880683274, established in 12 CAMDEN ROW, DUBLIN D08 R9CN, Ireland,

136. LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN (LMU), PIC 999978433, established in GESCHWISTER SCHOLL PLATZ 1, MUNCHEN 80539, Germany,

137. MYSCIENCEWORK (MSW), PIC 898132119, established in 101 RUE DE SEVRES LOT 1674, PARIS 75006, France,

138. FONDS NATIONAL DE LA RECHERCHE (FNR), PIC 998597153, established in 2 AVENUE DE L'UNIVERSITE, ESCH-SUR-ALZETTE 4365, Luxembourg,

139. NEMZETI KUTATASI FEJLESZTESI ES INNOVACIOS HIVATAL (NKFIH), PIC 999578696, established in KETHLY ANNA TER 1, BUDAPEST 1077, Hungary,

140. BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL (PEI), PIC 998217301, established in PAUL-EHRLICH-STRASSE 51-59, LANGEN 63225, Germany,

141. PARIS-LODRON-UNIVERSITAT SALZBURG (PLUS), PIC 999868047, established in KAPITELGASSE 4-6, SALZBURG 5020, Austria,

142. SORBONNE UNIVERSITE (SU), PIC 909875521, established in 21 RUE DE L'ECOLE DE MEDECINE, PARIS 75006, France,

143. UNIVERSITA DEGLI STUDI DI SIENA (UNISI), PIC 999898020, established in VIA BANCHI DI SOTTO 55, SIENA 53100, Italy,

144. VERKET FOR INNOVATIONSSYSTEM (Vinnova), PIC 999618757, established in MASTER SAMUELSG 56, STOCKHOLM 10158, Sweden,

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145. FUNDACIO CENTRE DE REGULACIO GENOMICA (CRG-CERCA), PIC 999544455, established in CARRER DOCTOR AIGUADER 88, BARCELONA 08003, Spain,

146. REGION SKANE (GMS-RS), PIC 998165794, established in REGION SKANE, KRISTIANSTAD 291 89, Sweden,

147. VIB VZW (VIB), PIC 999651931, established in SUZANNE TASSIERSTRAAT 1, ZWIJNAARDE - GENT 9052, Belgium,

148. INSTITUT DE PATHOLOGIE ET DE GENETIQUE ASBL (IPG), PIC 878266034, established in Av. Georges Lemaitre 25, GOSSELIES 6041, Belgium,

149. UNIVERSITY OF OTAGO (UO), PIC 998331567, established in SAINT DAVID STREET, DUNEDIN 9054, New Zealand,

150. HOSPITAL SANT JOAN DE DEU (HSJD), PIC 997929890, established in PASEO SANT JOAN DE DEU 2, ESPLUGUES DE LLOBREGAT 08950, Spain,

Unless otherwise specified, references to ‘beneficiary’ or ‘beneficiaries’ include the coordinator and affiliated entities (if any).

If only one beneficiary signs the grant agreement (‘mono-beneficiary grant’), all provisions referring to the ‘coordinator’ or the ‘beneficiaries’ will be considered — mutatis mutandis — as referring to the beneficiary.

The parties referred to above have agreed to enter into the Agreement.

By signing the Agreement and the accession forms, the beneficiaries accept the grant and agree to implement the action under their own responsibility and in accordance with the Agreement, with all the obligations and terms and conditions it sets out.

The Agreement is composed of:

Preamble

Terms and Conditions (including Data Sheet)

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Annex 1 Description of the action1

Annex 2 Estimated budget for the action

Annex 2a Additional information on unit costs and contributions (if applicable)

Annex 3 Accession forms (if applicable)2

Annex 3a Declaration on joint and several liability of affiliated entities (if applicable)3

Annex 4 Model for the financial statements

Annex 5 Specific rules (if applicable)

1 Template published on Portal Reference Documents. 2 Template published on Portal Reference Documents. 3 Template published on Portal Reference Documents.

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TERMS AND CONDITIONS

TABLE OF CONTENTS

GRANT AGREEMENT.................................................................................................................................................... 1

PREAMBLE........................................................................................................................................................................1

TERMS AND CONDITIONS.........................................................................................................................................12

DATASHEET.................................................................................................................................................................... 17

CHAPTER 1 GENERAL..............................................................................................................................................27

ARTICLE 1 — SUBJECT OF THE AGREEMENT ..................................................................................... 27

ARTICLE 2 — DEFINITIONS........................................................................................................................27

CHAPTER 2 ACTION................................................................................................................................................. 28

ARTICLE 3 — ACTION................................................................................................................................. 28

ARTICLE 4 — DURATION AND STARTING DATE...................................................................................28

CHAPTER 3 GRANT...................................................................................................................................................28

ARTICLE 5 — GRANT...................................................................................................................................28

5.1 Form of grant......................................................................................................................................28

5.2 Maximum grant amount..................................................................................................................... 29

5.3 Funding rate........................................................................................................................................29

5.4 Estimated budget, budget categories and forms of funding.............................................................. 29

5.5 Budget flexibility................................................................................................................................29

ARTICLE 6 — ELIGIBLE AND INELIGIBLE COSTS AND CONTRIBUTIONS......................................30

6.1 General eligibility conditions............................................................................................................. 30

6.2 Specific eligibility conditions for each budget category................................................................... 31

6.3 Ineligible costs and contributions...................................................................................................... 36

6.4 Consequences of non-compliance...................................................................................................... 37

CHAPTER 4 GRANT IMPLEMENTATION............................................................................................................ 37

SECTION 1 CONSORTIUM: BENEFICIARIES, AFFILIATED ENTITIES AND OTHER PARTICIPANTS........................................................................................................................................ 37

ARTICLE 7 — BENEFICIARIES................................................................................................................... 37

ARTICLE 8 — AFFILIATED ENTITIES....................................................................................................... 39

ARTICLE 9 — OTHER PARTICIPANTS INVOLVED IN THE ACTION................................................... 40

9.1 Associated partners.............................................................................................................................40

9.2 Third parties giving in-kind contributions to the action.................................................................... 41

9.3 Subcontractors.....................................................................................................................................42

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9.4 Recipients of financial support to third parties..................................................................................42

ARTICLE 10 — PARTICIPANTS WITH SPECIAL STATUS....................................................................... 42

10.1 Non-EU participants......................................................................................................................... 42

10.2 Participants which are international organisations...........................................................................43

10.3 Pillar-assessed participants............................................................................................................... 43

SECTION 2 RULES FOR CARRYING OUT THE ACTION...........................................................................45

ARTICLE 11 — PROPER IMPLEMENTATION OF THE ACTION............................................................ 45

11.1 Obligation to properly implement the action................................................................................... 46

11.2 Consequences of non-compliance.................................................................................................... 46

ARTICLE 12 — CONFLICT OF INTERESTS.............................................................................................. 46

12.1 Conflict of interests.......................................................................................................................... 46

12.2 Consequences of non-compliance.................................................................................................... 46

ARTICLE 13 — CONFIDENTIALITY AND SECURITY............................................................................ 46

13.1 Sensitive information........................................................................................................................46

13.2 Classified information...................................................................................................................... 47

13.3 Consequences of non-compliance.................................................................................................... 47

ARTICLE 14 — ETHICS AND VALUES...................................................................................................... 47

14.1 Ethics.................................................................................................................................................47

14.2 Values................................................................................................................................................48

14.3 Consequences of non-compliance.................................................................................................... 48

ARTICLE 15 — DATA PROTECTION.......................................................................................................... 48

15.1 Data processing by the granting authority....................................................................................... 48

15.2 Data processing by the beneficiaries............................................................................................... 48

15.3 Consequences of non-compliance.................................................................................................... 49

ARTICLE 16 — INTELLECTUAL PROPERTY RIGHTS (IPR) — BACKGROUND AND RESULTS — ACCESS RIGHTS AND RIGHTS OF USE................................................................................ 49

16.1 Background and access rights to background..................................................................................49

16.2 Ownership of results.........................................................................................................................49

16.3 Rights of use of the granting authority on materials, documents and information received for policy, information, communication, dissemination and publicity purposes...................................49

16.4 Specific rules on IPR, results and background................................................................................50

16.5 Consequences of non-compliance.................................................................................................... 51

ARTICLE 17 — COMMUNICATION, DISSEMINATION AND VISIBILITY............................................ 51

17.1 Communication — Dissemination — Promoting the action........................................................... 51

17.2 Visibility — European flag and funding statement......................................................................... 51

17.3 Quality of information — Disclaimer..............................................................................................52

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17.4 Specific communication, dissemination and visibility rules............................................................52

17.5 Consequences of non-compliance.................................................................................................... 52

ARTICLE 18 — SPECIFIC RULES FOR CARRYING OUT THE ACTION............................................... 52

18.1 Specific rules for carrying out the action........................................................................................ 52

18.2 Consequences of non-compliance.................................................................................................... 52

SECTION 3 GRANT ADMINISTRATION......................................................................................................... 52

ARTICLE 19 — GENERAL INFORMATION OBLIGATIONS....................................................................52

19.1 Information requests......................................................................................................................... 52

19.2 Participant Register data updates..................................................................................................... 53

19.3 Information about events and circumstances which impact the action............................................53

19.4 Consequences of non-compliance.................................................................................................... 53

ARTICLE 20 — RECORD-KEEPING............................................................................................................ 53

20.1 Keeping records and supporting documents.................................................................................... 53

20.2 Consequences of non-compliance.................................................................................................... 54

ARTICLE 21 — REPORTING........................................................................................................................ 55

21.1 Continuous reporting........................................................................................................................ 55

21.2 Periodic reporting: Technical reports and financial statements....................................................... 55

21.3 Currency for financial statements and conversion into euros..........................................................56

21.4 Reporting language...........................................................................................................................56

21.5 Consequences of non-compliance.................................................................................................... 56

ARTICLE 22 — PAYMENTS AND RECOVERIES — CALCULATION OF AMOUNTS DUE................. 56

22.1 Payments and payment arrangements.............................................................................................. 56

22.2 Recoveries.........................................................................................................................................57

22.3 Amounts due.....................................................................................................................................57

22.4 Enforced recovery.............................................................................................................................63

22.5 Consequences of non-compliance.................................................................................................... 64

ARTICLE 23 — GUARANTEES....................................................................................................................64

ARTICLE 24 — CERTIFICATES................................................................................................................... 64

24.1 Operational verification report (OVR).............................................................................................64

24.2 Certificate on the financial statements (CFS).................................................................................. 64

24.3 Certificate on the compliance of usual cost accounting practices (CoMUC).................................. 65

24.4 Systems and process audit (SPA).....................................................................................................65

24.5 Consequences of non-compliance.................................................................................................... 66

ARTICLE 25 — CHECKS, REVIEWS, AUDITS AND INVESTIGATIONS — EXTENSION OF FINDINGS..................................................................................................................................... 66

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25.1 Granting authority checks, reviews and audits................................................................................ 66

25.2 European Commission checks, reviews and audits in grants of other granting authorities..............67

25.3 Access to records for assessing simplified forms of funding.......................................................... 68

25.4 OLAF, EPPO and ECA audits and investigations........................................................................... 68

25.5 Consequences of checks, reviews, audits and investigations — Extension of results of reviews, audits or investigations.................................................................................................................... 68

25.6 Consequences of non-compliance.................................................................................................... 69

ARTICLE 26 — IMPACT EVALUATIONS................................................................................................... 70

26.1 Impact evaluation............................................................................................................................. 70

26.2 Consequences of non-compliance.................................................................................................... 70

CHAPTER 5 CONSEQUENCES OF NON-COMPLIANCE.................................................................................. 70

SECTION 1 REJECTIONS AND GRANT REDUCTION.................................................................................70

ARTICLE 27 — REJECTION OF COSTS AND CONTRIBUTIONS...........................................................70

27.1 Conditions......................................................................................................................................... 70

27.2 Procedure.......................................................................................................................................... 70

27.3 Effects............................................................................................................................................... 71

ARTICLE 28 — GRANT REDUCTION........................................................................................................ 71

28.1 Conditions......................................................................................................................................... 71

28.2 Procedure.......................................................................................................................................... 71

28.3 Effects............................................................................................................................................... 71

SECTION 2 SUSPENSION AND TERMINATION............................................................................................71

ARTICLE 29 — PAYMENT DEADLINE SUSPENSION............................................................................. 72

29.1 Conditions......................................................................................................................................... 72

29.2 Procedure.......................................................................................................................................... 72

ARTICLE 30 — PAYMENT SUSPENSION...................................................................................................72

30.1 Conditions......................................................................................................................................... 72

30.2 Procedure.......................................................................................................................................... 73

ARTICLE 31 — GRANT AGREEMENT SUSPENSION..............................................................................73

31.1 Consortium-requested GA suspension............................................................................................. 73

31.2 EU-initiated GA suspension.............................................................................................................74

ARTICLE 32 — GRANT AGREEMENT OR BENEFICIARY TERMINATION......................................... 75

32.1 Consortium-requested GA termination............................................................................................ 75

32.2 Consortium-requested beneficiary termination................................................................................ 76

32.3 EU-initiated GA or beneficiary termination.................................................................................... 77

SECTION 3 OTHER CONSEQUENCES: DAMAGES AND ADMINISTRATIVE SANCTIONS............... 80

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ARTICLE 33 — DAMAGES...........................................................................................................................81

33.1 Liability of the granting authority....................................................................................................81

33.2 Liability of the beneficiaries............................................................................................................ 81

ARTICLE 34 — ADMINISTRATIVE SANCTIONS AND OTHER MEASURES....................................... 81

SECTION 4 FORCE MAJEURE.......................................................................................................................... 81

ARTICLE 35 — FORCE MAJEURE..............................................................................................................81

CHAPTER 6 FINAL PROVISIONS...........................................................................................................................82

ARTICLE 36 — COMMUNICATION BETWEEN THE PARTIES...............................................................82

36.1 Forms and means of communication — Electronic management................................................... 82

36.2 Date of communication.................................................................................................................... 82

36.3 Addresses for communication.......................................................................................................... 82

ARTICLE 37 — INTERPRETATION OF THE AGREEMENT.....................................................................83

ARTICLE 38 — CALCULATION OF PERIODS AND DEADLINES..........................................................83

ARTICLE 39 — AMENDMENTS.................................................................................................................. 83

39.1 Conditions......................................................................................................................................... 83

39.2 Procedure.......................................................................................................................................... 83

ARTICLE 40 — ACCESSION AND ADDITION OF NEW BENEFICIARIES........................................... 84

40.1 Accession of the beneficiaries mentioned in the Preamble............................................................. 84

40.2 Addition of new beneficiaries.......................................................................................................... 84

ARTICLE 41 — TRANSFER OF THE AGREEMENT................................................................................. 84

ARTICLE 42 — ASSIGNMENTS OF CLAIMS FOR PAYMENT AGAINST THE GRANTING AUTHORITY.................................................................................................................................85

ARTICLE 43 — APPLICABLE LAW AND SETTLEMENT OF DISPUTES.............................................. 85

43.1 Applicable law..................................................................................................................................85

43.2 Dispute settlement............................................................................................................................ 85

ARTICLE 44 — ENTRY INTO FORCE.........................................................................................................85

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DATA SHEET

1.  General data

Project summary:

Project summary

The European Rare Diseases Research Alliance (ERDERA) aims to improve the health and well-being of the 30 million people living with a rare disease in Europe, by making Europe a world leader in Rare Disease (RD) research and innovation, to support concrete health benefits to rare disease patients, through better prevention, diagnosis and treatment. This Partnership will deliver a RD ecosystem that builds on the successes of previous programmes by supporting robust patient need-led research, developing new diagnostic methods and pathways, spearheading the digital transformational change connecting the dots between care, patient data and research, while ensuring strong alignment of strategies in RD research across countries and regions. Structuring goal-oriented public-private collaborations targeted at interventions all along the R&D value chain will ensure that the journey from knowledge to patient impact is expedited, thereby optimising EU innovation potential in RD. To support its ambition and missions ERDERA has been designed as a comprehensive and integrated ecosystem of which structure can be compared to an institute encompassing three main parts: (i) funding, (ii) internal (in house) Clinical Research Network that implements research activities targeting clinical trial readiness of RDs and accelerating diagnosis and translation of research discovery into improved patient care, and (iii) related supporting services (Data, Expertise, Education and Training) as well as an acceleration hub that serve external and internal RD community, all supported by all-embracing coordination and strategy and foundational (inter)national alignment.

Keywords:

– Rare diseases – translational research, diagnostics, therapy development, ATMPs, data, education, patients empowerment, public-

private collaboration, federated analysis, regulatory compliance, clinical trials

Project number: 101156595

Project name: EUROPEAN RARE DISEASES RESEARCH ALLIANCE

Project acronym: ERDERA

Call: HORIZON-HLTH-2023-DISEASE-07

Topic: HORIZON-HLTH-2023-DISEASE-07-01

Type of action: HORIZON Programme Cofund Actions

Granting authority: European Health and Digital Executive Agency

Grant managed through EU Funding & Tenders Portal: Yes (eGrants)

Project starting date: fixed date: 1 September 2024

Project end date: 31 August 2031

Project duration: 84 months

Consortium agreement: Yes

2. Participants

List of participants:

N° Role Short name Legal name Ctry PIC Total

eligible costs (BEN and AE)

Max grant amount

1 COO INSERM INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

FR 999997833 9 032 651.75 4 516 325.88

1.1 AE CAD Collecteur Analyseur de Données FR 880793078 716 000.00 358 000.00

2 BEN TEAMIT TEAMIT RESEARCH SL ES 896104237 1 493 375.00 746 687.50

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N° Role Short name Legal name Ctry PIC Total

eligible costs (BEN and AE)

Max grant amount

3 BEN DLR DEUTSCHES ZENTRUM FUR LUFT - UND RAUMFAHRT EV

DE 999981731 554 051.25 277 025.63

4 BEN ANR AGENCE NATIONALE DE LA RECHERCHE FR 998711613 13 041 861.75 3 907 279.49

5 BEN FTELE FONDAZIONE TELETHON ETS IT 999448716 3 628 310.00 1 401 160.30

6 BEN LMT Lietuvos mokslo taryba LT 997808446 465 802.25 152 891.05

7 BEN ZonMw ZORGONDERZOEK NEDERLAND ZON NL 999544164 5 940 632.50 1 990 795.26

8 BEN UT EBERHARD KARLS UNIVERSITAET TUEBINGEN DE 999991916 2 942 328.75 1 471 164.38

9 BEN UTARTU TARTU ULIKOOL EE 999895013 450 627.50 225 313.75

10 BEN CNAG CONSORCIO PARA LA EXPLOTACION DEL CENTRO NACIONAL DE ANALISIS GENOMICO

ES 882802433 1 962 785.71 981 392.86

11 BEN SRUMC STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM

NL 892057785 3 163 050.00 1 581 525.00

12 BEN TUM TECHNISCHE UNIVERSITAET MUENCHEN DE 999977463 683 125.00 341 562.50

13 BEN UKHD UNIVERSITATSKLINIKUM HEIDELBERG DE 999841081 3 181 611.25 1 590 805.63

14 BEN C-PATH CRITICAL PATH INSTITUTE STICHTING NL 887895418 924 601.25 462 300.63

15 BEN VHIR FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA

ES 999541642 1 543 050.00 771 525.00

16 BEN LUMC ACADEMISCH ZIEKENHUIS LEIDEN NL 999990849 1 297 155.00 648 577.50

17 BEN UTWENTE UNIVERSITEIT TWENTE NL 999900833 377 120.00 188 560.00

18 BEN UPM UNIVERSIDAD POLITECNICA DE MADRID ES 999974844 1 076 878.75 538 439.38

19 BEN UMCG ACADEMISCH ZIEKENHUIS GRONINGEN NL 999914801 1 593 566.25 796 783.13

20 BEN AUMC STICHTING AMSTERDAM UMC NL 919322739 668 798.75 334 399.38

21 BEN UM UNIVERSITEIT MAASTRICHT NL 999975911 367 325.00 183 662.50

22 BEN EATRIS EATRIS ERIC NL 941506445 1 016 710.00 508 355.00

23 BEN FGB FONDAZIONE PER LA RICERCA FARMACOLOGICA GIANNI BENZI ONLUS

IT 962478330 798 285.00 399 142.50

24 BEN APHP ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR 999645432 1 616 832.50 808 416.25

25 BEN UHASSELT UNIVERSITEIT HASSELT BE 999874934 294 187.50 147 093.75

26 BEN EURORDIS EURORDIS - RARE DISEASES EUROPE FR 965288323 1 622 066.25 811 033.13

27 BEN VULSK VIESOJI ISTAIGA VILNIAUS UNIVERSITETO LIGONINE SANTAROS KLINIKOS

LT 991636530 732 225.00 366 112.50

28 BEN UC UNIVERSIDADE DE COIMBRA PT 997826391 758 030.00 379 015.00

29 BEN FFRD FONDATION MALADIES RARES FR 954438485 1 966 776.25 983 388.13

30 BEN AFM ASS FRANCAISE CONTRE LES MYOPATHIES FR 959828387 364 500.00 182 250.00

31 BEN CCUH BERNU KLINISKA UNIVERSITATES SLIMNICA VALSTS SIA

LV 904374748 235 605.00 117 802.50

32 BEN AICIB AICIB - AGENCIA DE INVESTIGACAO CLINICA E INOVACAO BIOMEDICA

PT 892687606 160 420.00 80 210.00

33 BEN IMGGE INSTITUT ZA MOLEKULARNU GENETIKU I GENETICKO INZENJERSTVO

RS 986427921 56 447.50 28 223.75

34 BEN RDI RARE DISEASES INTERNATIONAL FR 881348694 222 375.00 111 187.50

35 BEN ACU ACIBADEM MEHMET ALI AYDINLAR UNIVERSITY TR 959704421 17 946.25 8 973.13

36 BEN AIT AIT AUSTRIAN INSTITUTE OF TECHNOLOGY GMBH AT 999584128 282 503.75 141 251.88

37 BEN AMU UNIVERSITE D'AIX MARSEILLE FR 955518483 221 450.00 110 725.00

38 BEN AOU Meyer IRCCS

AZIENDA OSPEDALIERA UNIVERSITARIA MEYER IRCCS

IT 998778543 76 875.00 38 437.50

39 BEN ASU-FC AZIENDA SANITARIA UNIVERSITARIA FRIULI CENTRALE

IT 894464355 330 225.00 165 112.50

40 BEN BIU BAR ILAN UNIVERSITY IL 999886574 99 617.50 49 808.75

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N° Role Short name Legal name Ctry PIC Total

eligible costs (BEN and AE)

Max grant amount

41 BEN BMBF BUNDESMINISTERIUM FUER BILDUNG UND FORSCHUNG

DE 999835455 9 008 750.00 2 683 005.99

42 BEN CCRI GmbH ST. ANNA KINDERKREBSFORSCHUNG GMBH AT 891384896 307 350.00 153 675.00

43 BEN CENTOGENE CENTOGENE GMBH DE 878333449 150 000.00 75 000.00

44 BEN CHARITE CHARITE - UNIVERSITAETSMEDIZIN BERLIN DE 999992692 656 251.25 328 125.63

45 BEN CHI CHECKIMMUNE GMBH DE 889852781 262 998.75 131 499.38

46 BEN CHU Dijon CENTRE HOSPITALIER REG UNIVERSITAIRE DIJON FR 959994451 223 125.00 111 562.50

47 BEN CE COPENHAGEN ECONOMICS AS DK 938801309 506 967.50 253 483.75

48 BEN REGIONH REGION HOVEDSTADEN DK 999654744 150 000.00 75 000.00

49 BEN CSO-MOH MINISTRY OF HEALTH IL 999596156 1 145 100.00 434 008.01

50 BEN CVBF CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE

IT 999717018 277 275.00 138 637.50

51 BEN DDF STICHTING DUCHENNE DATA FOUNDATION NL 899914882 284 825.00 142 412.50

52 BEN ELS ELSEVIER BV NL 999937014 421 200.00 210 600.00

53 BEN Erasmus MC ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM

NL 999988424 817 978.75 408 989.38

54 BEN F.R.S.-FNRS FONDS DE LA RECHERCHE SCIENTIFIQUE- FNRS BE 999540381 934 575.00 283 821.89

55 BEN FHG FRAUNHOFER GESELLSCHAFT ZUR FORDERUNG DER ANGEWANDTEN FORSCHUNG EV

DE 999984059 765 065.63 382 532.82

56 BEN FRRB FONDAZIONE REGIONALE PER LA RICERCA BIOMEDICA

IT 929009450 4 532 575.00 1 282 839.44

57 BEN ECRIN ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK

FR 948646712 57 850.00 28 925.00

58 BEN FWF FONDS ZUR FÖRDERUNG DER WISSENSCHAFTLICHEN FORSCHUNG

AT 998735960 2 747 575.00 794 930.67

59 BEN IFD INNOVATIONSFONDEN DK 939076692 3 031 475.00 864 008.80

60 BEN FSJD-CERCA FUNDACIO PRIVADA PER A LA RECERCA I LA DOCENCIA SANT JOAN DE DEU

ES 999565601 201 382.50 100 691.25

61 BEN FCT FUNDACAO PARA A CIENCIA E A TECNOLOGIA PT 999543970 1 073 950.00 316 043.45

62 BEN MZd MINISTERSTVO ZDRAVOTNICTVI CESKE REPUBLIKY

CZ 924144512 1 505 000.00 439 620.90

62.1 AE AZV AGENTURA PRO ZDRAVOTNICKY VYZKUM CESKE REPUBLIKY AZV CR

CZ 878890229 16 422.50 8 211.25

63 BEN GNT ASSOCIATION GENETHON FR 998217883 897 927.50 448 963.75

64 BEN GERAD GEORGIAN FOUNDATION FOR GENETIC AND RARE DISEASES

GE 953089312 40 718.75 20 359.38

65 BEN RS REGION STOCKHOLM SE 999522921 37 875.00 18 937.50

66 BEN GUF JOHANN WOLFGANG GOETHE-UNIVERSITAET FRANKFURT AM MAIN

DE 999978724 82 976.25 41 488.13

67 BEN HRB THE HEALTH RESEARCH BOARD IE 999546686 1 699 415.00 597 314.15

68 BEN İBG IZMIR BIYOTIP VE GENOM MERKEZI TR 906758620 444 621.25 222 310.63

69 BEN Rannis RANNSOKNAMIDSTOD ISLANDS IS 999547268 463 146.00 150 500.42

70 BEN IMAGINE IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION

FR 955276177 472 423.50 236 211.75

71 BEN IGC PAN INSTYTUT GENETYKI CZLOWIEKA POLSKIEJ AKADEMII NAUK

PL 982958425 52 500.00 26 250.00

72 BEN ISCIII INSTITUTO DE SALUD CARLOS III ES 999507886 5 425 418.75 1 899 967.27

73 BEN INSA INSTITUTO NACIONAL DE SAUDE DR. RICARDO JORGE

PT 998308190 88 560.00 44 280.00

74 BEN IOR ISTITUTO ORTOPEDICO RIZZOLI IT 999445709 32 500.00 16 250.00

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eligible costs (BEN and AE)

Max grant amount

75 BEN ISS ISTITUTO SUPERIORE DI SANITA IT 999978821 148 062.50 74 031.25

76 BEN IT-MOH MINISTERO DELLA SALUTE IT 999531942 3 033 325.00 865 673.80

77 BEN KU Leuven KATHOLIEKE UNIVERSITEIT LEUVEN BE 999991334 535 041.25 267 520.63

78 BEN LZP LATVIJAS ZINATNES PADOME LV 999546589 322 075.00 103 435.63

79 BEN MRT MAPI RESEARCH TRUST FR 925695542 276 700.00 138 350.00

80 BEN MUS MEDICAL UNIVERSITY SOFIA BG 999857571 52 500.00 26 250.00

81 BEN MUH FAKULTNI NEMOCNICE V MOTOLE CZ 964458391 313 600.00 156 800.00

82 BEN MUR Ministero dell'università e della ricerca IT 894763406 3 028 325.00 861 173.80

83 BEN NCBR NARODOWE CENTRUM BADAN I ROZWOJU PL 999519720 3 625 825.00 1 026 060.06

84 BEN NKUA ETHNIKO KAI KAPODISTRIAKO PANEPISTIMIO ATHINON

EL 999643007 52 500.00 26 250.00

85 BEN MoH SR MINISTERSTVO ZDRAVOTNICTVA SLOVENSKEJ REPUBLIKY

SK 999825173 112 350.00 56 175.00

86 BEN RDG ENOSI SPANION ASTHENON ELLADOS EL 883276763 79 850.00 39 925.00

87 BEN REGIONSYD REGION SYDDANMARK DK 999602073 150 000.00 75 000.00

88 BEN OPBG OSPEDALE PEDIATRICO BAMBINO GESU IT 998837810 174 375.00 87 187.50

89 BEN OSR OSPEDALE SAN RAFFAELE SRL IT 953176030 628 577.50 314 288.75

90 BEN JSI INSTITUT JOZEF STEFAN SI 999971837 77 850.00 38 925.00

91 BEN RCN NORGES FORSKNINGSRAD NO 999460453 1 581 182.00 464 271.41

92 BEN FWO FONDS VOOR WETENSCHAPPELIJK ONDERZOEK- VLAANDEREN

BE 997949484 2 139 575.00 620 594.41

93 BEN REUH RIGAS AUSTRUMU KLINISKA UNIVERSITATES SLIMNICA SIA

LV 973366483 189 825.00 94 912.50

94 BEN RIF IDRYMA EREVNAS KAI KAINOTOMIAS CY 999946035 3 038 457.50 870 292.51

95 BEN RCSI ROYAL COLLEGE OF SURGEONS IN IRELAND IE 999867368 76 875.00 38 437.50

96 BEN RSU RIGAS STRADINA UNIVERSITATE LV 999843118 30 860.00 15 430.00

97 BEN Sciensano SCIENSANO BE 906160809 406 875.00 203 437.50

98 BEN SPW SERVICE PUBLIC DE WALLONIE BE 999811884 3 039 575.00 871 298.80

99 BEN SFU SIGMUND FREUD PRIVATUNIVERSITAT WIEN GMBH AT 996709824 276 387.50 138 193.75

100 BEN ETAg SIHTASUTUS EESTI TEADUSAGENTUUR EE 998483760 170 825.00 60 526.56

101 BEN SAS SLOVENSKA AKADEMIA VIED SK 999530390 140 825.00 52 169.75

102 BEN Sonio SONIO FR 886915427 226 075.00 113 037.50

103 BEN MOSAE SOTSIAALMINISTEERIUM EE 998429731 171 325.00 60 976.56

104 BEN SRC VETENSKAPSRADET - SWEDISH RESEARCH COUNCIL

SE 999586165 1 491 983.00 437 258.38

105 BEN TEDDY TEDDY - EUROPEAN NETWORK OF EXCELLENCE FOR PAEDIATRIC CLINICAL RESEARCH

IT 910591963 71 800.00 35 900.00

106 BEN TEKKARE TEKKARE FR 881967069 540 000.00 270 000.00

107 BEN TIF THALASSAEMIA INTERNATIONAL FEDERATION CY 940669626 74 850.00 37 425.00

108 BEN TUBITAK TURKIYE BILIMSEL VE TEKNOLOJIK ARASTIRMA KURUMU

TR 999587135 1 545 275.00 458 588.15

109 BEN RT (TuscReg) REGIONE TOSCANA IT 998823842 932 217.50 281 700.20

110 BEN UAB UNIVERSITAT AUTONOMA DE BARCELONA ES 999986484 189 805.00 94 902.50

111 BEN UCD UNIVERSITY COLLEGE DUBLIN, NATIONAL UNIVERSITY OF IRELAND, DUBLIN

IE 999974359 58 130.00 29 065.00

112 BEN UCSC UNIVERSITA CATTOLICA DEL SACRO CUORE IT 999915771 162 463.75 81 231.88

113 BEN UEF ITA-SUOMEN YLIOPISTO FI 991207984 287 760.00 143 880.00

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N° Role Short name Legal name Ctry PIC Total

eligible costs (BEN and AE)

Max grant amount

114 BEN UKA UNIVERSITAETSKLINIKUM AACHEN DE 999897632 128 062.50 64 031.25

115 BEN UKLFR UNIVERSITAETSKLINIKUM FREIBURG DE 999881918 400 923.75 200 461.88

116 BEN UNICAMPANIA UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI

IT 999848356 76 875.00 38 437.50

117 BEN UoC PANEPISTIMIO KRITIS EL 999588978 52 500.00 26 250.00

118 BEN UGENT UNIVERSITEIT GENT BE 999986096 198 750.00 99 375.00

119 BEN UKCL UNIVERZITETNI KLINICNI CENTER LJUBLJANA SI 999882306 76 875.00 38 437.50

120 BEN WDO STICHTING WORLD DUCHENNE ORGANIZATION NL 930321860 193 078.75 96 539.38

121 BEN UU UPPSALA UNIVERSITET SE 999985029 493 887.50 246 943.75

122 BEN c4c-S CONECT4CHILDREN STICHTING NL 881618063 106 621.25 53 310.63

123 BEN CAU UNIVERSITE CADI AYYAD MA 998639930 68 993.75 34 496.88

124 BEN CNRS CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS

FR 999997930 212 352.50 106 176.25

125 BEN CIBER CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P.

ES 997154957 325 196.25 162 598.13

126 BEN UEFISCDI UNITATEA EXECUTIVA PENTRU FINANTAREA INVATAMANTULUI SUPERIOR A CERCETARII DEZVOLTARII SI INOVARII

RO 972130024 3 031 775.00 864 278.80

127 BEN UNIROMA1 UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA IT 999987745 85 178.75 42 589.38

128 BEN UANTWERPEN UNIVERSITEIT ANTWERPEN BE 999902870 51 250.00 25 625.00

129 BEN BNSF BULGARIAN NATIONAL SCIENCE FUND BG 984222335 253 325.00 88 168.60

130 BEN Aalborg UH REGION NORDJYLLAND (NORTH DENMARK REGION)

DK 997381064 52 500.00 26 250.00

131 BEN RDB REDKI BOLESTI BULGARIA BG 880694720 32 325.00 16 162.50

132 BEN BBMRI-ERIC BIOBANKS AND BIOMOLECULAR RESOURCES RESEARCH INFRASTRUCTURE CONSORTIUM (BBMRI-ERIC)

AT 946597878 111 878.75 55 939.38

133 BEN AarhusUH AARHUS UNIVERSITETSHOSPITAL DK 999643880 76 875.00 38 437.50

134 BEN IABS-EU ASSOCIATION INTERNATIONALE DE STANDARDISATION BIOLOGIQUE POUR L'EUROPE(IABS-EU)

FR 934774936 98 812.50 49 406.25

135 BEN HRCI HEALTH RESEARCH CHARITIES IRELAND COMPANY LIMITED BY GUARANTEE

IE 880683274 47 625.00 23 812.50

136 BEN LMU LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN DE 999978433 0.00 0.00

136.1 AE KUM KLINIKUM DER UNIVERSITAT MUNCHEN DE 995625946 631 666.25 315 833.13

137 BEN MSW MYSCIENCEWORK FR 898132119 315 000.00 157 500.00

138 BEN FNR FONDS NATIONAL DE LA RECHERCHE LU 998597153 1 029 315.00 313 158.33

139 BEN NKFIH NEMZETI KUTATASI FEJLESZTESI ES INNOVACIOS HIVATAL

HU 999578696 922 075.00 272 571.89

140 BEN PEI BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL

DE 998217301 257 069.38 128 534.69

141 BEN PLUS PARIS-LODRON-UNIVERSITAT SALZBURG AT 999868047 317 472.50 158 736.25

142 BEN SU SORBONNE UNIVERSITE FR 909875521 24 062.50 12 031.25

143 BEN UNISI UNIVERSITA DEGLI STUDI DI SIENA IT 999898020 76 875.00 38 437.50

144 BEN Vinnova VERKET FOR INNOVATIONSSYSTEM SE 999618757 1 447 611.00 425 956.62

145 BEN CRG-CERCA FUNDACIO CENTRE DE REGULACIO GENOMICA ES 999544455 141 750.00 70 875.00

146 BEN GMS-RS REGION SKANE SE 998165794 37 875.00 18 937.50

147 BEN VIB VIB VZW BE 999651931 25 625.00 12 812.50

148 BEN IPG INSTITUT DE PATHOLOGIE ET DE GENETIQUE ASBL BE 878266034 52 500.00 26 250.00

149 BEN UO UNIVERSITY OF OTAGO NZ 998331567 63 600.00 31 800.00

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N° Role Short name Legal name Ctry PIC Total

eligible costs (BEN and AE)

Max grant amount

150 BEN HSJD HOSPITAL SANT JOAN DE DEU ES 997929890 65 000.00 32 500.00

151 AP AZ ASTRAZENECA AB SE 999941379 0.00 0.00

152 AP CHEO-RI CHILDREN'S HOSPITAL OF EASTERN ONTARIO RESEARCH INSTITUTE INC

CA 907681187 0.00 0.00

153 AP CIHR CANADIAN INSTITUTES OF HEALTH RESEARCH CA 960368386 0.00 0.00

154 AP DFG DEUTSCHE FORSCHUNGSGEMEINSCHAFT EV DE 999547462 0.00 0.00

155 AP FDB FUJIFILM DIOSYNTH BIOTECHNOLOGIES UK LIMITED

UK 962770494 0.00 0.00

156 AP FRQS FONDS DE RECHERCHE DU QUEBEC - SANTE CA 963122410 0.00 0.00

157 AP HIPRA HIPRA HUMAN HEALTH SL ES 890821326 0.00 0.00

158 AP LAB HIPRA LABORATORIOS HIPRA SA ES 991274041 0.00 0.00

159 AP MICYRN MICYRN CA 880745451 0.00 0.00

160 AP Miltenyi MILTENYI BIOTEC BV & CO KG DE 897602402 0.00 0.00

161 AP (IO)

Pfizer PFIZER INC US 887211374 0.00 0.00

162 AP RCC DEPARTMENT OF HEALTH GOVERNMENT OF WESTERN AUSTRALIA

AU 960480421 0.00 0.00

163 AP REI REITHERA SRL IT 998129419 0.00 0.00

164 AP RJF LABORATORIO REIG JOFRE SA ES 951005073 0.00 0.00

165 AP ROCHE F. HOFFMANN-LA ROCHE AG CH 999601782 0.00 0.00

166 AP SNSF SCHWEIZERISCHER NATIONALFONDS ZUR FORDERUNG DER WISSENSCHAFTLICHEN FORSCHUNG

CH 999540575 0.00 0.00

167 AP UCB UCB BIOPHARMA BE 937666409 0.00 0.00

168 AP Fraunhofer UK FRAUNHOFER UK RESEARCH LIMITED UK 952487039 0.00 0.00

169 AP GA UK GENETIC ALLIANCE UK LTD UK 999790932 0.00 0.00

170 AP UNEW UNIVERSITY OF NEWCASTLE UPON TYNE UK 999985417 0.00 0.00

171 AP CUH Cambridge University Hospitals NHS Foundation Trust UK 991793961 0.00 0.00

172 AP ULEIC UNIVERSITY OF LEICESTER UK 999985514 0.00 0.00

173 AP QUB-UK THE QUEEN'S UNIVERSITY OF BELFAST UK 999992013 0.00 0.00

174 AP UCL UNIVERSITY COLLEGE LONDON UK 999975620 0.00 0.00

175 AP UCAM THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE

UK 999977172 0.00 0.00

Total 145 830 619.47 56 317 412.61

Coordinator:

– INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)

3. Grant

Maximum grant amount, total estimated eligible costs and contributions and funding rate:

Total eligible costs (BEN and AE)

Funding rate (%)

Maximum grant amount (Annex 2)

Maximum grant amount (award decision)

145 830 619.47 50 56 317 412.61 56 317 412.61

Grant form: Budget-based

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Grant mode: Action grant

Budget categories/activity types:

- A. Personnel costs - A.1 Employees, A.2 Natural persons under direct contract, A.3 Seconded persons - A.4 SME owners and natural person beneficiaries

- B. Subcontracting costs

- C. Purchase costs - C.1 Travel and subsistence - C.2 Equipment - C.3 Other goods, works and services

- D. Other cost categories - D.1 Financial support to third parties - D.2 Internally invoiced goods and services

- E. Indirect costs

Cost eligibility options:

- In-kind contributions eligible costs

- Parental leave

- Project-based supplementary payments

- Average personnel costs (unit cost according to usual cost accounting practices)

- Limitation for subcontracting

- Travel and subsistence: - Travel: Actual costs - Accommodation: Actual costs - Subsistence: Actual costs

- Equipment: depreciation only

- Costs for providing financial support to third parties (actual cost; max amount for each recipient: EUR 10 000 000.00)

- Indirect cost flat-rate: 25% of the eligible direct costs (categories A-D, except volunteers costs, subcontracting costs, financial support to third parties and exempted specific cost categories, if any)

- VAT: Yes

- Other ineligible costs

Budget flexibility: Yes (no flexibility cap)

4. Reporting, payments and recoveries

4.1 Continuous reporting (art 21)

Deliverables: see Funding & Tenders Portal Continuous Reporting tool

4.2 Periodic reporting and payments

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Reporting and payment schedule (art 21, 22):

Reporting Payments

Reporting periods Type Deadline Type Deadline (time to pay)

RP No Month from Month to

Initial prefinancing

30 days from entry into force/10 days

before starting date – whichever

is the latest

1 1 26 Periodic report 60 days after end of reporting period

Interim payment 90 days from receiving

periodic report

2 27 44 Periodic report 60 days after end of reporting period

Interim payment 90 days from receiving

periodic report

3 45 62 Periodic report 60 days after end of reporting period

Interim payment 90 days from receiving

periodic report

4 63 74 Periodic report 60 days after end of reporting period

Interim payment 90 days from receiving

periodic report

5 75 84 Periodic report 60 days after end of reporting period

Final payment 90 days from receiving

periodic report

Prefinancing payments and guarantees:

Prefinancing payment

Type Amount

Prefinancing 1 (initial) 22 526 965.04

Reporting and payment modalities (art 21, 22):

Mutual Insurance Mechanism (MIM): Yes

MIM contribution: 5% of the maximum grant amount (2 815 870.63), retained from the initial prefinancing

Restrictions on distribution of initial prefinancing: The prefinancing may be distributed only if the minimum number of beneficiaries set out in the call condititions (if any) have acceded to the Agreement and only to beneficiaries that have acceded.

Interim payment ceiling (if any): 90% of the maximum grant amount

Exception for revenues: Yes

No-profit rule: Yes

Late payment interest: ECB + 3.5%

Bank account for payments:

FR7610071750000000100507196

Conversion into euros: Double conversion

Reporting language: Language of the Agreement

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4.3 Certificates (art 24):

Certificates on the financial statements (CFS):

Conditions:

Schedule: only at final payment, if threshold is reached

Standard threshold (beneficiary-level):

- financial statement: requested EU contribution to costs ≥ EUR 430 000.00

Special threshold for beneficiaries with a systems and process audit(see Article 24): financial statement: requested EU contribution to costs ≥ EUR 725 000.00

4.4 Recoveries (art 22)

First-line liability for recoveries:

Beneficiary termination: Beneficiary concerned

Final payment: Each beneficiary for their own debt

After final payment: Beneficiary concerned

Joint and several liability for enforced recoveries (in case of non-payment):

Individual financial responsibility: Each beneficiary is liable only for its own debts (and those of its affiliated entities, if any)

Joint and several liability of affiliated entities — n/a

5. Consequences of non-compliance, applicable law & dispute settlement forum

Suspension and termination:

Additional suspension grounds (art 31)

Additional termination grounds (art 32)

Applicable law (art 43):

Standard applicable law regime: EU law + law of Belgium

Dispute settlement forum (art 43):

Standard dispute settlement forum:

EU beneficiaries: EU General Court + EU Court of Justice (on appeal)

Non-EU beneficiaries: Courts of Brussels, Belgium (unless an international agreement provides for the enforceability of EU court judgements)

6. Other

Specific rules (Annex 5): Yes

Standard time-limits after project end:

Confidentiality (for X years after final payment): 5

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Record-keeping (for X years after final payment): 5 (or 3 for grants of not more than EUR 60 000)

Reviews (up to X years after final payment): 2

Audits (up to X years after final payment): 2

Extension of findings from other grants to this grant (no later than X years after final payment): 2

Impact evaluation (up to X years after final payment): 5 (or 3 for grants of not more than EUR 60 000)

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CHAPTER 1 GENERAL

ARTICLE 1 — SUBJECT OF THE AGREEMENT

This Agreement sets out the rights and obligations and terms and conditions applicable to the grant awarded for the implementation of the action set out in Chapter 2.

ARTICLE 2 — DEFINITIONS

For the purpose of this Agreement, the following definitions apply:

Actions — The project which is being funded in the context of this Agreement.

Grant — The grant awarded in the context of this Agreement.

EU grants — Grants awarded by EU institutions, bodies, offices or agencies (including EU executive agencies, EU regulatory agencies, EDA, joint undertakings, etc.).

Participants — Entities participating in the action as beneficiaries, affiliated entities, associated partners, third parties giving in-kind contributions, subcontractors or recipients of financial support to third parties.

Beneficiaries (BEN) — The signatories of this Agreement (either directly or through an accession form).

Affiliated entities (AE) — Entities affiliated to a beneficiary within the meaning of Article 187 of EU Financial Regulation 2018/10464 which participate in the action with similar rights and obligations as the beneficiaries (obligation to implement action tasks and right to charge costs and claim contributions).

Associated partners (AP) — Entities which participate in the action, but without the right to charge costs or claim contributions.

Purchases — Contracts for goods, works or services needed to carry out the action (e.g. equipment, consumables and supplies) but which are not part of the action tasks (see Annex 1).

Subcontracting — Contracts for goods, works or services that are part of the action tasks (see Annex 1).

In-kind contributions — In-kind contributions within the meaning of Article 2(36) of EU Financial

4 For the definition, see Article 187 Regulation (EU, Euratom) 2018/1046 of the European Parliament and of the Council of 18 July 2018 on the financial rules applicable to the general budget of the Union, amending Regulations (EU) No 1296/2013, (EU) No 1301/2013, (EU) No 1303/2013, (EU) No 1304/2013, (EU) No 1309/2013, (EU) No 1316/2013, (EU) No 223/2014, (EU) No 283/2014, and Decision No 541/2014/EU and repealing Regulation (EU, Euratom) No 966/2012 (‘EU Financial Regulation’) (OJ L 193, 30.7.2018, p. 1): “affiliated entities [are]: (a) entities that form a sole beneficiary [(i.e. where an entity is formed of several entities that satisfy the criteria for

being awarded a grant, including where the entity is specifically established for the purpose of implementing an action to be financed by a grant)];

(b) entities that satisfy the eligibility criteria and that do not fall within one of the situations referred to in Article 136(1) and 141(1) and that have a link with the beneficiary, in particular a legal or capital link, which is neither limited to the action nor established for the sole purpose of its implementation”.

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Regulation 2018/1046, i.e. non-financial resources made available free of charge by third parties.

Fraud — Fraud within the meaning of Article 3 of EU Directive 2017/13715 and Article 1 of the Convention on the protection of the European Communities’ financial interests, drawn up by the Council Act of 26 July 19956, as well as any other wrongful or criminal deception intended to result in financial or personal gain.

Irregularities — Any type of breach (regulatory or contractual) which could impact the EU financial interests, including irregularities within the meaning of Article 1(2) of EU Regulation 2988/957.

Grave professional misconduct — Any type of unacceptable or improper behaviour in exercising one’s profession, especially by employees, including grave professional misconduct within the meaning of Article 136(1)(c) of EU Financial Regulation 2018/1046.

Applicable EU, international and national law — Any legal acts or other (binding or non-binding) rules and guidance in the area concerned.

Portal — EU Funding & Tenders Portal; electronic portal and exchange system managed by the European Commission and used by itself and other EU institutions, bodies, offices or agencies for the management of their funding programmes (grants, procurements, prizes, etc.).

CHAPTER 2 ACTION

ARTICLE 3 — ACTION

The grant is awarded for the action 101156595 — ERDERA (‘action’), as described in Annex 1.

ARTICLE 4 — DURATION AND STARTING DATE

The duration and the starting date of the action are set out in the Data Sheet (see Point 1).

CHAPTER 3 GRANT

ARTICLE 5 — GRANT

5.1 Form of grant

The grant is an action grant8 which takes the form of a budget-based mixed actual cost grant (i.e. a

5 Directive (EU) 2017/1371 of the European Parliament and of the Council of 5 July 2017 on the fight against fraud to the Union’s financial interests by means of criminal law (OJ L 198, 28.7.2017, p. 29).

6 OJ C 316, 27.11.1995, p. 48. 7 Council Regulation (EC, Euratom) No 2988/95 of 18 December 1995 on the protection of the European Communities

financial interests (OJ L 312, 23.12.1995, p. 1). 8 For the definition, see Article 180(2)(a) EU Financial Regulation 2018/1046: ‘action grant’ means an EU grant to

finance “an action intended to help achieve a Union policy objective”.

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grant based on actual costs incurred, but which may also include other forms of funding, such as unit costs or contributions, flat-rate costs or contributions, lump sum costs or contributions or financing not linked to costs).

5.2 Maximum grant amount

The maximum grant amount is set out in the Data Sheet (see Point 3) and in the estimated budget (Annex 2).

5.3 Funding rate

The funding rate for costs is 50% of the action’s eligible costs.

Contributions are not subject to any funding rate.

5.4 Estimated budget, budget categories and forms of funding

The estimated budget for the action is set out in Annex 2.

It contains the estimated eligible costs and contributions for the action, broken down by participant and budget category.

Annex 2 also shows the types of costs and contributions (forms of funding)9 to be used for each budget category.

If unit costs or contributions are used, the details on the calculation will be explained in Annex 2a.

5.5 Budget flexibility

The budget breakdown may be adjusted — without an amendment (see Article 39) — by transfers (between participants and budget categories), as long as this does not imply any substantive or important change to the description of the action in Annex 1.

However:

- changes to the budget category for volunteers (if used) always require an amendment

- changes to budget categories with lump sums costs or contributions (if used; including financing not linked to costs) always require an amendment

- changes to budget categories with higher funding rates or budget ceilings (if used) always require an amendment

- addition of amounts for subcontracts not provided for in Annex 1 either require an amendment or simplified approval in accordance with Article 6.2

- other changes require an amendment or simplified approval, if specifically provided for in Article 6.2

- flexibility caps: not applicable.

9 See Article 125 EU Financial Regulation 2018/1046.

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ARTICLE 6 — ELIGIBLE AND INELIGIBLE COSTS AND CONTRIBUTIONS

In order to be eligible, costs and contributions must meet the eligibility conditions set out in this Article.

6.1 General eligibility conditions

The general eligibility conditions are the following:

(a) for actual costs:

(i) they must be actually incurred by the beneficiary

(ii) they must be incurred in the period set out in Article 4 (with the exception of costs relating to the submission of the final periodic report, which may be incurred afterwards; see Article 21)

(iii) they must be declared under one of the budget categories set out in Article 6.2 and Annex 2

(iv) they must be incurred in connection with the action as described in Annex 1 and necessary for its implementation

(v) they must be identifiable and verifiable, in particular recorded in the beneficiary’s accounts in accordance with the accounting standards applicable in the country where the beneficiary is established and with the beneficiary’s usual cost accounting practices

(vi) they must comply with the applicable national law on taxes, labour and social security and

(vii) they must be reasonable, justified and must comply with the principle of sound financial management, in particular regarding economy and efficiency

(b) for unit costs or contributions (if any):

(i) they must be declared under one of the budget categories set out in Article 6.2 and Annex 2

(ii) the units must:

- be actually used or produced by the beneficiary in the period set out in Article 4 (with the exception of units relating to the submission of the final periodic report, which may be used or produced afterwards; see Article 21)

- be necessary for the implementation of the action and

(iii) the number of units must be identifiable and verifiable, in particular supported by records and documentation (see Article 20)

(c) for flat-rate costs or contributions (if any):

(i) they must be declared under one of the budget categories set out in Article 6.2 and Annex 2

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(ii) the costs or contributions to which the flat-rate is applied must:

- be eligible

- relate to the period set out in Article 4 (with the exception of costs or contributions relating to the submission of the final periodic report, which may be incurred afterwards; see Article 21)

(d) for lump sum costs or contributions (if any):

(i) they must be declared under one of the budget categories set out in Article 6.2 and Annex 2

(ii) the work must be properly implemented by the beneficiary in accordance with Annex 1

(iii) the deliverables/outputs must be achieved in the period set out in Article 4 (with the exception of deliverables/outputs relating to the submission of the final periodic report, which may be achieved afterwards; see Article 21)

(e) for unit, flat-rate or lump sum costs or contributions according to usual cost accounting practices (if any):

(i) they must fulfil the general eligibility conditions for the type of cost concerned

(ii) the cost accounting practices must be applied in a consistent manner, based on objective criteria, regardless of the source of funding

(f) for financing not linked to costs (if any): the results must be achieved or the conditions must be fulfilled as described in Annex 1.

In addition, for direct cost categories (e.g. personnel, travel & subsistence, subcontracting and other direct costs) only costs that are directly linked to the action implementation and can therefore be attributed to it directly are eligible. They must not include any indirect costs (i.e. costs that are only indirectly linked to the action, e.g. via cost drivers).

In-kind contributions provided by third parties free of charge may be declared as eligible direct costs by the beneficiaries which use them (under the same conditions as if they were their own, provided that they concern only direct costs and that the third parties and their in-kind contributions are set out in Annex 1 (or approved ex post in the periodic report, if their use does not entail changes to the Agreement which would call into question the decision awarding the grant or breach the principle of equal treatment of applicants; ‘simplified approval procedure’).

6.2 Specific eligibility conditions for each budget category

For each budget category, the specific eligibility conditions are as follows:

Direct costs

A. Personnel costs

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A.1 Costs for employees (or equivalent) are eligible as personnel costs if they fulfil the general eligibility conditions and are related to personnel working for the beneficiary under an employment contract (or equivalent appointing act) and assigned to the action.

They must be limited to salaries (including net payments during parental leave), social security contributions, taxes and other costs linked to the remuneration, if they arise from national law or the employment contract (or equivalent appointing act) and be calculated on the basis of the costs actually incurred, in accordance with the following method:

{daily rate for the person

multiplied by

number of day-equivalents worked on the action (rounded up or down to the nearest half-day)}.

The daily rate must be calculated as:

{annual personnel costs for the person

divided by

215}.

The number of day-equivalents declared for a person must be identifiable and verifiable (see Article 20).

The actual time spent on parental leave by a person assigned to the action may be deducted from the 215 days indicated in the above formula.

The total number of day-equivalents declared in EU grants, for a person for a year, cannot be higher than 215, minus time spent on parental leave (if any).

For personnel which receives supplementary payments for work in projects (project-based remuneration), the personnel costs must be calculated at a rate which:

- corresponds to the actual remuneration costs paid by the beneficiary for the time worked by the person in the action over the reporting period

- does not exceed the remuneration costs paid by the beneficiary for work in similar projects funded by national schemes (‘national projects reference’)

- is defined based on objective criteria allowing to determine the amount to which the person is entitled

and

- reflects the usual practice of the beneficiary to pay consistently bonuses or supplementary payments for work in projects funded by national schemes.

The national projects reference is the remuneration defined in national law, collective labour agreement or written internal rules of the beneficiary applicable to work in projects funded by national schemes.

If there is no such national law, collective labour agreement or written internal rules or if the project- based remuneration is not based on objective criteria, the national project reference will be the average

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remuneration of the person in the last full calendar year covered by the reporting period, excluding remuneration paid for work in EU actions.

If the beneficiary uses average personnel costs (unit cost according to usual cost accounting practices), the personnel costs must fulfil the general eligibility conditions for such unit costs and the daily rate must be calculated:

- using the actual personnel costs recorded in the beneficiary’s accounts and excluding any costs which are ineligible or already included in other budget categories; the actual personnel costs may be adjusted on the basis of budgeted or estimated elements, if they are relevant for calculating the personnel costs, reasonable and correspond to objective and verifiable information

and

- according to usual cost accounting practices which are applied in a consistent manner, based on objective criteria, regardless of the source of funding.

A.2 and A.3 Costs for natural persons working under a direct contract other than an employment contract and costs for seconded persons by a third party against payment are also eligible as personnel costs, if they are assigned to the action, fulfil the general eligibility conditions and:

(a) work under conditions similar to those of an employee (in particular regarding the way the work is organised, the tasks that are performed and the premises where they are performed) and

(b) the result of the work belongs to the beneficiary (unless agreed otherwise).

They must be calculated on the basis of a rate which corresponds to the costs actually incurred for the direct contract or secondment and must not be significantly different from those for personnel performing similar tasks under an employment contract with the beneficiary.

A.4 The work of SME owners for the action (i.e. owners of beneficiaries that are small and medium- sized enterprises10 not receiving a salary) or natural person beneficiaries (i.e. beneficiaries that are natural persons not receiving a salary) may be declared as personnel costs, if they fulfil the general eligibility conditions and are calculated as unit costs in accordance with the method set out in Annex 2a.

B. Subcontracting costs

Subcontracting costs for the action (including related duties, taxes and charges, such as non- deductible or non-refundable value added tax (VAT)) are eligible, if they are calculated on the basis of the costs actually incurred, fulfil the general eligibility conditions and are awarded using the

10 For the definition, see Commission Recommendation 2003/361/EC: micro, small or medium-sized enterprise (SME) are enterprises

- engaged in an economic activity, irrespective of their legal form (including, in particular, self- employed persons and family businesses engaged in craft or other activities, and partnerships or associations regularly engaged in an economic activity) and

- employing fewer than 250 persons (expressed in ‘annual working units’ as defined in Article 5 of the Recommendation) and which have an annual turnover not exceeding EUR 50 million, and/or an annual balance sheet total not exceeding EUR 43 million.

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beneficiary’s usual purchasing practices — provided these ensure subcontracts with best value for money (or if appropriate the lowest price) and that there is no conflict of interests (see Article 12).

Beneficiaries that are ‘contracting authorities/entities’ within the meaning of the EU Directives on public procurement must also comply with the applicable national law on public procurement.

Subcontracting may cover only a limited part of the action.

The tasks to be subcontracted and the estimated cost for each subcontract must be set out in Annex 1 and the total estimated costs of subcontracting per beneficiary must be set out in Annex 2 (or may be approved ex post in the periodic report, if the use of subcontracting does not entail changes to the Agreement which would call into question the decision awarding the grant or breach the principle of equal treatment of applicants; ‘simplified approval procedure’).

C. Purchase costs

Purchase costs for the action (including related duties, taxes and charges, such as non-deductible or non-refundable value added tax (VAT)) are eligible if they fulfil the general eligibility conditions and are bought using the beneficiary’s usual purchasing practices — provided these ensure purchases with best value for money (or if appropriate the lowest price) and that there is no conflict of interests (see Article 12).

Beneficiaries that are ‘contracting authorities/entities’ within the meaning of the EU Directives on public procurement must also comply with the applicable national law on public procurement.

C.1 Travel and subsistence

Purchases for travel, accommodation and subsistence must be calculated as follows:

- travel: on the basis of the costs actually incurred and in line with the beneficiary’s usual practices on travel

- accommodation: on the basis of the costs actually incurred and in line with the beneficiary’s usual practices on travel

- subsistence: on the basis of the costs actually incurred and in line with the beneficiary’s usual practices on travel .

C.2 Equipment

Purchases of equipment, infrastructure or other assets used for the action must be declared as depreciation costs, calculated on the basis of the costs actually incurred and written off in accordance with international accounting standards and the beneficiary’s usual accounting practices.

Only the portion of the costs that corresponds to the rate of actual use for the action during the action duration can be taken into account.

Costs for renting or leasing equipment, infrastructure or other assets are also eligible, if they do not exceed the depreciation costs of similar equipment, infrastructure or assets and do not include any financing fees.

C.3 Other goods, works and services

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Purchases of other goods, works and services must be calculated on the basis of the costs actually incurred.

Such goods, works and services include, for instance, consumables and supplies, promotion, dissemination, protection of results, translations, publications, certificates and financial guarantees, if required under the Agreement.

D. Other cost categories

D.1 Financial support to third parties

Costs for providing financial support to third parties (in the form of grants, prizes or similar forms of support; if any) are eligible, if and as declared eligible in the call conditions, if they fulfil the general eligibility conditions, are calculated on the basis of the costs actually incurred and the support is implemented in accordance with the conditions set out in Annex 1.

These conditions must ensure objective and transparent selection procedures and include at least the following:

(a) for grants (or similar):

(i) the maximum amount of financial support for each third party (‘recipient’); this amount may not exceed the amount set out in the Data Sheet (see Point 3) or otherwise agreed with the granting authority

(ii) the criteria for calculating the exact amount of the financial support

(iii) the different types of activity that qualify for financial support, on the basis of a closed list

(iv) the persons or categories of persons that will be supported and

(v) the criteria and procedures for giving financial support

(b) for prizes (or similar):

(i) the eligibility and award criteria

(ii) the amount of the prize and

(iii) the payment arrangements.

This cost will not be taken into account for the indirect cost flat-rate.

D.2 Internally invoiced goods and services

Costs for internally invoiced goods and services directly used for the action may be declared as unit cost according to usual cost accounting practices, if and as declared eligible in the call conditions, if they fulfil the general eligibility conditions for such unit costs and the amount per unit is calculated:

- using the actual costs for the good or service recorded in the beneficiary’s accounts, attributed either by direct measurement or on the basis of cost drivers, and excluding any cost which are ineligible or already included in other budget categories; the actual costs may be adjusted

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on the basis of budgeted or estimated elements, if they are relevant for calculating the costs, reasonable and correspond to objective and verifiable information

and

- according to usual cost accounting practices which are applied in a consistent manner, based on objective criteria, regardless of the source of funding.

‘Internally invoiced goods and services’ means goods or services which are provided within the beneficiary’s organisation directly for the action and which the beneficiary values on the basis of its usual cost accounting practices.

This cost will not be taken into account for the indirect cost flat-rate.

Indirect costs

E. Indirect costs

Indirect costs will be reimbursed at the flat-rate of 25% of the eligible direct costs (categories A-D, except volunteers costs, subcontracting costs, financial support to third parties and exempted specific cost categories, if any).

Contributions

Not applicable

6.3 Ineligible costs and contributions

The following costs or contributions are ineligible:

(a) costs or contributions that do not comply with the conditions set out above (Article 6.1 and 6.2), in particular:

(i) costs related to return on capital and dividends paid by a beneficiary

(ii) debt and debt service charges

(iii) provisions for future losses or debts

(iv) interest owed

(v) currency exchange losses

(vi) bank costs charged by the beneficiary’s bank for transfers from the granting authority

(vii) excessive or reckless expenditure

(viii) deductible or refundable VAT (including VAT paid by public bodies acting as public authority)

(ix) costs incurred or contributions for activities implemented during grant agreement suspension (see Article 31)

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(x) in-kind contributions by third parties: not applicable

(b) costs or contributions declared under other EU grants (or grants awarded by an EU Member State, non-EU country or other body implementing the EU budget), except for the following cases:

(i) Synergy actions: not applicable

(ii) if the action grant is combined with an operating grant11 running during the same period and the beneficiary can demonstrate that the operating grant does not cover any (direct or indirect) costs of the action grant

(c) costs or contributions for staff of a national (or regional/local) administration, for activities that are part of the administration’s normal activities (i.e. not undertaken only because of the grant)

(d) costs or contributions (especially travel and subsistence) for staff or representatives of EU institutions, bodies or agencies

(e) other :

(i) country restrictions for eligible costs: not applicable

(ii) costs or contributions declared specifically ineligible in the call conditions.

6.4 Consequences of non-compliance

If a beneficiary declares costs or contributions that are ineligible, they will be rejected (see Article 27).

This may also lead to other measures described in Chapter 5.

CHAPTER 4 GRANT IMPLEMENTATION

SECTION 1 CONSORTIUM: BENEFICIARIES, AFFILIATED ENTITIES AND OTHER PARTICIPANTS

ARTICLE 7 — BENEFICIARIES

The beneficiaries, as signatories of the Agreement, are fully responsible towards the granting authority for implementing it and for complying with all its obligations.

They must implement the Agreement to their best abilities, in good faith and in accordance with all the obligations and terms and conditions it sets out.

They must have the appropriate resources to implement the action and implement the action under their own responsibility and in accordance with Article 11. If they rely on affiliated entities or other

11 For the definition, see Article 180(2)(b) of EU Financial Regulation 2018/1046: ‘operating grant’ means an EU grant to finance “the functioning of a body which has an objective forming part of and supporting an EU policy”.

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participants (see Articles 8 and 9), they retain sole responsibility towards the granting authority and the other beneficiaries.

They are jointly responsible for the technical implementation of the action. If one of the beneficiaries fails to implement their part of the action, the other beneficiaries must ensure that this part is implemented by someone else (without being entitled to an increase of the maximum grant amount and subject to an amendment; see Article 39). The financial responsibility of each beneficiary in case of recoveries is governed by Article 22.

The beneficiaries (and their action) must remain eligible under the EU programme funding the grant for the entire duration of the action. Costs and contributions will be eligible only as long as the beneficiary and the action are eligible.

The internal roles and responsibilities of the beneficiaries are divided as follows:

(a) Each beneficiary must:

(i) keep information stored in the Portal Participant Register up to date (see Article 19)

(ii) inform the granting authority (and the other beneficiaries) immediately of any events or circumstances likely to affect significantly or delay the implementation of the action (see Article 19)

(iii) submit to the coordinator in good time:

- the prefinancing guarantees (if required; see Article 23)

- the financial statements and certificates on the financial statements (CFS) (if required; see Articles 21 and 24.2 and Data Sheet, Point 4.3)

- the contribution to the deliverables and technical reports (see Article 21)

- any other documents or information required by the granting authority under the Agreement

(iv) submit via the Portal data and information related to the participation of their affiliated entities.

(b) The coordinator must:

(i) monitor that the action is implemented properly (see Article 11)

(ii) act as the intermediary for all communications between the consortium and the granting authority, unless the Agreement or granting authority specifies otherwise, and in particular:

- submit the prefinancing guarantees to the granting authority (if any)

- request and review any documents or information required and verify their quality and completeness before passing them on to the granting authority

- submit the deliverables and reports to the granting authority

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- inform the granting authority about the payments made to the other beneficiaries (report on the distribution of payments; if required, see Articles 22 and 32)

(iii) distribute the payments received from the granting authority to the other beneficiaries without unjustified delay (see Article 22).

The coordinator may not delegate or subcontract the above-mentioned tasks to any other beneficiary or third party (including affiliated entities).

However, coordinators which are public bodies may delegate the tasks set out in Point (b)(ii) last indent and (iii) above to entities with ‘authorisation to administer’ which they have created or which are controlled by or affiliated to them. In this case, the coordinator retains sole responsibility for the payments and for compliance with the obligations under the Agreement.

Moreover, coordinators which are ‘sole beneficiaries’12 (or similar, such as European research infrastructure consortia (ERICs)) may delegate the tasks set out in Point (b)(i) to (iii) above to one of their members. The coordinator retains sole responsibility for compliance with the obligations under the Agreement.

The beneficiaries must have internal arrangements regarding their operation and co-ordination, to ensure that the action is implemented properly.

If required by the granting authority (see Data Sheet, Point 1), these arrangements must be set out in a written consortium agreement between the beneficiaries, covering for instance:

- the internal organisation of the consortium

- the management of access to the Portal

- different distribution keys for the payments and financial responsibilities in case of recoveries (if any)

- additional rules on rights and obligations related to background and results (see Article 16)

- settlement of internal disputes

- liability, indemnification and confidentiality arrangements between the beneficiaries.

The internal arrangements must not contain any provision contrary to this Agreement.

ARTICLE 8 — AFFILIATED ENTITIES

The following entities which are linked to a beneficiary will participate in the action as ‘affiliated entities’:

- Collecteur Analyseur de Données (CAD), PIC 880793078, linked to INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)

12 For the definition, see Article 187(2) EU Financial Regulation 2018/1046: “Where several entities satisfy the criteria for being awarded a grant and together form one entity, that entity may be treated as the sole beneficiary, including where it is specifically established for the purpose of implementing the action financed by the grant.”

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- AGENTURA PRO ZDRAVOTNICKY VYZKUM CESKE REPUBLIKY AZV CR (AZV), PIC 878890229, linked to MINISTERSTVO ZDRAVOTNICTVI CESKE REPUBLIKY (MZd)

- KLINIKUM DER UNIVERSITAT MUNCHEN (KUM), PIC 995625946, linked to LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN (LMU)

Affiliated entities can charge costs and contributions to the action under the same conditions as the beneficiaries and must implement the action tasks attributed to them in Annex 1 in accordance with Article 11.

Their costs and contributions will be included in Annex 2 and will be taken into account for the calculation of the grant.

The beneficiaries must ensure that all their obligations under this Agreement also apply to their affiliated entities.

The beneficiaries must ensure that the bodies mentioned in Article 25 (e.g. granting authority, OLAF, Court of Auditors (ECA), etc.) can exercise their rights also towards the affiliated entities.

Breaches by affiliated entities will be handled in the same manner as breaches by beneficiaries. Recovery of undue amounts will be handled through the beneficiaries.

If the granting authority requires joint and several liability of affiliated entities (see Data Sheet, Point 4.4), they must sign the declaration set out in Annex 3a and may be held liable in case of enforced recoveries against their beneficiaries (see Article 22.2 and 22.4).

ARTICLE 9 — OTHER PARTICIPANTS INVOLVED IN THE ACTION

9.1 Associated partners

The following entities which cooperate with a beneficiary will participate in the action as ‘associated partners’:

- ASTRAZENECA AB (AZ), PIC 999941379

- CHILDREN'S HOSPITAL OF EASTERN ONTARIO RESEARCH INSTITUTE INC (CHEO-RI), PIC 907681187

- CANADIAN INSTITUTES OF HEALTH RESEARCH (CIHR), PIC 960368386

- DEUTSCHE FORSCHUNGSGEMEINSCHAFT EV (DFG), PIC 999547462

- FUJIFILM DIOSYNTH BIOTECHNOLOGIES UK LIMITED (FDB), PIC 962770494

- FONDS DE RECHERCHE DU QUEBEC - SANTE (FRQS), PIC 963122410

- HIPRA HUMAN HEALTH SL (HIPRA), PIC 890821326

- LABORATORIOS HIPRA SA (LAB HIPRA), PIC 991274041

- MICYRN (MICYRN), PIC 880745451

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- MILTENYI BIOTEC BV & CO KG (Miltenyi), PIC 897602402

- PFIZER INC (Pfizer), PIC 887211374

- DEPARTMENT OF HEALTH GOVERNMENT OF WESTERN AUSTRALIA (RCC), PIC 960480421

- REITHERA SRL (REI), PIC 998129419

- LABORATORIO REIG JOFRE SA (RJF), PIC 951005073

- F. HOFFMANN-LA ROCHE AG (ROCHE), PIC 999601782

- SCHWEIZERISCHER NATIONALFONDS ZUR FORDERUNG DER WISSENSCHAFTLICHEN FORSCHUNG (SNSF), PIC 999540575

- UCB BIOPHARMA (UCB), PIC 937666409

- FRAUNHOFER UK RESEARCH LIMITED (Fraunhofer UK), PIC 952487039

- GENETIC ALLIANCE UK LTD (GA UK), PIC 999790932

- UNIVERSITY OF NEWCASTLE UPON TYNE (UNEW), PIC 999985417

- Cambridge University Hospitals NHS Foundation Trust (CUH), PIC 991793961

- UNIVERSITY OF LEICESTER (ULEIC), PIC 999985514

- THE QUEEN'S UNIVERSITY OF BELFAST (QUB-UK), PIC 999992013

- UNIVERSITY COLLEGE LONDON (UCL), PIC 999975620

- THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE (UCAM), PIC 999977172

Associated partners must implement the action tasks attributed to them in Annex 1 in accordance with Article 11. They may not charge costs or contributions to the action and the costs for their tasks are not eligible.

The tasks must be set out in Annex 1.

The beneficiaries must ensure that their contractual obligations under Articles 11 (proper implementation), 12 (conflict of interests), 13 (confidentiality and security), 14 (ethics), 17.2 (visibility), 18 (specific rules for carrying out action), 19 (information) and 20 (record-keeping) also apply to the associated partners.

The beneficiaries must ensure that the bodies mentioned in Article 25 (e.g. granting authority, OLAF, Court of Auditors (ECA), etc.) can exercise their rights also towards the associated partners.

9.2 Third parties giving in-kind contributions to the action

Other third parties may give in-kind contributions to the action (i.e. personnel, equipment, other goods, works and services, etc. which are free-of-charge) if necessary for the implementation.

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Third parties giving in-kind contributions do not implement any action tasks. They may not charge costs or contributions to the action, but the costs for the in-kind contributions are eligible and may be charged by the beneficiaries which use them, under the conditions set out in Article 6. The costs will be included in Annex 2 as part of the beneficiaries’ costs.

The third parties and their in-kind contributions should be set out in Annex 1.

The beneficiaries must ensure that the bodies mentioned in Article 25 (e.g. granting authority, OLAF, Court of Auditors (ECA), etc.) can exercise their rights also towards the third parties giving in-kind contributions.

9.3 Subcontractors

Subcontractors may participate in the action, if necessary for the implementation.

Subcontractors must implement their action tasks in accordance with Article 11. The costs for the subcontracted tasks (invoiced price from the subcontractor) are eligible and may be charged by the beneficiaries, under the conditions set out in Article 6. The costs will be included in Annex 2 as part of the beneficiaries’ costs.

The beneficiaries must ensure that their contractual obligations under Articles 11 (proper implementation), 12 (conflict of interest), 13 (confidentiality and security), 14 (ethics), 17.2 (visibility), 18 (specific rules for carrying out action), 19 (information) and 20 (record-keeping) also apply to the subcontractors.

The beneficiaries must ensure that the bodies mentioned in Article 25 (e.g. granting authority, OLAF, Court of Auditors (ECA), etc.) can exercise their rights also towards the subcontractors.

9.4 Recipients of financial support to third parties

If the action includes providing financial support to third parties (e.g. grants, prizes or similar forms of support), the beneficiaries must ensure that their contractual obligations under Articles 12 (conflict of interest), 13 (confidentiality and security), 14 (ethics), 17.2 (visibility), 18 (specific rules for carrying out action), 19 (information) and 20 (record-keeping)also apply to the third parties receiving the support (recipients).

The beneficiaries must also ensure that the bodies mentioned in Article 25 (e.g. granting authority, OLAF, Court of Auditors (ECA), etc.) can exercise their rights also towards the recipients.

ARTICLE 10 — PARTICIPANTS WITH SPECIAL STATUS

10.1 Non-EU participants

Participants which are established in a non-EU country (if any) undertake to comply with their obligations under the Agreement and:

- to respect general principles (including fundamental rights, values and ethical principles, environmental and labour standards, rules on classified information, intellectual property rights, visibility of funding and protection of personal data)

- for the submission of certificates under Article 24: to use qualified external auditors which

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are independent and comply with comparable standards as those set out in EU Directive 2006/43/EC13

- for the controls under Article 25: to allow for checks, reviews, audits and investigations (including on-the-spot checks, visits and inspections) by the bodies mentioned in that Article (e.g. granting authority, OLAF, Court of Auditors (ECA), etc.).

Special rules on dispute settlement apply (see Data Sheet, Point 5).

10.2 Participants which are international organisations

Participants which are international organisations (IOs; if any) undertake to comply with their obligations under the Agreement and:

- to respect general principles (including fundamental rights, values and ethical principles, environmental and labour standards, rules on classified information, intellectual property rights, visibility of funding and protection of personal data)

- for the submission of certificates under Article 24: to use either independent public officers or external auditors which comply with comparable standards as those set out in EU Directive 2006/43/EC

- for the controls under Article 25: to allow for the checks, reviews, audits and investigations by the bodies mentioned in that Article, taking into account the specific agreements concluded by them and the EU (if any).

For such participants, nothing in the Agreement will be interpreted as a waiver of their privileges or immunities, as accorded by their constituent documents or international law.

Special rules on applicable law and dispute settlement apply (see Article 43 and Data Sheet, Point 5).

10.3 Pillar-assessed participants

Pillar-assessed participants (if any) may rely on their own systems, rules and procedures, in so far as they have been positively assessed and do not call into question the decision awarding the grant or breach the principle of equal treatment of applicants or beneficiaries.

‘Pillar-assessment’ means a review by the European Commission on the systems, rules and procedures which participants use for managing EU grants (in particular internal control system, accounting system, external audits, financing of third parties, rules on recovery and exclusion, information on recipients and protection of personal data; see Article 154 EU Financial Regulation 2018/1046).

Participants with a positive pillar assessment may rely on their own systems, rules and procedures, in particular for:

- record-keeping (Article 20): may be done in accordance with internal standards, rules and procedures

13 Directive 2006/43/EC of the European Parliament and of the Council of 17 May 2006 on statutory audits of annual accounts and consolidated accounts or similar national regulations (OJ L 157, 9.6.2006, p. 87).

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- currency conversion for financial statements (Article 21): may be done in accordance with usual accounting practices

- guarantees (Article 23): for public law bodies, prefinancing guarantees are not needed

- certificates (Article 24):

- certificates on the financial statements (CFS): may be provided by their regular internal or external auditors and in accordance with their internal financial regulations and procedures

- certificates on usual accounting practices (CoMUC): are not needed if those practices are covered by an ex-ante assessment

and use the following specific rules, for:

- recoveries (Article 22): in case of financial support to third parties, there will be no recovery if the participant has done everything possible to retrieve the undue amounts from the third party receiving the support (including legal proceedings) and non-recovery is not due to an error or negligence on its part

- checks, reviews, audits and investigations by the EU (Article 25): will be conducted taking into account the rules and procedures specifically agreed between them and the framework agreement (if any)

- impact evaluation (Article 26): will be conducted in accordance with the participant’s internal rules and procedures and the framework agreement (if any)

- grant agreement suspension (Article 31): certain costs incurred during grant suspension are eligible (notably, minimum costs necessary for a possible resumption of the action and costs relating to contracts which were entered into before the pre-information letter was received and which could not reasonably be suspended, reallocated or terminated on legal grounds)

- grant agreement termination (Article 32): the final grant amount and final payment will be calculated taking into account also costs relating to contracts due for execution only after termination takes effect, if the contract was entered into before the pre-information letter was received and could not reasonably be terminated on legal grounds

- liability for damages (Article 33.2): the granting authority must be compensated for damage it sustains as a result of the implementation of the action or because the action was not implemented in full compliance with the Agreement only if the damage is due to an infringement of the participant’s internal rules and procedures or due to a violation of third parties’ rights by the participant or one of its employees or individual for whom the employees are responsible.

Participants whose pillar assessment covers procurement and granting procedures may also do purchases, subcontracting and financial support to third parties (Article 6.2) in accordance with their internal rules and procedures for purchases, subcontracting and financial support.

Participants whose pillar assessment covers data protection rules may rely on their internal standards, rules and procedures for data protection (Article 15).

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The participants may however not rely on provisions which would breach the principle of equal treatment of applicants or beneficiaries or call into question the decision awarding the grant, such as in particular:

- eligibility (Article 6)

- consortium roles and set-up (Articles 7-9)

- security and ethics (Articles 13, 14)

- IPR (including background and results, access rights and rights of use), communication, dissemination and visibility (Articles 16 and 17)

- information obligation (Article 19)

- payment, reporting and amendments (Articles 21, 22 and 39)

- rejections, reductions, suspensions and terminations (Articles 27, 28, 29-32)

If the pillar assessment was subject to remedial measures, reliance on the internal systems, rules and procedures is subject to compliance with those remedial measures.

Participants whose assessment has not yet been updated to cover (the new rules on) data protection may rely on their internal systems, rules and procedures, provided that they ensure that personal data is:

- processed lawfully, fairly and in a transparent manner in relation to the data subject

- collected for specified, explicit and legitimate purposes and not further processed in a manner that is incompatible with those purposes

- adequate, relevant and limited to what is necessary in relation to the purposes for which they are processed

- accurate and, where necessary, kept up to date

- kept in a form which permits identification of data subjects for no longer than is necessary for the purposes for which the data is processed and

- processed in a manner that ensures appropriate security of the personal data.

Participants must inform the coordinator without delay of any changes to the systems, rules and procedures that were part of the pillar assessment. The coordinator must immediately inform the granting authority.

Pillar-assessed participants that have also concluded a framework agreement with the EU, may moreover — under the same conditions as those above (i.e. not call into question the decision awarding the grant or breach the principle of equal treatment of applicants or beneficiaries) — rely on the provisions set out in that framework agreement.

SECTION 2 RULES FOR CARRYING OUT THE ACTION

ARTICLE 11 — PROPER IMPLEMENTATION OF THE ACTION

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11.1 Obligation to properly implement the action

The beneficiaries must implement the action as described in Annex 1 and in compliance with the provisions of the Agreement, the call conditions and all legal obligations under applicable EU, international and national law.

11.2 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 12 — CONFLICT OF INTERESTS

12.1 Conflict of interests

The beneficiaries must take all measures to prevent any situation where the impartial and objective implementation of the Agreement could be compromised for reasons involving family, emotional life, political or national affinity, economic interest or any other direct or indirect interest (‘conflict of interests’).

They must formally notify the granting authority without delay of any situation constituting or likely to lead to a conflict of interests and immediately take all the necessary steps to rectify this situation.

The granting authority may verify that the measures taken are appropriate and may require additional measures to be taken by a specified deadline.

12.2 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28) and the grant or the beneficiary may be terminated (see Article 32).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 13 — CONFIDENTIALITY AND SECURITY

13.1 Sensitive information

The parties must keep confidential any data, documents or other material (in any form) that is identified as sensitive in writing (‘sensitive information’) — during the implementation of the action and for at least until the time-limit set out in the Data Sheet (see Point 6).

If a beneficiary requests, the granting authority may agree to keep such information confidential for a longer period.

Unless otherwise agreed between the parties, they may use sensitive information only to implement the Agreement.

The beneficiaries may disclose sensitive information to their personnel or other participants involved in the action only if they:

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(a) need to know it in order to implement the Agreement and

(b) are bound by an obligation of confidentiality.

The granting authority may disclose sensitive information to its staff and to other EU institutions and bodies.

It may moreover disclose sensitive information to third parties, if:

(a) this is necessary to implement the Agreement or safeguard the EU financial interests and

(b) the recipients of the information are bound by an obligation of confidentiality.

The confidentiality obligations no longer apply if:

(a) the disclosing party agrees to release the other party

(b) the information becomes publicly available, without breaching any confidentiality obligation

(c) the disclosure of the sensitive information is required by EU, international or national law.

Specific confidentiality rules (if any) are set out in Annex 5.

13.2 Classified information

The parties must handle classified information in accordance with the applicable EU, international or national law on classified information (in particular, Decision 2015/44414 and its implementing rules).

Deliverables which contain classified information must be submitted according to special procedures agreed with the granting authority.

Action tasks involving classified information may be subcontracted only after explicit approval (in writing) from the granting authority.

Classified information may not be disclosed to any third party (including participants involved in the action implementation) without prior explicit written approval from the granting authority.

Specific security rules (if any) are set out in Annex 5.

13.3 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 14 — ETHICS AND VALUES

14.1 Ethics

14 Commission Decision 2015/444/EC, Euratom of 13 March 2015 on the security rules for protecting EU classified information (OJ L 72, 17.3.2015, p. 53).

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The action must be carried out in line with the highest ethical standards and the applicable EU, international and national law on ethical principles.

Specific ethics rules (if any) are set out in Annex 5.

14.2 Values

The beneficiaries must commit to and ensure the respect of basic EU values (such as respect for human dignity, freedom, democracy, equality, the rule of law and human rights, including the rights of minorities).

Specific rules on values (if any) are set out in Annex 5.

14.3 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 15 — DATA PROTECTION

15.1 Data processing by the granting authority

Any personal data under the Agreement will be processed under the responsibility of the data controller of the granting authority in accordance with and for the purposes set out in the Portal Privacy Statement.

For grants where the granting authority is the European Commission, an EU regulatory or executive agency, joint undertaking or other EU body, the processing will be subject to Regulation 2018/172515.

15.2 Data processing by the beneficiaries

The beneficiaries must process personal data under the Agreement in compliance with the applicable EU, international and national law on data protection (in particular, Regulation 2016/67916).

They must ensure that personal data is:

- processed lawfully, fairly and in a transparent manner in relation to the data subjects

- collected for specified, explicit and legitimate purposes and not further processed in a manner that is incompatible with those purposes

15 Regulation (EU) 2018/1725 of the European Parliament and of the Council of 23 October 2018 on the protection of natural persons with regard to the processing of personal data by the Union institutions, bodies, offices and agencies and on the free movement of such data, and repealing Regulation (EC) No 45/2001 and Decision No 1247/2002/EC (OJ L 295, 21.11.2018, p. 39).

16 Regulation (EU) 2016/679 of the European Parliament and of the Council of 27 April 2016 on the protection of natural persons with regard to the processing of personal data and on the free movement of such data, and repealing Directive 95/46/EC (‘GDPR’) (OJ L 119, 4.5.2016, p. 1).

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- adequate, relevant and limited to what is necessary in relation to the purposes for which they are processed

- accurate and, where necessary, kept up to date

- kept in a form which permits identification of data subjects for no longer than is necessary for the purposes for which the data is processed and

- processed in a manner that ensures appropriate security of the data.

The beneficiaries may grant their personnel access to personal data only if it is strictly necessary for implementing, managing and monitoring the Agreement. The beneficiaries must ensure that the personnel is under a confidentiality obligation.

The beneficiaries must inform the persons whose data are transferred to the granting authority and provide them with the Portal Privacy Statement.

15.3 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 16 — INTELLECTUAL PROPERTY RIGHTS (IPR) — BACKGROUND AND RESULTS —ACCESS RIGHTS AND RIGHTS OF USE

16.1 Background and access rights to background

The beneficiaries must give each other and the other participants access to the background identified as needed for implementing the action, subject to any specific rules in Annex 5.

‘Background’ means any data, know-how or information — whatever its form or nature (tangible or intangible), including any rights such as intellectual property rights — that is:

(a) held by the beneficiaries before they acceded to the Agreement and

(b) needed to implement the action or exploit the results.

If background is subject to rights of a third party, the beneficiary concerned must ensure that it is able to comply with its obligations under the Agreement.

16.2 Ownership of results

The granting authority does not obtain ownership of the results produced under the action.

‘Results’ means any tangible or intangible effect of the action, such as data, know-how or information, whatever its form or nature, whether or not it can be protected, as well as any rights attached to it, including intellectual property rights.

16.3 Rights of use of the granting authority on materials, documents and information received for policy, information, communication, dissemination and publicity purposes

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The granting authority has the right to use non-sensitive information relating to the action and materials and documents received from the beneficiaries (notably summaries for publication, deliverables, as well as any other material, such as pictures or audio-visual material, in paper or electronic form) for policy, information, communication, dissemination and publicity purposes — during the action or afterwards.

The right to use the beneficiaries’ materials, documents and information is granted in the form of a royalty-free, non-exclusive and irrevocable licence, which includes the following rights:

(a) use for its own purposes (in particular, making them available to persons working for the granting authority or any other EU service (including institutions, bodies, offices, agencies, etc.) or EU Member State institution or body; copying or reproducing them in whole or in part, in unlimited numbers; and communication through press information services)

(b) distribution to the public (in particular, publication as hard copies and in electronic or digital format, publication on the internet, as a downloadable or non-downloadable file, broadcasting by any channel, public display or presentation, communicating through press information services, or inclusion in widely accessible databases or indexes)

(c) editing or redrafting (including shortening, summarising, inserting other elements (e.g. meta-data, legends, other graphic, visual, audio or text elements), extracting parts (e.g. audio or video files), dividing into parts, use in a compilation)

(d) translation

(e) storage in paper, electronic or other form

(f) archiving, in line with applicable document-management rules

(g) the right to authorise third parties to act on its behalf or sub-license to third parties the modes of use set out in Points (b), (c), (d) and (f), if needed for the information, communication and publicity activity of the granting authority

(h) processing, analysing, aggregating the materials, documents and information received and producing derivative works.

The rights of use are granted for the whole duration of the industrial or intellectual property rights concerned.

If materials or documents are subject to moral rights or third party rights (including intellectual property rights or rights of natural persons on their image and voice), the beneficiaries must ensure that they comply with their obligations under this Agreement (in particular, by obtaining the necessary licences and authorisations from the rights holders concerned).

Where applicable, the granting authority will insert the following information:

“© – [year] – [name of the copyright owner]. All rights reserved. Licensed to the [name of granting authority] under conditions.”

16.4 Specific rules on IPR, results and background

Specific rules regarding intellectual property rights, results and background (if any) are set out in Annex 5.

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16.5 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such a breach may also lead to other measures described in Chapter 5.

ARTICLE 17 — COMMUNICATION, DISSEMINATION AND VISIBILITY

17.1 Communication — Dissemination — Promoting the action

Unless otherwise agreed with the granting authority, the beneficiaries must promote the action and its results by providing targeted information to multiple audiences (including the media and the public), in accordance with Annex 1 and in a strategic, coherent and effective manner.

Before engaging in a communication or dissemination activity expected to have a major media impact, the beneficiaries must inform the granting authority.

17.2 Visibility — European flag and funding statement

Unless otherwise agreed with the granting authority, communication activities of the beneficiaries related to the action (including media relations, conferences, seminars, information material, such as brochures, leaflets, posters, presentations, etc., in electronic form, via traditional or social media, etc.), dissemination activities and any infrastructure, equipment, vehicles, supplies or major result funded by the grant must acknowledge EU support and display the European flag (emblem) and funding statement (translated into local languages, where appropriate):

The emblem must remain distinct and separate and cannot be modified by adding other visual marks, brands or text.

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Apart from the emblem, no other visual identity or logo may be used to highlight the EU support.

When displayed in association with other logos (e.g. of beneficiaries or sponsors), the emblem must be displayed at least as prominently and visibly as the other logos.

For the purposes of their obligations under this Article, the beneficiaries may use the emblem without first obtaining approval from the granting authority. This does not, however, give them the right to exclusive use. Moreover, they may not appropriate the emblem or any similar trademark or logo, either by registration or by any other means.

17.3 Quality of information — Disclaimer

Any communication or dissemination activity related to the action must use factually accurate information.

Moreover, it must indicate the following disclaimer (translated into local languages where appropriate):

“Funded by the European Union. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or [name of the granting authority]. Neither the European Union nor the granting authority can be held responsible for them.”

17.4 Specific communication, dissemination and visibility rules

Specific communication, dissemination and visibility rules (if any) are set out in Annex 5.

17.5 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 18 — SPECIFIC RULES FOR CARRYING OUT THE ACTION

18.1 Specific rules for carrying out the action

Specific rules for implementing the action (if any) are set out in Annex 5.

18.2 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such a breach may also lead to other measures described in Chapter 5.

SECTION 3 GRANT ADMINISTRATION

ARTICLE 19 — GENERAL INFORMATION OBLIGATIONS

19.1 Information requests

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The beneficiaries must provide — during the action or afterwards and in accordance with Article 7 — any information requested in order to verify eligibility of the costs or contributions declared, proper implementation of the action and compliance with the other obligations under the Agreement.

The information provided must be accurate, precise and complete and in the format requested, including electronic format.

19.2 Participant Register data updates

The beneficiaries must keep — at all times, during the action or afterwards — their information stored in the Portal Participant Register up to date, in particular, their name, address, legal representatives, legal form and organisation type.

19.3 Information about events and circumstances which impact the action

The beneficiaries must immediately inform the granting authority (and the other beneficiaries) of any of the following:

(a) events which are likely to affect or delay the implementation of the action or affect the EU’s financial interests, in particular:

(i) changes in their legal, financial, technical, organisational or ownership situation (including changes linked to one of the exclusion grounds listed in the declaration of honour signed before grant signature)

(ii) linked action information: not applicable

(b) circumstances affecting:

(i) the decision to award the grant or

(ii) compliance with requirements under the Agreement.

19.4 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 20 — RECORD-KEEPING

20.1 Keeping records and supporting documents

The beneficiaries must — at least until the time-limit set out in the Data Sheet (see Point 6) — keep records and other supporting documents to prove the proper implementation of the action in line with the accepted standards in the respective field (if any).

In addition, the beneficiaries must — for the same period — keep the following to justify the amounts declared:

(a) for actual costs: adequate records and supporting documents to prove the costs declared (such

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as contracts, subcontracts, invoices and accounting records); in addition, the beneficiaries’ usual accounting and internal control procedures must enable direct reconciliation between the amounts declared, the amounts recorded in their accounts and the amounts stated in the supporting documents

(b) for flat-rate costs and contributions (if any): adequate records and supporting documents to prove the eligibility of the costs or contributions to which the flat-rate is applied

(c) for the following simplified costs and contributions: the beneficiaries do not need to keep specific records on the actual costs incurred, but must keep:

(i) for unit costs and contributions (if any): adequate records and supporting documents to prove the number of units declared

(ii) for lump sum costs and contributions (if any): adequate records and supporting documents to prove proper implementation of the work as described in Annex 1

(iii) for financing not linked to costs (if any): adequate records and supporting documents to prove the achievement of the results or the fulfilment of the conditions as described in Annex 1

(d) for unit, flat-rate and lump sum costs and contributions according to usual cost accounting practices (if any): the beneficiaries must keep any adequate records and supporting documents to prove that their cost accounting practices have been applied in a consistent manner, based on objective criteria, regardless of the source of funding, and that they comply with the eligibility conditions set out in Articles 6.1 and 6.2.

Moreover, the following is needed for specific budget categories:

(e) for personnel costs: time worked for the beneficiary under the action must be supported by declarations signed monthly by the person and their supervisor, unless another reliable time-record system is in place; the granting authority may accept alternative evidence supporting the time worked for the action declared, if it considers that it offers an adequate level of assurance

(f) additional record-keeping rules: not applicable

The records and supporting documents must be made available upon request (see Article 19) or in the context of checks, reviews, audits or investigations (see Article 25).

If there are on-going checks, reviews, audits, investigations, litigation or other pursuits of claims under the Agreement (including the extension of findings; see Article 25), the beneficiaries must keep these records and other supporting documentation until the end of these procedures.

The beneficiaries must keep the original documents. Digital and digitalised documents are considered originals if they are authorised by the applicable national law. The granting authority may accept non-original documents if they offer a comparable level of assurance.

20.2 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, costs or contributions insufficiently

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substantiated will be ineligible (see Article 6) and will be rejected (see Article 27), and the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 21 — REPORTING

21.1 Continuous reporting

The beneficiaries must continuously report on the progress of the action (e.g. deliverables, milestones, outputs/outcomes, critical risks, indicators, etc; if any), in the Portal Continuous Reporting tool and in accordance with the timing and conditions it sets out (as agreed with the granting authority).

Standardised deliverables (e.g. progress reports not linked to payments, reports on cumulative expenditure, special reports, etc; if any) must be submitted using the templates published on the Portal.

21.2 Periodic reporting: Technical reports and financial statements

In addition, the beneficiaries must provide reports to request payments, in accordance with the schedule and modalities set out in the Data Sheet (see Point 4.2):

- for additional prefinancings (if any): an additional prefinancing report

- for interim payments (if any) and the final payment: a periodic report.

The prefinancing and periodic reports include a technical and financial part.

The technical part includes an overview of the action implementation. It must be prepared using the template available in the Portal Periodic Reporting tool.

The financial part of the additional prefinancing report includes a statement on the use of the previous prefinancing payment.

The financial part of the periodic report includes:

- the financial statements (individual and consolidated; for all beneficiaries/affiliated entities)

- the explanation on the use of resources (or detailed cost reporting table, if required)

- the certificates on the financial statements (CFS) (if required; see Article 24.2 and Data Sheet, Point 4.3).

The financial statements must detail the eligible costs and contributions for each budget category and, for the final payment, also the revenues for the action (see Articles 6 and 22).

All eligible costs and contributions incurred should be declared, even if they exceed the amounts indicated in the estimated budget (see Annex 2). Amounts that are not declared in the individual financial statements will not be taken into account by the granting authority.

By signing the financial statements (directly in the Portal Periodic Reporting tool), the beneficiaries confirm that:

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- the information provided is complete, reliable and true

- the costs and contributions declared are eligible (see Article 6)

- the costs and contributions can be substantiated by adequate records and supporting documents (see Article 20) that will be produced upon request (see Article 19) or in the context of checks, reviews, audits and investigations (see Article 25)

- for the final periodic report: all the revenues have been declared (if required; see Article 22).

Beneficiaries will have to submit also the financial statements of their affiliated entities (if any). In case of recoveries (see Article 22), beneficiaries will be held responsible also for the financial statements of their affiliated entities.

21.3 Currency for financial statements and conversion into euros

The financial statements must be drafted in euro.

Beneficiaries with general accounts established in a currency other than the euro must convert the costs recorded in their accounts into euro, at the average of the daily exchange rates published in the C series of the Official Journal of the European Union (ECB website), calculated over the corresponding reporting period.

If no daily euro exchange rate is published in the Official Journal for the currency in question, they must be converted at the average of the monthly accounting exchange rates published on the European Commission website (InforEuro), calculated over the corresponding reporting period.

Beneficiaries with general accounts in euro must convert costs incurred in another currency into euro according to their usual accounting practices.

21.4 Reporting language

The reporting must be in the language of the Agreement, unless otherwise agreed with the granting authority (see Data Sheet, Point 4.2).

21.5 Consequences of non-compliance

If a report submitted does not comply with this Article, the granting authority may suspend the payment deadline (see Article 29) and apply other measures described in Chapter 5.

If the coordinator breaches its reporting obligations, the granting authority may terminate the grant or the coordinator’s participation (see Article 32) or apply other measures described in Chapter 5.

ARTICLE 22 — PAYMENTS AND RECOVERIES — CALCULATION OF AMOUNTS DUE

22.1 Payments and payment arrangements

Payments will be made in accordance with the schedule and modalities set out in the Data Sheet (see Point 4.2).

They will be made in euro to the bank account indicated by the coordinator (see Data Sheet, Point 4.2)

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and must be distributed without unjustified delay (restrictions may apply to distribution of the initial prefinancing payment; see Data Sheet, Point 4.2).

Payments to this bank account will discharge the granting authority from its payment obligation.

The cost of payment transfers will be borne as follows:

- the granting authority bears the cost of transfers charged by its bank

- the beneficiary bears the cost of transfers charged by its bank

- the party causing a repetition of a transfer bears all costs of the repeated transfer.

Payments by the granting authority will be considered to have been carried out on the date when they are debited to its account.

22.2 Recoveries

Recoveries will be made, if — at beneficiary termination, final payment or afterwards — it turns out that the granting authority has paid too much and needs to recover the amounts undue.

Each beneficiary’s financial responsibility in case of recovery is in principle limited to their own debt and undue amounts of their affiliated entities.

In case of enforced recoveries (see Article 22.4), affiliated entities will be held liable for repaying debts of their beneficiaries, if required by the granting authority (see Data Sheet, Point 4.4).

22.3 Amounts due

22.3.1 Prefinancing payments

The aim of the prefinancing is to provide the beneficiaries with a float.

It remains the property of the EU until the final payment.

For initial prefinancings (if any), the amount due, schedule and modalities are set out in the Data Sheet (see Point 4.2).

For additional prefinancings (if any), the amount due, schedule and modalities are also set out in the Data Sheet (see Point 4.2). However, if the statement on the use of the previous prefinancing payment shows that less than 70% was used, the amount set out in the Data Sheet will be reduced by the difference between the 70% threshold and the amount used.

The contribution to the Mutual Insurance Mechanism will be retained from the prefinancing payments (at the rate and in accordance with the modalities set out in the Data Sheet, see Point 4.2) and transferred to the Mechanism.

Prefinancing payments (or parts of them) may be offset (without the beneficiaries’ consent) against amounts owed by a beneficiary to the granting authority — up to the amount due to that beneficiary.

For grants where the granting authority is the European Commission or an EU executive agency, offsetting may also be done against amounts owed to other Commission services or executive agencies.

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Payments will not be made if the payment deadline or payments are suspended (see Articles 29 and 30).

22.3.2 Amount due at beneficiary termination — Recovery

In case of beneficiary termination, the granting authority will determine the provisional amount due for the beneficiary concerned. Payments (if any) will be made with the next interim or final payment.

The amount due will be calculated in the following step:

Step 1 — Calculation of the total accepted EU contribution

Step 1 — Calculation of the total accepted EU contribution

The granting authority will first calculate the ‘accepted EU contribution’ for the beneficiary for all reporting periods, by calculating the ‘maximum EU contribution to costs’ (applying the funding rate to the accepted costs of the beneficiary), taking into account requests for a lower contribution to costs and CFS threshold cappings (if any; see Article 24.5) and adding the contributions (accepted unit, flat-rate or lump sum contributions and financing not linked to costs, if any).

After that, the granting authority will take into account grant reductions (if any). The resulting amount is the ‘total accepted EU contribution’ for the beneficiary.

The balance is then calculated by deducting the payments received (if any; see report on the distribution of payments in Article 32), from the total accepted EU contribution:

{total accepted EU contribution for the beneficiary

minus

{prefinancing and interim payments received (if any)}}.

If the balance is positive, the amount will be included in the next interim or final payment to the consortium.

If the balance is negative, it will be recovered in accordance with the following procedure:

The granting authority will send a pre-information letter to the beneficiary concerned:

- formally notifying the intention to recover, the amount due, the amount to be recovered and the reasons why and

- requesting observations within 30 days of receiving notification.

If no observations are submitted (or the granting authority decides to pursue recovery despite the observations it has received), it will confirm the amount to be recovered and ask this amount to be paid to the coordinator (confirmation letter).

If payment is not made to the coordinator by the date specified in the confirmation letter, the granting authority may call on the Mutual Insurance Mechanism to intervene, if continuation of the action is guaranteed and the conditions set out in the rules governing the Mechanism are met.

In this case, it will send a beneficiary recovery letter, together with a debit note with the terms and date for payment.

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The debit note for the beneficiary will include the amount calculated for the affiliated entities which also had to end their participation (if any).

If payment is not made by the date specified in the debit note, the granting authority will enforce recovery in accordance with Article 22.4.

The amounts will later on also be taken into account for the next interim or final payment.

22.3.3 Interim payments

Interim payments reimburse the eligible costs and contributions claimed for the implementation of the action during the reporting periods (if any).

Interim payments (if any) will be made in accordance with the schedule and modalities set out the Data Sheet (see Point 4.2).

Payment is subject to the approval of the periodic report. Its approval does not imply recognition of compliance, authenticity, completeness or correctness of its content.

The interim payment will be calculated by the granting authority in the following steps:

Step 1 — Calculation of the total accepted EU contribution

Step 2 — Limit to the interim payment ceiling

Step 1 — Calculation of the total accepted EU contribution

The granting authority will calculate the ‘accepted EU contribution’ for the action for the reporting period, by first calculating the ‘maximum EU contribution to costs’ (applying the funding rate to the accepted costs of each beneficiary), taking into account requests for a lower contribution to costs, and CFS threshold cappings (if any; see Article 24.5) and adding the contributions (accepted unit, flat-rate or lump sum contributions and financing not linked to costs, if any).

After that, the granting authority will take into account grant reductions from beneficiary termination (if any). The resulting amount is the ‘total accepted EU contribution’.

Step 2 — Limit to the interim payment ceiling

The resulting amount is then capped to ensure that the total amount of prefinancing and interim payments (if any) does not exceed the interim payment ceiling set out in the Data Sheet (see Point 4.2).

Interim payments (or parts of them) may be offset (without the beneficiaries’ consent) against amounts owed by a beneficiary to the granting authority — up to the amount due to that beneficiary.

For grants where the granting authority is the European Commission or an EU executive agency, offsetting may also be done against amounts owed to other Commission services or executive agencies.

Payments will not be made if the payment deadline or payments are suspended (see Articles 29 and 30).

22.3.4 Final payment — Final grant amount — Revenues and Profit — Recovery

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The final payment (payment of the balance) reimburses the remaining part of the eligible costs and contributions claimed for the implementation of the action (if any).

The final payment will be made in accordance with the schedule and modalities set out in the Data Sheet (see Point 4.2).

Payment is subject to the approval of the final periodic report. Its approval does not imply recognition of compliance, authenticity, completeness or correctness of its content.

The final grant amount for the action will be calculated in the following steps:

Step 1 — Calculation of the total accepted EU contribution

Step 2 — Limit to the maximum grant amount

Step 3 — Reduction due to the no-profit rule

Step 1 — Calculation of the total accepted EU contribution

The granting authority will first calculate the ‘accepted EU contribution’ for the action for all reporting periods, by calculating the ‘maximum EU contribution to costs’ (applying the funding rate to the total accepted costs of each beneficiary), taking into account requests for a lower contribution to costs, CFS threshold cappings (if any; see Article 24.5) and adding the contributions (accepted unit, flat-rate or lump sum contributions and financing not linked to costs, if any).

After that, the granting authority will take into account grant reductions (if any). The resulting amount is the ‘total accepted EU contribution’.

Step 2 — Limit to the maximum grant amount

If the resulting amount is higher than the maximum grant amount set out in Article 5.2, it will be limited to the latter.

Step 3 — Reduction due to the no-profit rule

If the no-profit rule is provided for in the Data Sheet (see Point 4.2), the grant must not produce a profit (i.e. surplus of the amount obtained following Step 2 plus the action’s revenues, over the eligible costs and contributions approved by the granting authority).

‘Revenue’ is all income generated by the action, during its duration (see Article 4), for beneficiaries that are profit legal entities (— with the exception of income generated by the exploitation of results, which are not considered as revenues).

If there is a profit, it will be deducted in proportion to the final rate of reimbursement of the eligible costs approved by the granting authority (as compared to the amount calculated following Steps 1 and 2 minus the contributions).

The balance (final payment) is then calculated by deducting the total amount of prefinancing and interim payments already made (if any), from the final grant amount:

{final grant amount

minus

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{prefinancing and interim payments made (if any)}}.

If the balance is positive, it will be paid to the coordinator.

The amount retained for the Mutual Insurance Mechanism (see above) will be released and paid to the coordinator (in accordance with the rules governing the Mechanism).

The final payment (or part of it) may be offset (without the beneficiaries’ consent) against amounts owed by a beneficiary to the granting authority — up to the amount due to that beneficiary.

For grants where the granting authority is the European Commission or an EU executive agency, offsetting may also be done against amounts owed to other Commission services or executive agencies.

Payments will not be made if the payment deadline or payments are suspended (see Articles 29 and 30).

If — despite the release of the Mutual Insurance Mechanism contribution — the balance is negative, it will be recovered in accordance with the following procedure:

The granting authority will send a pre-information letter to the coordinator:

- formally notifying the intention to recover, the final grant amount, the amount to be recovered and the reasons why

- requesting a report on the distribution of payments to the beneficiaries within 30 days of receiving notification and

- requesting observations within 30 days of receiving notification.

If no observations are submitted (or the granting authority decides to pursue recovery despite the observations it has received) and the coordinator has submitted the report on the distribution of payments, it will calculate the share of the debt per beneficiary, by:

(a) identifying the beneficiaries for which the amount calculated as follows is negative:

{{{total accepted EU contribution for the beneficiary

divided by

total accepted EU contribution for the action}

multiplied by

final grant amount for the action},

minus

{prefinancing and interim payments received by the beneficiary (if any)}} and

(b) dividing the debt:

{{amount calculated according to point (a) for the beneficiary concerned

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divided by

the sum of the amounts calculated according to point (a) for all the beneficiaries identified according to point (a)}

multiplied by

the amount to be recovered}.

and confirm the amount to be recovered from each beneficiary concerned (confirmation letter), together with debit notes with the terms and date for payment.

The debit notes for beneficiaries will include the amounts calculated for their affiliated entities (if any).

If the coordinator has not submitted the report on the distribution of payments, the granting authority will recover the full amount from the coordinator (confirmation letter and debit note with the terms and date for payment).

If payment is not made by the date specified in the debit note, the granting authority will enforce recovery in accordance with Article 22.4.

22.3.5 Audit implementation after final payment — Revised final grant amount — Recovery

If — after the final payment (in particular, after checks, reviews, audits or investigations; see Article 25) — the granting authority rejects costs or contributions (see Article 27) or reduces the grant (see Article 28), it will calculate the revised final grant amount for the beneficiary concerned.

The beneficiary revised final grant amount will be calculated in the following step:

Step 1 — Calculation of the revised total accepted EU contribution

Step 1 — Calculation of the revised total accepted EU contribution

The granting authority will first calculate the ‘revised accepted EU contribution’ for the beneficiary, by calculating the ‘revised accepted costs’ and ‘revised accepted contributions’.

After that, it will take into account grant reductions (if any). The resulting ‘revised total accepted EU contribution’ is the beneficiary revised final grant amount.

If the revised final grant amount is lower than the beneficiary’s final grant amount (i.e. its share in the final grant amount for the action), it will be recovered in accordance with the following procedure:

The beneficiary final grant amount (i.e. share in the final grant amount for the action) is calculated as follows:

{{total accepted EU contribution for the beneficiary

divided by

total accepted EU contribution for the action}

multiplied by

final grant amount for the action}.

The granting authority will send a pre-information letter to the beneficiary concerned:

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- formally notifying the intention to recover, the amount to be recovered and the reasons why and

- requesting observations within 30 days of receiving notification.

If no observations are submitted (or the granting authority decides to pursue recovery despite the observations it has received), it will confirm the amount to be recovered (confirmation letter), together with a debit note with the terms and the date for payment.

Recoveries against affiliated entities (if any) will be handled through their beneficiaries.

If payment is not made by the date specified in the debit note, the granting authority will enforce recovery in accordance with Article 22.4.

22.4 Enforced recovery

If payment is not made by the date specified in the debit note, the amount due will be recovered:

(a) by offsetting the amount — without the coordinator or beneficiary’s consent — against any amounts owed to the coordinator or beneficiary by the granting authority.

In exceptional circumstances, to safeguard the EU financial interests, the amount may be offset before the payment date specified in the debit note.

For grants where the granting authority is the European Commission or an EU executive agency, debts may also be offset against amounts owed by other Commission services or executive agencies.

(b) financial guarantee(s): not applicable

(c) joint and several liability of beneficiaries: not applicable

(d) by holding affiliated entities jointly and severally liable (if any, see Data Sheet, Point 4.4)

(e) by taking legal action (see Article 43) or, provided that the granting authority is the European Commission or an EU executive agency, by adopting an enforceable decision under Article 299 of the Treaty on the Functioning of the EU (TFEU) and Article 100(2) of EU Financial Regulation 2018/1046.

If the Mutual Insurance Mechanism was called on by the granting authority to intervene, recovery will be continued in the name of the Mutual Insurance Mechanism. If two debit notes were sent, the second one (in the name of the Mutual Insurance Mechanism) will be considered to replace the first one (in the name of the granting authority). Where the MIM intervened, offsetting, enforceable decisions or any other of the above-mentioned forms of enforced recovery may be used mutatis mutandis.

The amount to be recovered will be increased by late-payment interest at the rate set out in Article 22.5, from the day following the payment date in the debit note, up to and including the date the full payment is received.

Partial payments will be first credited against expenses, charges and late-payment interest and then against the principal.

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Bank charges incurred in the recovery process will be borne by the beneficiary, unless Directive 2015/236617 applies.

For grants where the granting authority is an EU executive agency, enforced recovery by offsetting or enforceable decision will be done by the services of the European Commission (see also Article 43).

22.5 Consequences of non-compliance

22.5.1 If the granting authority does not pay within the payment deadlines (see above), the beneficiaries are entitled to late-payment interest at the rate applied by the European Central Bank (ECB) for its main refinancing operations in euros (‘reference rate’), plus the rate specified in the Data Sheet (Point 4.2). The reference rate is the rate in force on the first day of the month in which the payment deadline expires, as published in the C series of the Official Journal of the European Union.

If the late-payment interest is lower than or equal to EUR 200, it will be paid to the coordinator only on request submitted within two months of receiving the late payment.

Late-payment interest is not due if all beneficiaries are EU Member States (including regional and local government authorities or other public bodies acting on behalf of a Member State for the purpose of this Agreement).

If payments or the payment deadline are suspended (see Articles 29 and 30), payment will not be considered as late.

Late-payment interest covers the period running from the day following the due date for payment (see above), up to and including the date of payment.

Late-payment interest is not considered for the purposes of calculating the final grant amount.

22.5.2 If the coordinator breaches any of its obligations under this Article, the grant may be reduced (see Article 28) and the grant or the coordinator may be terminated (see Article 32).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 23 — GUARANTEES

Not applicable

ARTICLE 24 — CERTIFICATES

24.1 Operational verification report (OVR)

Not applicable

24.2 Certificate on the financial statements (CFS)

If required by the granting authority (see Data Sheet, Point 4.3), the beneficiaries must provide

17 Directive (EU) 2015/2366 of the European Parliament and of the Council of 25 November 2015 on payment services in the internal market, amending Directives 2002/65/EC, 2009/110/EC and 2013/36/EU and Regulation (EU) No 1093/2010, and repealing Directive 2007/64/EC (OJ L 337, 23.12.2015, p. 35).

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certificates on their financial statements (CFS), in accordance with the schedule, threshold and conditions set out in the Data Sheet.

The coordinator must submit them as part of the periodic report (see Article 21).

The certificates must be drawn up using the template published on the Portal, cover the costs declared on the basis of actual costs and costs according to usual cost accounting practices (if any), and fulfil the following conditions:

(a) be provided by a qualified approved external auditor which is independent and complies with Directive 2006/43/EC18 (or for public bodies: by a competent independent public officer)

(b) the verification must be carried out according to the highest professional standards to ensure that the financial statements comply with the provisions under the Agreement and that the costs declared are eligible.

The certificates will not affect the granting authority's right to carry out its own checks, reviews or audits, nor preclude the European Court of Auditors (ECA), the European Public Prosecutor’s Office (EPPO) or the European Anti-Fraud Office (OLAF) from using their prerogatives for audits and investigations under the Agreement (see Article 25).

If the costs (or a part of them) were already audited by the granting authority, these costs do not need to be covered by the certificate and will not be counted for calculating the threshold (if any).

24.3 Certificate on the compliance of usual cost accounting practices (CoMUC)

Not applicable

24.4 Systems and process audit (SPA)

Beneficiaries which:

- use unit, flat rate or lump sum costs or contributions according to documented (i.e. formally approved and in writing) usual costs accounting practices (if any) or

- have formalised documentation on the systems and processes for calculating their costs and contributions (i.e. formally approved and in writing), have participated in at least 150 actions under Horizon 2020 or the Euratom Research and Training Programme (2014-2018 or 2019-2020) and participate in at least 3 ongoing actions under Horizon Europe or the Euratom Research and Training Programme (2021-2025 or 2026-2027)

may apply to the granting authority for a systems and process audit (SPA).

This audit will be carried out as follows:

Step 1 – Application by the beneficiary.

Step 2 – If the application is accepted, the granting authority will carry out the systems and process

18 Directive 2006/43/EC of the European Parliament and of the Council of 17 May 2006 on statutory audits of annual accounts and consolidated accounts or similar national regulations (OJ L 157, 9.6.2006, p. 87).

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audit, complemented by an audit of transactions (on a sample of the beneficiary’s Horizon Europe or the Euratom Research and Training Programme financial statements).

Step 3 – The audit result will take the form of a risk assessment classification for the beneficiary: low, medium or high.

Low-risk beneficiaries will benefit from less (or less in-depth) ex-post audits (see Article 25) and a higher threshold for submitting certificates on the financial statements (CFS; see Articles 21 and 24.2 and Data Sheet, Point 4.3).

24.5 Consequences of non-compliance

If a beneficiary does not submit a certificate on the financial statements (CFS) or the certificate is rejected, the accepted EU contribution to costs will be capped to reflect the CFS threshold.

If a beneficiary breaches any of its other obligations under this Article, the granting authority may apply the measures described in Chapter 5.

ARTICLE 25 — CHECKS, REVIEWS, AUDITS AND INVESTIGATIONS — EXTENSION OF FINDINGS

25.1 Granting authority checks, reviews and audits

25.1.1 Internal checks

The granting authority may — during the action or afterwards — check the proper implementation of the action and compliance with the obligations under the Agreement, including assessing costs and contributions, deliverables and reports.

25.1.2 Project reviews

The granting authority may carry out reviews on the proper implementation of the action and compliance with the obligations under the Agreement (general project reviews or specific issues reviews).

Such project reviews may be started during the implementation of the action and until the time-limit set out in the Data Sheet (see Point 6). They will be formally notified to the coordinator or beneficiary concerned and will be considered to start on the date of the notification.

If needed, the granting authority may be assisted by independent, outside experts. If it uses outside experts, the coordinator or beneficiary concerned will be informed and have the right to object on grounds of commercial confidentiality or conflict of interest.

The coordinator or beneficiary concerned must cooperate diligently and provide — within the deadline requested — any information and data in addition to deliverables and reports already submitted (including information on the use of resources). The granting authority may request beneficiaries to provide such information to it directly. Sensitive information and documents will be treated in accordance with Article 13.

The coordinator or beneficiary concerned may be requested to participate in meetings, including with the outside experts.

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For on-the-spot visits, the beneficiary concerned must allow access to sites and premises (including to the outside experts) and must ensure that information requested is readily available.

Information provided must be accurate, precise and complete and in the format requested, including electronic format.

On the basis of the review findings, a project review report will be drawn up.

The granting authority will formally notify the project review report to the coordinator or beneficiary concerned, which has 30 days from receiving notification to make observations.

Project reviews (including project review reports) will be in the language of the Agreement, unless otherwise agreed with the granting authority (see Data Sheet, Point 4.2).

25.1.3 Audits

The granting authority may carry out audits on the proper implementation of the action and compliance with the obligations under the Agreement.

Such audits may be started during the implementation of the action and until the time-limit set out in the Data Sheet (see Point 6). They will be formally notified to the beneficiary concerned and will be considered to start on the date of the notification.

The granting authority may use its own audit service, delegate audits to a centralised service or use external audit firms. If it uses an external firm, the beneficiary concerned will be informed and have the right to object on grounds of commercial confidentiality or conflict of interest.

The beneficiary concerned must cooperate diligently and provide — within the deadline requested — any information (including complete accounts, individual salary statements or other personal data) to verify compliance with the Agreement. Sensitive information and documents will be treated in accordance with Article 13.

For on-the-spot visits, the beneficiary concerned must allow access to sites and premises (including for the external audit firm) and must ensure that information requested is readily available.

Information provided must be accurate, precise and complete and in the format requested, including electronic format.

On the basis of the audit findings, a draft audit report will be drawn up.

The auditors will formally notify the draft audit report to the beneficiary concerned, which has 30 days from receiving notification to make observations (contradictory audit procedure).

The final audit report will take into account observations by the beneficiary concerned and will be formally notified to them.

Audits (including audit reports) will be in the language of the Agreement, unless otherwise agreed with the granting authority (see Data Sheet, Point 4.2).

25.2 European Commission checks, reviews and audits in grants of other granting authorities

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Where the granting authority is not the European Commission, the latter has the same rights of checks, reviews and audits as the granting authority.

25.3 Access to records for assessing simplified forms of funding

The beneficiaries must give the European Commission access to their statutory records for the periodic assessment of simplified forms of funding which are used in EU programmes.

25.4 OLAF, EPPO and ECA audits and investigations

The following bodies may also carry out checks, reviews, audits and investigations — during the action or afterwards:

- the European Anti-Fraud Office (OLAF) under Regulations No 883/201319 and No 2185/9620

- the European Public Prosecutor’s Office (EPPO) under Regulation 2017/1939

- the European Court of Auditors (ECA) under Article 287 of the Treaty on the Functioning of the EU (TFEU) and Article 257 of EU Financial Regulation 2018/1046.

If requested by these bodies, the beneficiary concerned must provide full, accurate and complete information in the format requested (including complete accounts, individual salary statements or other personal data, including in electronic format) and allow access to sites and premises for on-the-spot visits or inspections — as provided for under these Regulations.

To this end, the beneficiary concerned must keep all relevant information relating to the action, at least until the time-limit set out in the Data Sheet (Point 6) and, in any case, until any ongoing checks, reviews, audits, investigations, litigation or other pursuits of claims have been concluded.

25.5 Consequences of checks, reviews, audits and investigations — Extension of results of reviews, audits or investigations

25.5.1 Consequences of checks, reviews, audits and investigations in this grant

Findings in checks, reviews, audits or investigations carried out in the context of this grant may lead to rejections (see Article 27), grant reduction (see Article 28) or other measures described in Chapter 5.

Rejections or grant reductions after the final payment will lead to a revised final grant amount (see Article 22).

Findings in checks, reviews, audits or investigations during the action implementation may lead to a request for amendment (see Article 39), to change the description of the action set out in Annex 1.

Checks, reviews, audits or investigations that find systemic or recurrent errors, irregularities, fraud

19 Regulation (EU, Euratom) No 883/2013 of the European Parliament and of the Council of 11 September 2013 concerning investigations conducted by the European Anti-Fraud Office (OLAF) and repealing Regulation (EC) No 1073/1999 of the European Parliament and of the Council and Council Regulation (Euratom) No 1074/1999 (OJ L 248, 18/09/2013, p. 1).

20 Council Regulation (Euratom, EC) No 2185/96 of 11 November 1996 concerning on-the-spot checks and inspections carried out by the Commission in order to protect the European Communities' financial interests against fraud and other irregularities (OJ L 292, 15/11/1996, p. 2).

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or breach of obligations in any EU grant may also lead to consequences in other EU grants awarded under similar conditions (‘extension to other grants’).

Moreover, findings arising from an OLAF or EPPO investigation may lead to criminal prosecution under national law.

25.5.2 Extension from other grants

Results of checks, reviews, audits or investigations in other grants may be extended to this grant, if:

(a) the beneficiary concerned is found, in other EU grants awarded under similar conditions, to have committed systemic or recurrent errors, irregularities, fraud or breach of obligations that have a material impact on this grant and

(b) those findings are formally notified to the beneficiary concerned — together with the list of grants affected by the findings — within the time-limit for audits set out in the Data Sheet (see Point 6).

The granting authority will formally notify the beneficiary concerned of the intention to extend the findings and the list of grants affected.

If the extension concerns rejections of costs or contributions: the notification will include:

(a) an invitation to submit observations on the list of grants affected by the findings

(b) the request to submit revised financial statements for all grants affected

(c) the correction rate for extrapolation, established on the basis of the systemic or recurrent errors, to calculate the amounts to be rejected, if the beneficiary concerned:

(i) considers that the submission of revised financial statements is not possible or practicable or

(ii) does not submit revised financial statements.

If the extension concerns grant reductions: the notification will include:

(a) an invitation to submit observations on the list of grants affected by the findings and

(b) the correction rate for extrapolation, established on the basis of the systemic or recurrent errors and the principle of proportionality.

The beneficiary concerned has 60 days from receiving notification to submit observations, revised financial statements or to propose a duly substantiated alternative correction method/rate.

On the basis of this, the granting authority will analyse the impact and decide on the implementation (i.e. start rejection or grant reduction procedures, either on the basis of the revised financial statements or the announced/alternative method/rate or a mix of those; see Articles 27 and 28).

25.6 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, costs or contributions insufficiently

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substantiated will be ineligible (see Article 6) and will be rejected (see Article 27), and the grant may be reduced (see Article 28).

Such breaches may also lead to other measures described in Chapter 5.

ARTICLE 26 — IMPACT EVALUATIONS

26.1 Impact evaluation

The granting authority may carry out impact evaluations of the action, measured against the objectives and indicators of the EU programme funding the grant.

Such evaluations may be started during implementation of the action and until the time-limit set out in the Data Sheet (see Point 6). They will be formally notified to the coordinator or beneficiaries and will be considered to start on the date of the notification.

If needed, the granting authority may be assisted by independent outside experts.

The coordinator or beneficiaries must provide any information relevant to evaluate the impact of the action, including information in electronic format.

26.2 Consequences of non-compliance

If a beneficiary breaches any of its obligations under this Article, the granting authority may apply the measures described in Chapter 5.

CHAPTER 5 CONSEQUENCES OF NON-COMPLIANCE

SECTION 1 REJECTIONS AND GRANT REDUCTION

ARTICLE 27 — REJECTION OF COSTS AND CONTRIBUTIONS

27.1 Conditions

The granting authority will — at beneficiary termination, interim payment, final payment or afterwards — reject any costs or contributions which are ineligible (see Article 6), in particular following checks, reviews, audits or investigations (see Article 25).

The rejection may also be based on the extension of findings from other grants to this grant (see Article 25).

Ineligible costs or contributions will be rejected.

27.2 Procedure

If the rejection does not lead to a recovery, the granting authority will formally notify the coordinator or beneficiary concerned of the rejection, the amounts and the reasons why. The coordinator or beneficiary concerned may — within 30 days of receiving notification — submit observations if it disagrees with the rejection (payment review procedure).

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If the rejection leads to a recovery, the granting authority will follow the contradictory procedure with pre-information letter set out in Article 22.

27.3 Effects

If the granting authority rejects costs or contributions, it will deduct them from the costs or contributions declared and then calculate the amount due (and, if needed, make a recovery; see Article 22).

ARTICLE 28 — GRANT REDUCTION

28.1 Conditions

The granting authority may — at beneficiary termination, final payment or afterwards — reduce the grant for a beneficiary, if:

(a) the beneficiary (or a person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed:

(i) substantial errors, irregularities or fraud or

(ii) serious breach of obligations under this Agreement or during its award (including improper implementation of the action, non-compliance with the call conditions, submission of false information, failure to provide required information, breach of ethics or security rules (if applicable), etc.), or

(b) the beneficiary (or a person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed — in other EU grants awarded to it under similar conditions — systemic or recurrent errors, irregularities, fraud or serious breach of obligations that have a material impact on this grant (see Article 25).

The amount of the reduction will be calculated for each beneficiary concerned and proportionate to the seriousness and the duration of the errors, irregularities or fraud or breach of obligations, by applying an individual reduction rate to their accepted EU contribution.

28.2 Procedure

If the grant reduction does not lead to a recovery, the granting authority will formally notify the coordinator or beneficiary concerned of the reduction, the amount to be reduced and the reasons why. The coordinator or beneficiary concerned may — within 30 days of receiving notification — submit observations if it disagrees with the reduction (payment review procedure).

If the grant reduction leads to a recovery, the granting authority will follow the contradictory procedure with pre-information letter set out in Article 22.

28.3 Effects

If the granting authority reduces the grant, it will deduct the reduction and then calculate the amount due (and, if needed, make a recovery; see Article 22).

SECTION 2 SUSPENSION AND TERMINATION

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ARTICLE 29 — PAYMENT DEADLINE SUSPENSION

29.1 Conditions

The granting authority may — at any moment — suspend the payment deadline if a payment cannot be processed because:

(a) the required report (see Article 21) has not been submitted or is not complete or additional information is needed

(b) there are doubts about the amount to be paid (e.g. ongoing audit extension procedure, queries about eligibility, need for a grant reduction, etc.) and additional checks, reviews, audits or investigations are necessary, or

(c) there are other issues affecting the EU financial interests.

29.2 Procedure

The granting authority will formally notify the coordinator of the suspension and the reasons why.

The suspension will take effect the day the notification is sent.

If the conditions for suspending the payment deadline are no longer met, the suspension will be lifted — and the remaining time to pay (see Data Sheet, Point 4.2) will resume.

If the suspension exceeds two months, the coordinator may request the granting authority to confirm if the suspension will continue.

If the payment deadline has been suspended due to the non-compliance of the report and the revised report is not submitted (or was submitted but is also rejected), the granting authority may also terminate the grant or the participation of the coordinator (see Article 32).

ARTICLE 30 — PAYMENT SUSPENSION

30.1 Conditions

The granting authority may — at any moment — suspend payments, in whole or in part for one or more beneficiaries, if:

(a) a beneficiary (or a person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed or is suspected of having committed:

(i) substantial errors, irregularities or fraud or

(ii) serious breach of obligations under this Agreement or during its award (including improper implementation of the action, non-compliance with the call conditions, submission of false information, failure to provide required information, breach of ethics or security rules (if applicable), etc.), or

(b) a beneficiary (or a person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed — in other EU grants

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awarded to it under similar conditions — systemic or recurrent errors, irregularities, fraud or serious breach of obligations that have a material impact on this grant.

If payments are suspended for one or more beneficiaries, the granting authority will make partial payment(s) for the part(s) not suspended. If suspension concerns the final payment, the payment (or recovery) of the remaining amount after suspension is lifted will be considered to be the payment that closes the action.

30.2 Procedure

Before suspending payments, the granting authority will send a pre-information letter to the beneficiary concerned:

- formally notifying the intention to suspend payments and the reasons why and

- requesting observations within 30 days of receiving notification.

If the granting authority does not receive observations or decides to pursue the procedure despite the observations it has received, it will confirm the suspension (confirmation letter). Otherwise, it will formally notify that the procedure is discontinued.

At the end of the suspension procedure, the granting authority will also inform the coordinator.

The suspension will take effect the day after the confirmation notification is sent.

If the conditions for resuming payments are met, the suspension will be lifted. The granting authority will formally notify the beneficiary concerned (and the coordinator) and set the suspension end date.

During the suspension, no prefinancing will be paid to the beneficiaries concerned. For interim payments, the periodic reports for all reporting periods except the last one (see Article 21) must not contain any financial statements from the beneficiary concerned (or its affiliated entities). The coordinator must include them in the next periodic report after the suspension is lifted or — if suspension is not lifted before the end of the action — in the last periodic report.

ARTICLE 31 — GRANT AGREEMENT SUSPENSION

31.1 Consortium-requested GA suspension

31.1.1 Conditions and procedure

The beneficiaries may request the suspension of the grant or any part of it, if exceptional circumstances — in particular force majeure (see Article 35) — make implementation impossible or excessively difficult.

The coordinator must submit a request for amendment (see Article 39), with:

- the reasons why

- the date the suspension takes effect; this date may be before the date of the submission of the amendment request and

- the expected date of resumption.

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The suspension will take effect on the day specified in the amendment.

Once circumstances allow for implementation to resume, the coordinator must immediately request another amendment of the Agreement to set the suspension end date, the resumption date (one day after suspension end date), extend the duration and make other changes necessary to adapt the action to the new situation (see Article 39) — unless the grant has been terminated (see Article 32). The suspension will be lifted with effect from the suspension end date set out in the amendment. This date may be before the date of the submission of the amendment request.

During the suspension, no prefinancing will be paid. Costs incurred or contributions for activities implemented during grant suspension are not eligible (see Article 6.3).

31.2 EU-initiated GA suspension

31.2.1 Conditions

The granting authority may suspend the grant or any part of it, if:

(a) a beneficiary (or a person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed or is suspected of having committed:

(i) substantial errors, irregularities or fraud or

(ii) serious breach of obligations under this Agreement or during its award (including improper implementation of the action, non-compliance with the call conditions, submission of false information, failure to provide required information, breach of ethics or security rules (if applicable), etc.), or

(b) a beneficiary (or a person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed — in other EU grants awarded to it under similar conditions — systemic or recurrent errors, irregularities, fraud or serious breach of obligations that have a material impact on this grant

(c) other:

(i) linked action issues: not applicable

(ii) the action has lost its scientific or technological relevance, for EIC Accelerator actions: the action has lost its economic relevance, for challenge-based EIC Pathfinder actions and Horizon Europe Missions: the action has lost its relevance as part of the Portfolio for which it has been initially selected

31.2.2 Procedure

Before suspending the grant, the granting authority will send a pre-information letter to the coordinator:

- formally notifying the intention to suspend the grant and the reasons why and

- requesting observations within 30 days of receiving notification.

If the granting authority does not receive observations or decides to pursue the procedure despite the

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observations it has received, it will confirm the suspension (confirmation letter). Otherwise, it will formally notify that the procedure is discontinued.

The suspension will take effect the day after the confirmation notification is sent (or on a later date specified in the notification).

Once the conditions for resuming implementation of the action are met, the granting authority will formally notify the coordinator a lifting of suspension letter, in which it will set the suspension end date and invite the coordinator to request an amendment of the Agreement to set the resumption date (one day after suspension end date), extend the duration and make other changes necessary to adapt the action to the new situation (see Article 39) — unless the grant has been terminated (see Article 32). The suspension will be lifted with effect from the suspension end date set out in the lifting of suspension letter. This date may be before the date on which the letter is sent.

During the suspension, no prefinancing will be paid. Costs incurred or contributions for activities implemented during suspension are not eligible (see Article 6.3).

The beneficiaries may not claim damages due to suspension by the granting authority (see Article 33).

Grant suspension does not affect the granting authority’s right to terminate the grant or a beneficiary (see Article 32) or reduce the grant (see Article 28).

ARTICLE 32 — GRANT AGREEMENT OR BENEFICIARY TERMINATION

32.1 Consortium-requested GA termination

32.1.1 Conditions and procedure

The beneficiaries may request the termination of the grant.

The coordinator must submit a request for amendment (see Article 39), with:

- the reasons why

- the date the consortium ends work on the action (‘end of work date’) and

- the date the termination takes effect (‘termination date’); this date must be after the date of the submission of the amendment request.

The termination will take effect on the termination date specified in the amendment.

If no reasons are given or if the granting authority considers the reasons do not justify termination, it may consider the grant terminated improperly.

32.1.2 Effects

The coordinator must — within 60 days from when termination takes effect — submit a periodic report (for the open reporting period until termination).

The granting authority will calculate the final grant amount and final payment on the basis of the report submitted and taking into account the costs incurred and contributions for activities implemented

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before the end of work date (see Article 22). Costs relating to contracts due for execution only after the end of work are not eligible.

If the granting authority does not receive the report within the deadline, only costs and contributions which are included in an approved periodic report will be taken into account (no costs/contributions if no periodic report was ever approved).

Improper termination may lead to a grant reduction (see Article 28).

After termination, the beneficiaries’ obligations (in particular Articles 13 (confidentiality and security), 16 (IPR), 17 (communication, dissemination and visibility), 21 (reporting), 25 (checks, reviews, audits and investigations), 26 (impact evaluation), 27 (rejections), 28 (grant reduction) and 42 (assignment of claims)) continue to apply.

32.2 Consortium-requested beneficiary termination

32.2.1 Conditions and procedure

The coordinator may request the termination of the participation of one or more beneficiaries, on request of the beneficiary concerned or on behalf of the other beneficiaries.

The coordinator must submit a request for amendment (see Article 39), with:

- the reasons why

- the opinion of the beneficiary concerned (or proof that this opinion has been requested in writing)

- the date the beneficiary ends work on the action (‘end of work date’)

- the date the termination takes effect (‘termination date’); this date must be after the date of the submission of the amendment request.

If the termination concerns the coordinator and is done without its agreement, the amendment request must be submitted by another beneficiary (acting on behalf of the consortium).

The termination will take effect on the termination date specified in the amendment.

If no information is given or if the granting authority considers that the reasons do not justify termination, it may consider the beneficiary to have been terminated improperly.

32.2.2 Effects

The coordinator must — within 60 days from when termination takes effect — submit:

(i) a report on the distribution of payments to the beneficiary concerned

(ii) a termination report from the beneficiary concerned, for the open reporting period until termination, containing an overview of the progress of the work, the financial statement, the explanation on the use of resources, and, if applicable, the certificate on the financial statement (CFS; see Articles 21 and 24.2 and Data Sheet, Point 4.3)

(iii) a second request for amendment (see Article 39) with other amendments needed (e.g.

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reallocation of the tasks and the estimated budget of the terminated beneficiary; addition of a new beneficiary to replace the terminated beneficiary; change of coordinator, etc.).

The granting authority will calculate the amount due to the beneficiary on the basis of the report submitted and taking into account the costs incurred and contributions for activities implemented before the end of work date (see Article 22). Costs relating to contracts due for execution only after the end of work are not eligible.

The information in the termination report must also be included in the periodic report for the next reporting period (see Article 21).

If the granting authority does not receive the termination report within the deadline, only costs and contributions which are included in an approved periodic report will be taken into account (no costs/ contributions if no periodic report was ever approved).

If the granting authority does not receive the report on the distribution of payments within the deadline, it will consider that:

- the coordinator did not distribute any payment to the beneficiary concerned and that

- the beneficiary concerned must not repay any amount to the coordinator.

If the second request for amendment is accepted by the granting authority, the Agreement is amended to introduce the necessary changes (see Article 39).

If the second request for amendment is rejected by the granting authority (because it calls into question the decision awarding the grant or breaches the principle of equal treatment of applicants), the grant may be terminated (see Article 32).

Improper termination may lead to a reduction of the grant (see Article 31) or grant termination (see Article 32).

After termination, the concerned beneficiary’s obligations (in particular Articles 13 (confidentiality and security), 16 (IPR), 17 (communication, dissemination and visibility), 21 (reporting), 25 (checks, reviews, audits and investigations), 26 (impact evaluation), 27 (rejections), 28 (grant reduction) and 42 (assignment of claims)) continue to apply.

32.3 EU-initiated GA or beneficiary termination

32.3.1 Conditions

The granting authority may terminate the grant or the participation of one or more beneficiaries, if:

(a) one or more beneficiaries do not accede to the Agreement (see Article 40)

(b) a change to the action or the legal, financial, technical, organisational or ownership situation of a beneficiary is likely to substantially affect the implementation of the action or calls into question the decision to award the grant (including changes linked to one of the exclusion grounds listed in the declaration of honour)

(c) following termination of one or more beneficiaries, the necessary changes to the Agreement

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(and their impact on the action) would call into question the decision awarding the grant or breach the principle of equal treatment of applicants

(d) implementation of the action has become impossible or the changes necessary for its continuation would call into question the decision awarding the grant or breach the principle of equal treatment of applicants

(e) a beneficiary (or person with unlimited liability for its debts) is subject to bankruptcy proceedings or similar (including insolvency, winding-up, administration by a liquidator or court, arrangement with creditors, suspension of business activities, etc.)

(f) a beneficiary (or person with unlimited liability for its debts) is in breach of social security or tax obligations

(g) a beneficiary (or person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has been found guilty of grave professional misconduct

(h) a beneficiary (or person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed fraud, corruption, or is involved in a criminal organisation, money laundering, terrorism-related crimes (including terrorism financing), child labour or human trafficking

(i) a beneficiary (or person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) was created under a different jurisdiction with the intent to circumvent fiscal, social or other legal obligations in the country of origin (or created another entity with this purpose)

(j) a beneficiary (or person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed:

(i) substantial errors, irregularities or fraud or

(ii) serious breach of obligations under this Agreement or during its award (including improper implementation of the action, non-compliance with the call conditions, submission of false information, failure to provide required information, breach of ethics or security rules (if applicable), etc.)

(k) a beneficiary (or person having powers of representation, decision-making or control, or person essential for the award/implementation of the grant) has committed — in other EU grants awarded to it under similar conditions — systemic or recurrent errors, irregularities, fraud or serious breach of obligations that have a material impact on this grant (extension of findings from other grants to this grant; see Article 25)

(l) despite a specific request by the granting authority, a beneficiary does not request — through the coordinator — an amendment to the Agreement to end the participation of one of its affiliated entities or associated partners that is in one of the situations under points (d), (f), (e), (g), (h), (i) or (j) and to reallocate its tasks, or

(m) other:

(i) linked action issues: not applicable

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(ii) the action has lost its scientific or technological relevance, for EIC Accelerator actions: the action has lost its economic relevance, for challenge-based EIC Pathfinder actions and Horizon Europe Missions: the action has lost its relevance as part of the Portfolio for which it has been initially selected

32.3.2 Procedure

Before terminating the grant or participation of one or more beneficiaries, the granting authority will send a pre-information letter to the coordinator or beneficiary concerned:

- formally notifying the intention to terminate and the reasons why and

- requesting observations within 30 days of receiving notification.

If the granting authority does not receive observations or decides to pursue the procedure despite the observations it has received, it will confirm the termination and the date it will take effect (confirmation letter). Otherwise, it will formally notify that the procedure is discontinued.

For beneficiary terminations, the granting authority will — at the end of the procedure — also inform the coordinator.

The termination will take effect the day after the confirmation notification is sent (or on a later date specified in the notification; ‘termination date’).

32.3.3 Effects

(a) for GA termination:

The coordinator must — within 60 days from when termination takes effect — submit a periodic report (for the last open reporting period until termination).

The granting authority will calculate the final grant amount and final payment on the basis of the report submitted and taking into account the costs incurred and contributions for activities implemented before termination takes effect (see Article 22). Costs relating to contracts due for execution only after termination are not eligible.

If the grant is terminated for breach of the obligation to submit reports, the coordinator may not submit any report after termination.

If the granting authority does not receive the report within the deadline, only costs and contributions which are included in an approved periodic report will be taken into account (no costs/contributions if no periodic report was ever approved).

Termination does not affect the granting authority’s right to reduce the grant (see Article 28) or to impose administrative sanctions (see Article 34).

The beneficiaries may not claim damages due to termination by the granting authority (see Article 33).

After termination, the beneficiaries’ obligations (in particular Articles 13 (confidentiality and security), 16 (IPR), 17 (communication, dissemination and visibility), 21 (reporting), 25

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(checks, reviews, audits and investigations), 26 (impact evaluation), 27 (rejections), 28 (grant reduction) and 42 (assignment of claims)) continue to apply.

(b) for beneficiary termination:

The coordinator must — within 60 days from when termination takes effect — submit:

(i) a report on the distribution of payments to the beneficiary concerned

(ii) a termination report from the beneficiary concerned, for the open reporting period until termination, containing an overview of the progress of the work, the financial statement, the explanation on the use of resources, and, if applicable, the certificate on the financial statement (CFS; see Articles 21 and 24.2 and Data Sheet, Point 4.3)

(iii) a request for amendment (see Article 39) with any amendments needed (e.g. reallocation of the tasks and the estimated budget of the terminated beneficiary; addition of a new beneficiary to replace the terminated beneficiary; change of coordinator, etc.).

The granting authority will calculate the amount due to the beneficiary on the basis of the report submitted and taking into account the costs incurred and contributions for activities implemented before termination takes effect (see Article 22). Costs relating to contracts due for execution only after termination are not eligible.

The information in the termination report must also be included in the periodic report for the next reporting period (see Article 21).

If the granting authority does not receive the termination report within the deadline, only costs and contributions included in an approved periodic report will be taken into account (no costs/ contributions if no periodic report was ever approved).

If the granting authority does not receive the report on the distribution of payments within the deadline, it will consider that:

- the coordinator did not distribute any payment to the beneficiary concerned and that

- the beneficiary concerned must not repay any amount to the coordinator.

If the request for amendment is accepted by the granting authority, the Agreement is amended to introduce the necessary changes (see Article 39).

If the request for amendment is rejected by the granting authority (because it calls into question the decision awarding the grant or breaches the principle of equal treatment of applicants), the grant may be terminated (see Article 32).

After termination, the concerned beneficiary’s obligations (in particular Articles 13 (confidentiality and security), 16 (IPR), 17 (communication, dissemination and visibility), 21 (reporting), 25 (checks, reviews, audits and investigations), 26 (impact evaluation), 27 (rejections), 28 (grant reduction) and 42 (assignment of claims)) continue to apply.

SECTION 3 OTHER CONSEQUENCES: DAMAGES AND ADMINISTRATIVE SANCTIONS

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ARTICLE 33 — DAMAGES

33.1 Liability of the granting authority

The granting authority cannot be held liable for any damage caused to the beneficiaries or to third parties as a consequence of the implementation of the Agreement, including for gross negligence.

The granting authority cannot be held liable for any damage caused by any of the beneficiaries or other participants involved in the action, as a consequence of the implementation of the Agreement.

33.2 Liability of the beneficiaries

The beneficiaries must compensate the granting authority for any damage it sustains as a result of the implementation of the action or because the action was not implemented in full compliance with the Agreement, provided that it was caused by gross negligence or wilful act.

The liability does not extend to indirect or consequential losses or similar damage (such as loss of profit, loss of revenue or loss of contracts), provided such damage was not caused by wilful act or by a breach of confidentiality.

ARTICLE 34 — ADMINISTRATIVE SANCTIONS AND OTHER MEASURES

Nothing in this Agreement may be construed as preventing the adoption of administrative sanctions (i.e. exclusion from EU award procedures and/or financial penalties) or other public law measures, in addition or as an alternative to the contractual measures provided under this Agreement (see, for instance, Articles 135 to 145 EU Financial Regulation 2018/1046 and Articles 4 and 7 of Regulation 2988/9521).

SECTION 4 FORCE MAJEURE

ARTICLE 35 — FORCE MAJEURE

A party prevented by force majeure from fulfilling its obligations under the Agreement cannot be considered in breach of them.

‘Force majeure’ means any situation or event that:

- prevents either party from fulfilling their obligations under the Agreement,

- was unforeseeable, exceptional situation and beyond the parties’ control,

- was not due to error or negligence on their part (or on the part of other participants involved in the action), and

- proves to be inevitable in spite of exercising all due diligence.

Any situation constituting force majeure must be formally notified to the other party without delay, stating the nature, likely duration and foreseeable effects.

21 Council Regulation (EC, Euratom) No 2988/95 of 18 December 1995 on the protection of the European Communities financial interests (OJ L 312, 23.12.1995, p. 1).

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The parties must immediately take all the necessary steps to limit any damage due to force majeure and do their best to resume implementation of the action as soon as possible.

CHAPTER 6 FINAL PROVISIONS

ARTICLE 36 — COMMUNICATION BETWEEN THE PARTIES

36.1 Forms and means of communication — Electronic management

EU grants are managed fully electronically through the EU Funding & Tenders Portal (‘Portal’).

All communications must be made electronically through the Portal, in accordance with the Portal Terms and Conditions and using the forms and templates provided there (except if explicitly instructed otherwise by the granting authority).

Communications must be made in writing and clearly identify the grant agreement (project number and acronym).

Communications must be made by persons authorised according to the Portal Terms and Conditions. For naming the authorised persons, each beneficiary must have designated — before the signature of this Agreement — a ‘legal entity appointed representative (LEAR)’. The role and tasks of the LEAR are stipulated in their appointment letter (see Portal Terms and Conditions).

If the electronic exchange system is temporarily unavailable, instructions will be given on the Portal.

36.2 Date of communication

The sending date for communications made through the Portal will be the date and time of sending, as indicated by the time logs.

The receiving date for communications made through the Portal will be the date and time the communication is accessed, as indicated by the time logs. Formal notifications that have not been accessed within 10 days after sending, will be considered to have been accessed (see Portal Terms and Conditions).

If a communication is exceptionally made on paper (by e-mail or postal service), general principles apply (i.e. date of sending/receipt). Formal notifications by registered post with proof of delivery will be considered to have been received either on the delivery date registered by the postal service or the deadline for collection at the post office.

If the electronic exchange system is temporarily unavailable, the sending party cannot be considered in breach of its obligation to send a communication within a specified deadline.

36.3 Addresses for communication

The Portal can be accessed via the Europa website.

The address for paper communications to the granting authority (if exceptionally allowed) is the official mailing address indicated on its website.

For beneficiaries, it is the legal address specified in the Portal Participant Register.

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ARTICLE 37 — INTERPRETATION OF THE AGREEMENT

The provisions in the Data Sheet take precedence over the rest of the Terms and Conditions of the Agreement.

Annex 5 takes precedence over the Terms and Conditions; the Terms and Conditions take precedence over the Annexes other than Annex 5.

Annex 2 takes precedence over Annex 1.

ARTICLE 38 — CALCULATION OF PERIODS AND DEADLINES

In accordance with Regulation No 1182/7122, periods expressed in days, months or years are calculated from the moment the triggering event occurs.

The day during which that event occurs is not considered as falling within the period.

‘Days’ means calendar days, not working days.

ARTICLE 39 — AMENDMENTS

39.1 Conditions

The Agreement may be amended, unless the amendment entails changes to the Agreement which would call into question the decision awarding the grant or breach the principle of equal treatment of applicants.

Amendments may be requested by any of the parties.

39.2 Procedure

The party requesting an amendment must submit a request for amendment signed directly in the Portal Amendment tool.

The coordinator submits and receives requests for amendment on behalf of the beneficiaries (see Annex 3). If a change of coordinator is requested without its agreement, the submission must be done by another beneficiary (acting on behalf of the other beneficiaries).

The request for amendment must include:

- the reasons why

- the appropriate supporting documents and

- for a change of coordinator without its agreement: the opinion of the coordinator (or proof that this opinion has been requested in writing).

The granting authority may request additional information.

22 Regulation (EEC, Euratom) No 1182/71 of the Council of 3 June 1971 determining the rules applicable to periods, dates and time-limits (OJ L 124, 8/6/1971, p. 1).

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If the party receiving the request agrees, it must sign the amendment in the tool within 45 days of receiving notification (or any additional information the granting authority has requested). If it does not agree, it must formally notify its disagreement within the same deadline. The deadline may be extended, if necessary for the assessment of the request. If no notification is received within the deadline, the request is considered to have been rejected.

An amendment enters into force on the day of the signature of the receiving party.

An amendment takes effect on the date of entry into force or other date specified in the amendment.

ARTICLE 40 — ACCESSION AND ADDITION OF NEW BENEFICIARIES

40.1 Accession of the beneficiaries mentioned in the Preamble

The beneficiaries which are not coordinator must accede to the grant by signing the accession form (see Annex 3) directly in the Portal Grant Preparation tool, within 30 days after the entry into force of the Agreement (see Article 44).

They will assume the rights and obligations under the Agreement with effect from the date of its entry into force (see Article 44).

If a beneficiary does not accede to the grant within the above deadline, the coordinator must — within 30 days — request an amendment (see Article 39) to terminate the beneficiary and make any changes necessary to ensure proper implementation of the action. This does not affect the granting authority’s right to terminate the grant (see Article 32).

40.2 Addition of new beneficiaries

In justified cases, the beneficiaries may request the addition of a new beneficiary.

For this purpose, the coordinator must submit a request for amendment in accordance with Article 39. It must include an accession form (see Annex 3) signed by the new beneficiary directly in the Portal Amendment tool.

New beneficiaries will assume the rights and obligations under the Agreement with effect from the date of their accession specified in the accession form (see Annex 3).

Additions are also possible in mono-beneficiary grants.

ARTICLE 41 — TRANSFER OF THE AGREEMENT

In justified cases, the beneficiary of a mono-beneficiary grant may request the transfer of the grant to a new beneficiary, provided that this would not call into question the decision awarding the grant or breach the principle of equal treatment of applicants.

The beneficiary must submit a request for amendment (see Article 39), with

- the reasons why

- the accession form (see Annex 3) signed by the new beneficiary directly in the Portal Amendment tool and

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- additional supporting documents (if required by the granting authority).

The new beneficiary will assume the rights and obligations under the Agreement with effect from the date of accession specified in the accession form (see Annex 3).

ARTICLE 42 — ASSIGNMENTS OF CLAIMS FOR PAYMENT AGAINST THE GRANTING AUTHORITY

The beneficiaries may not assign any of their claims for payment against the granting authority to any third party, except if expressly approved in writing by the granting authority on the basis of a reasoned, written request by the coordinator (on behalf of the beneficiary concerned).

If the granting authority has not accepted the assignment or if the terms of it are not observed, the assignment will have no effect on it.

In no circumstances will an assignment release the beneficiaries from their obligations towards the granting authority.

ARTICLE 43 — APPLICABLE LAW AND SETTLEMENT OF DISPUTES

43.1 Applicable law

The Agreement is governed by the applicable EU law, supplemented if necessary by the law of Belgium.

Special rules may apply for beneficiaries which are international organisations (if any; see Data Sheet, Point 5).

43.2 Dispute settlement

If a dispute concerns the interpretation, application or validity of the Agreement, the parties must bring action before the EU General Court — or, on appeal, the EU Court of Justice — under Article 272 of the Treaty on the Functioning of the EU (TFEU).

For non-EU beneficiaries (if any), such disputes must be brought before the courts of Brussels, Belgium — unless an international agreement provides for the enforceability of EU court judgements.

For beneficiaries with arbitration as special dispute settlement forum (if any; see Data Sheet, Point 5), the dispute will — in the absence of an amicable settlement — be settled in accordance with the Rules for Arbitration published on the Portal.

If a dispute concerns administrative sanctions, offsetting or an enforceable decision under Article 299 TFEU (see Articles 22 and 34), the beneficiaries must bring action before the General Court — or, on appeal, the Court of Justice — under Article 263 TFEU.

For grants where the granting authority is an EU executive agency (see Preamble), actions against offsetting and enforceable decisions must be brought against the European Commission (not against the granting authority; see also Article 22).

ARTICLE 44 — ENTRY INTO FORCE

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The Agreement will enter into force on the day of signature by the granting authority or the coordinator, depending on which is later.

SIGNATURES

For the coordinator For the granting authority

[--TGSMark#signature-999997833_75_210--] [--TGSMark#signature-service_75_210--]

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ANNEX 1

Horizon Europe (HORIZON)

Description of the action (DoA)

Part A

Part B

Associated with document Ref. Ares(2024)5602406 - 02/08/2024

Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

DESCRIPTION OF THE ACTION (PART A)

COVER PAGE

Part A of the Description of the Action (DoA) must be completed directly on the Portal Grant Preparation screens.

PROJECT

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Project number: 101156595

Project name: EUROPEAN RARE DISEASES RESEARCH ALLIANCE

Project acronym: ERDERA

Call: HORIZON-HLTH-2023-DISEASE-07

Topic: HORIZON-HLTH-2023-DISEASE-07-01

Type of action: HORIZON-COFUND

Service: HADEA/A/03

Project starting date: fixed date: 1 September 2024

Project duration: 84 months

TABLE OF CONTENTS

Project summary ......................................................................................................................................................3

List of participants .................................................................................................................................................. 3

List of work packages ...........................................................................................................................................11

Staff effort ............................................................................................................................................................. 46

List of deliverables ................................................................................................................................................68

List of milestones (outputs/outcomes) ................................................................................................................ 109

List of critical risks ............................................................................................................................................. 118

Project reviews .................................................................................................................................................... 124

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PROJECT SUMMARY

Project summary

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The European Rare Diseases Research Alliance (ERDERA) aims to improve the health and well-being of the 30 million people living with a rare disease in Europe, by making Europe a world leader in Rare Disease (RD) research and innovation, to support concrete health benefits to rare disease patients, through better prevention, diagnosis and treatment. This Partnership will deliver a RD ecosystem that builds on the successes of previous programmes by supporting robust patient need-led research, developing new diagnostic methods and pathways, spearheading the digital transformational change connecting the dots between care, patient data and research, while ensuring strong alignment of strategies in RD research across countries and regions. Structuring goal-oriented public-private collaborations targeted at interventions all along the R&D value chain will ensure that the journey from knowledge to patient impact is expedited, thereby optimising EU innovation potential in RD. To support its ambition and missions ERDERA has been designed as a comprehensive and integrated ecosystem of which structure can be compared to an institute encompassing three main parts: (i) funding, (ii) internal (in house) Clinical Research Network that implements research activities targeting clinical trial readiness of RDs and accelerating diagnosis and translation of research discovery into improved patient care, and (iii) related supporting services (Data, Expertise, Education and Training) as well as an acceleration hub that serve external and internal RD community, all supported by all-embracing coordination and strategy and foundational (inter)national alignment.

LIST OF PARTICIPANTS

PARTICIPANTS

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Number Role Short name Legal name Country PIC

1 COO INSERM INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE

FR 999997833

1.1 AE CAD Collecteur Analyseur de Données FR 880793078

2 BEN TEAMIT TEAMIT RESEARCH SL ES 896104237

3 BEN DLR DEUTSCHES ZENTRUM FUR LUFT - UND RAUMFAHRT EV

DE 999981731

4 BEN ANR AGENCE NATIONALE DE LA RECHERCHE FR 998711613

5 BEN FTELE FONDAZIONE TELETHON ETS IT 999448716

6 BEN LMT Lietuvos mokslo taryba LT 997808446

7 BEN ZonMw ZORGONDERZOEK NEDERLAND ZON NL 999544164

8 BEN UT EBERHARD KARLS UNIVERSITAET TUEBINGEN

DE 999991916

9 BEN UTARTU TARTU ULIKOOL EE 999895013

10 BEN CNAG CONSORCIO PARA LA EXPLOTACION DEL CENTRO NACIONAL DE ANALISIS GENOMICO

ES 882802433

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

11 BEN SRUMC STICHTING RADBOUD UNIVERSITAIR MEDISCH CENTRUM

NL 892057785

12 BEN TUM TECHNISCHE UNIVERSITAET MUENCHEN DE 999977463

13 BEN UKHD UNIVERSITATSKLINIKUM HEIDELBERG DE 999841081

14 BEN C-PATH CRITICAL PATH INSTITUTE STICHTING NL 887895418

15 BEN VHIR FUNDACIO HOSPITAL UNIVERSITARI VALL D'HEBRON - INSTITUT DE RECERCA

ES 999541642

16 BEN LUMC ACADEMISCH ZIEKENHUIS LEIDEN NL 999990849

17 BEN UTWENTE UNIVERSITEIT TWENTE NL 999900833

18 BEN UPM UNIVERSIDAD POLITECNICA DE MADRID ES 999974844

19 BEN UMCG ACADEMISCH ZIEKENHUIS GRONINGEN NL 999914801

20 BEN AUMC STICHTING AMSTERDAM UMC NL 919322739

21 BEN UM UNIVERSITEIT MAASTRICHT NL 999975911

22 BEN EATRIS EATRIS ERIC NL 941506445

23 BEN FGB FONDAZIONE PER LA RICERCA FARMACOLOGICA GIANNI BENZI ONLUS

IT 962478330

24 BEN APHP ASSISTANCE PUBLIQUE HOPITAUX DE PARIS FR 999645432

25 BEN UHASSELT UNIVERSITEIT HASSELT BE 999874934

26 BEN EURORDIS EURORDIS - RARE DISEASES EUROPE FR 965288323

27 BEN VULSK VIESOJI ISTAIGA VILNIAUS UNIVERSITETO LIGONINE SANTAROS KLINIKOS

LT 991636530

28 BEN UC UNIVERSIDADE DE COIMBRA PT 997826391

29 BEN FFRD FONDATION MALADIES RARES FR 954438485

30 BEN AFM ASS FRANCAISE CONTRE LES MYOPATHIES FR 959828387

31 BEN CCUH BERNU KLINISKA UNIVERSITATES SLIMNICA VALSTS SIA

LV 904374748

32 BEN AICIB AICIB - AGENCIA DE INVESTIGACAO CLINICA E INOVACAO BIOMEDICA

PT 892687606

33 BEN IMGGE INSTITUT ZA MOLEKULARNU GENETIKU I GENETICKO INZENJERSTVO

RS 986427921

34 BEN RDI RARE DISEASES INTERNATIONAL FR 881348694

35 BEN ACU ACIBADEM MEHMET ALI AYDINLAR UNIVERSITY

TR 959704421

36 BEN AIT AIT AUSTRIAN INSTITUTE OF TECHNOLOGY GMBH

AT 999584128

37 BEN AMU UNIVERSITE D'AIX MARSEILLE FR 955518483

38 BEN AOU Meyer IRCCS AZIENDA OSPEDALIERA UNIVERSITARIA MEYER IRCCS

IT 998778543

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

39 BEN ASU-FC AZIENDA SANITARIA UNIVERSITARIA FRIULI CENTRALE

IT 894464355

40 BEN BIU BAR ILAN UNIVERSITY IL 999886574

41 BEN BMBF BUNDESMINISTERIUM FUER BILDUNG UND FORSCHUNG

DE 999835455

42 BEN CCRI GmbH ST. ANNA KINDERKREBSFORSCHUNG GMBH AT 891384896

43 BEN CENTOGENE CENTOGENE GMBH DE 878333449

44 BEN CHARITE CHARITE - UNIVERSITAETSMEDIZIN BERLIN DE 999992692

45 BEN CHI CHECKIMMUNE GMBH DE 889852781

46 BEN CHU Dijon CENTRE HOSPITALIER REG UNIVERSITAIRE DIJON

FR 959994451

47 BEN CE COPENHAGEN ECONOMICS AS DK 938801309

48 BEN REGIONH REGION HOVEDSTADEN DK 999654744

49 BEN CSO-MOH MINISTRY OF HEALTH IL 999596156

50 BEN CVBF CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE

IT 999717018

51 BEN DDF STICHTING DUCHENNE DATA FOUNDATION NL 899914882

52 BEN ELS ELSEVIER BV NL 999937014

53 BEN Erasmus MC ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM

NL 999988424

54 BEN F.R.S.-FNRS FONDS DE LA RECHERCHE SCIENTIFIQUE- FNRS

BE 999540381

55 BEN FHG FRAUNHOFER GESELLSCHAFT ZUR FORDERUNG DER ANGEWANDTEN FORSCHUNG EV

DE 999984059

56 BEN FRRB FONDAZIONE REGIONALE PER LA RICERCA BIOMEDICA

IT 929009450

57 BEN ECRIN ECRIN EUROPEAN CLINICAL RESEARCH INFRASTRUCTURE NETWORK

FR 948646712

58 BEN FWF FONDS ZUR FÖRDERUNG DER WISSENSCHAFTLICHEN FORSCHUNG

AT 998735960

59 BEN IFD INNOVATIONSFONDEN DK 939076692

60 BEN FSJD-CERCA FUNDACIO PRIVADA PER A LA RECERCA I LA DOCENCIA SANT JOAN DE DEU

ES 999565601

61 BEN FCT FUNDACAO PARA A CIENCIA E A TECNOLOGIA

PT 999543970

62 BEN MZd MINISTERSTVO ZDRAVOTNICTVI CESKE REPUBLIKY

CZ 924144512

62.1 AE AZV AGENTURA PRO ZDRAVOTNICKY VYZKUM CESKE REPUBLIKY AZV CR

CZ 878890229

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

63 BEN GNT ASSOCIATION GENETHON FR 998217883

64 BEN GERAD GEORGIAN FOUNDATION FOR GENETIC AND RARE DISEASES

GE 953089312

65 BEN RS REGION STOCKHOLM SE 999522921

66 BEN GUF JOHANN WOLFGANG GOETHE- UNIVERSITAET FRANKFURT AM MAIN

DE 999978724

67 BEN HRB THE HEALTH RESEARCH BOARD IE 999546686

68 BEN İBG IZMIR BIYOTIP VE GENOM MERKEZI TR 906758620

69 BEN Rannis RANNSOKNAMIDSTOD ISLANDS IS 999547268

70 BEN IMAGINE IMAGINE INSTITUT DES MALADIES GENETIQUES NECKER ENFANTS MALADES FONDATION

FR 955276177

71 BEN IGC PAN INSTYTUT GENETYKI CZLOWIEKA POLSKIEJ AKADEMII NAUK

PL 982958425

72 BEN ISCIII INSTITUTO DE SALUD CARLOS III ES 999507886

73 BEN INSA INSTITUTO NACIONAL DE SAUDE DR. RICARDO JORGE

PT 998308190

74 BEN IOR ISTITUTO ORTOPEDICO RIZZOLI IT 999445709

75 BEN ISS ISTITUTO SUPERIORE DI SANITA IT 999978821

76 BEN IT-MOH MINISTERO DELLA SALUTE IT 999531942

77 BEN KU Leuven KATHOLIEKE UNIVERSITEIT LEUVEN BE 999991334

78 BEN LZP LATVIJAS ZINATNES PADOME LV 999546589

79 BEN MRT MAPI RESEARCH TRUST FR 925695542

80 BEN MUS MEDICAL UNIVERSITY SOFIA BG 999857571

81 BEN MUH FAKULTNI NEMOCNICE V MOTOLE CZ 964458391

82 BEN MUR Ministero dell'università e della ricerca IT 894763406

83 BEN NCBR NARODOWE CENTRUM BADAN I ROZWOJU PL 999519720

84 BEN NKUA ETHNIKO KAI KAPODISTRIAKO PANEPISTIMIO ATHINON

EL 999643007

85 BEN MoH SR MINISTERSTVO ZDRAVOTNICTVA SLOVENSKEJ REPUBLIKY

SK 999825173

86 BEN RDG ENOSI SPANION ASTHENON ELLADOS EL 883276763

87 BEN REGIONSYD REGION SYDDANMARK DK 999602073

88 BEN OPBG OSPEDALE PEDIATRICO BAMBINO GESU IT 998837810

89 BEN OSR OSPEDALE SAN RAFFAELE SRL IT 953176030

90 BEN JSI INSTITUT JOZEF STEFAN SI 999971837

91 BEN RCN NORGES FORSKNINGSRAD NO 999460453

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

92 BEN FWO FONDS VOOR WETENSCHAPPELIJK ONDERZOEK-VLAANDEREN

BE 997949484

93 BEN REUH RIGAS AUSTRUMU KLINISKA UNIVERSITATES SLIMNICA SIA

LV 973366483

94 BEN RIF IDRYMA EREVNAS KAI KAINOTOMIAS CY 999946035

95 BEN RCSI ROYAL COLLEGE OF SURGEONS IN IRELAND IE 999867368

96 BEN RSU RIGAS STRADINA UNIVERSITATE LV 999843118

97 BEN Sciensano SCIENSANO BE 906160809

98 BEN SPW SERVICE PUBLIC DE WALLONIE BE 999811884

99 BEN SFU SIGMUND FREUD PRIVATUNIVERSITAT WIEN GMBH

AT 996709824

100 BEN ETAg SIHTASUTUS EESTI TEADUSAGENTUUR EE 998483760

101 BEN SAS SLOVENSKA AKADEMIA VIED SK 999530390

102 BEN Sonio SONIO FR 886915427

103 BEN MOSAE SOTSIAALMINISTEERIUM EE 998429731

104 BEN SRC VETENSKAPSRADET - SWEDISH RESEARCH COUNCIL

SE 999586165

105 BEN TEDDY TEDDY - EUROPEAN NETWORK OF EXCELLENCE FOR PAEDIATRIC CLINICAL RESEARCH

IT 910591963

106 BEN TEKKARE TEKKARE FR 881967069

107 BEN TIF THALASSAEMIA INTERNATIONAL FEDERATION

CY 940669626

108 BEN TUBITAK TURKIYE BILIMSEL VE TEKNOLOJIK ARASTIRMA KURUMU

TR 999587135

109 BEN RT (TuscReg) REGIONE TOSCANA IT 998823842

110 BEN UAB UNIVERSITAT AUTONOMA DE BARCELONA ES 999986484

111 BEN UCD UNIVERSITY COLLEGE DUBLIN, NATIONAL UNIVERSITY OF IRELAND, DUBLIN

IE 999974359

112 BEN UCSC UNIVERSITA CATTOLICA DEL SACRO CUORE IT 999915771

113 BEN UEF ITA-SUOMEN YLIOPISTO FI 991207984

114 BEN UKA UNIVERSITAETSKLINIKUM AACHEN DE 999897632

115 BEN UKLFR UNIVERSITAETSKLINIKUM FREIBURG DE 999881918

116 BEN UNICAMPANIA UNIVERSITA DEGLI STUDI DELLA CAMPANIA LUIGI VANVITELLI

IT 999848356

117 BEN UoC PANEPISTIMIO KRITIS EL 999588978

118 BEN UGENT UNIVERSITEIT GENT BE 999986096

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

119 BEN UKCL UNIVERZITETNI KLINICNI CENTER LJUBLJANA

SI 999882306

120 BEN WDO STICHTING WORLD DUCHENNE ORGANIZATION

NL 930321860

121 BEN UU UPPSALA UNIVERSITET SE 999985029

122 BEN c4c-S CONECT4CHILDREN STICHTING NL 881618063

123 BEN CAU UNIVERSITE CADI AYYAD MA 998639930

124 BEN CNRS CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE CNRS

FR 999997930

125 BEN CIBER CONSORCIO CENTRO DE INVESTIGACION BIOMEDICA EN RED M.P.

ES 997154957

126 BEN UEFISCDI UNITATEA EXECUTIVA PENTRU FINANTAREA INVATAMANTULUI SUPERIOR A CERCETARII DEZVOLTARII SI INOVARII

RO 972130024

127 BEN UNIROMA1 UNIVERSITA DEGLI STUDI DI ROMA LA SAPIENZA

IT 999987745

128 BEN UANTWERPEN UNIVERSITEIT ANTWERPEN BE 999902870

129 BEN BNSF BULGARIAN NATIONAL SCIENCE FUND BG 984222335

130 BEN Aalborg UH REGION NORDJYLLAND (NORTH DENMARK REGION)

DK 997381064

131 BEN RDB REDKI BOLESTI BULGARIA BG 880694720

132 BEN BBMRI-ERIC BIOBANKS AND BIOMOLECULAR RESOURCES RESEARCH INFRASTRUCTURE CONSORTIUM (BBMRI-ERIC)

AT 946597878

133 BEN AarhusUH AARHUS UNIVERSITETSHOSPITAL DK 999643880

134 BEN IABS-EU ASSOCIATION INTERNATIONALE DE STANDARDISATION BIOLOGIQUE POUR L'EUROPE(IABS-EU)

FR 934774936

135 BEN HRCI HEALTH RESEARCH CHARITIES IRELAND COMPANY LIMITED BY GUARANTEE

IE 880683274

136 BEN LMU LUDWIG-MAXIMILIANS-UNIVERSITAET MUENCHEN

DE 999978433

136.1 AE KUM KLINIKUM DER UNIVERSITAT MUNCHEN DE 995625946

137 BEN MSW MYSCIENCEWORK FR 898132119

138 BEN FNR FONDS NATIONAL DE LA RECHERCHE LU 998597153

139 BEN NKFIH NEMZETI KUTATASI FEJLESZTESI ES INNOVACIOS HIVATAL

HU 999578696

140 BEN PEI BUNDESINSTITUT FUR IMPFSTOFFE UND BIOMEDIZINISCHE ARZNEIMITTEL

DE 998217301

141 BEN PLUS PARIS-LODRON-UNIVERSITAT SALZBURG AT 999868047

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

142 BEN SU SORBONNE UNIVERSITE FR 909875521

143 BEN UNISI UNIVERSITA DEGLI STUDI DI SIENA IT 999898020

144 BEN Vinnova VERKET FOR INNOVATIONSSYSTEM SE 999618757

145 BEN CRG-CERCA FUNDACIO CENTRE DE REGULACIO GENOMICA

ES 999544455

146 BEN GMS-RS REGION SKANE SE 998165794

147 BEN VIB VIB VZW BE 999651931

148 BEN IPG INSTITUT DE PATHOLOGIE ET DE GENETIQUE ASBL

BE 878266034

149 BEN UO UNIVERSITY OF OTAGO NZ 998331567

150 BEN HSJD HOSPITAL SANT JOAN DE DEU ES 997929890

151 AP AZ ASTRAZENECA AB SE 999941379

152 AP CHEO-RI CHILDREN'S HOSPITAL OF EASTERN ONTARIO RESEARCH INSTITUTE INC

CA 907681187

153 AP CIHR CANADIAN INSTITUTES OF HEALTH RESEARCH

CA 960368386

154 AP DFG DEUTSCHE FORSCHUNGSGEMEINSCHAFT EV DE 999547462

155 AP FDB FUJIFILM DIOSYNTH BIOTECHNOLOGIES UK LIMITED

UK 962770494

156 AP FRQS FONDS DE RECHERCHE DU QUEBEC - SANTE CA 963122410

157 AP HIPRA HIPRA HUMAN HEALTH SL ES 890821326

158 AP LAB HIPRA LABORATORIOS HIPRA SA ES 991274041

159 AP MICYRN MICYRN CA 880745451

160 AP Miltenyi MILTENYI BIOTEC BV & CO KG DE 897602402

161 AP Pfizer PFIZER INC US 887211374

162 AP RCC DEPARTMENT OF HEALTH GOVERNMENT OF WESTERN AUSTRALIA

AU 960480421

163 AP REI REITHERA SRL IT 998129419

164 AP RJF LABORATORIO REIG JOFRE SA ES 951005073

165 AP ROCHE F. HOFFMANN-LA ROCHE AG CH 999601782

166 AP SNSF SCHWEIZERISCHER NATIONALFONDS ZUR FORDERUNG DER WISSENSCHAFTLICHEN FORSCHUNG

CH 999540575

167 AP UCB UCB BIOPHARMA BE 937666409

168 AP Fraunhofer UK FRAUNHOFER UK RESEARCH LIMITED UK 952487039

169 AP GA UK GENETIC ALLIANCE UK LTD UK 999790932

170 AP UNEW UNIVERSITY OF NEWCASTLE UPON TYNE UK 999985417

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PARTICIPANTS

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Number Role Short name Legal name Country PIC

171 AP CUH Cambridge University Hospitals NHS Foundation Trust

UK 991793961

172 AP ULEIC UNIVERSITY OF LEICESTER UK 999985514

173 AP QUB-UK THE QUEEN'S UNIVERSITY OF BELFAST UK 999992013

174 AP UCL UNIVERSITY COLLEGE LONDON UK 999975620

175 AP UCAM THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE

UK 999977172

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LIST OF WORK PACKAGES

Work packages

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Work Package No

Work Package name Lead Beneficiary Effort (Person- Months)

Start Month

End Month

Deliverables

WP1 Coordination and management 1 - INSERM 607.00 1 84 D1.1 – Data Management Plan D1.2 – Annual Work Plan Y2 D1.3 – Annual Work Plan Y3 D1.4 – Annual Work Plan Y4 D1.5 – Annual Work Plan Y5 D1.6 – Annual Work Plan Y6 D1.7 – Annual Work Plan Y7 D1.8 – Periodic Progress Report 1 D1.9 – Periodic Progress Report 2 D1.10 – Periodic Progress Report 3 D1.11 – Periodic Progress Report 4 D1.12 – Periodic Progress Report 5 D1.13 – RDP SRIA Update D1.14 – Report on cumulative expenditure incurred

WP2 Communication and dissemination 2 - TEAMIT 72.00 1 84 D2.1 – Project website D2.2 – Communication and dissemination strategy

WP3 Joint Transnational Calls for collaborative research projects

3 - DLR 132.00 1 84 D3.1 – Joint Transnational Call documents 1 D3.2 – Joint Transnational Call documents 2 D3.3 – Joint Transnational Call documents 3 D3.4 – Joint Transnational Call documents 4

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Work packages

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Work Package name Lead Beneficiary Effort (Person- Months)

Start Month

End Month

Deliverables

D3.5 – Joint Transnational Call documents 5 D3.6 – Joint Transnational Call documents 6 D3.7 – Joint Transnational Call documents 7 D3.8 – Evaluation Report of the JTCs 1 D3.9 – Evaluation Report of the JTCs 2 D3.10 – Evaluation Report of the JTCs 3 D3.11 – Evaluation Report of the JTCs 4 D3.12 – Evaluation Report of the JTCs 5 D3.13 – Evaluation Report of the JTCs 6 D3.14 – Evaluation Report of the JTCs 7

WP4 Clinical trial call management 5 - FTELE 28.00 1 84 D4.1 – Clinical Trial Call documents D4.2 – Evaluation Report of the Clinical Trial Call

WP5 Networking to share knowledge 6 - LMT 22.00 1 84 D5.1 – NSS Call documents D5.2 – List of funded networks 1 D5.3 – List of funded networks 2

WP6 Diagnostic data availability 8 - UT 614.00 1 84 D6.1 – Roadmap for genomic federated data analysis approach D6.2 – Research dataset on returned diagnostic results 1 D6.3 – Research dataset on returned diagnostic results 2

WP7 Genome re-analysis research pipeline 10 - CNAG 490.00 1 84 D7.1 – Coordinated re-analysis diagnostic pipeline D7.2 – Best practice guidelines for

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Work packages

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Work Package name Lead Beneficiary Effort (Person- Months)

Start Month

End Month

Deliverables

diagnostic re-analysis & reinterpretation strategies

WP8 Innovation to shorten time to RD diagnosis 11 - SRUMC 347.00 1 84 D8.1 – Software for long-read pipelines optimized for RD diagnoses D8.2 – Software update for integrated DNARNA pipeline optimized for RD discoveries 1 D8.3 – Software update for integrated DNARNA pipeline optimized for RD discoveries 2 D8.4 – Software for multi-omics pipeline optimized for RD discoveries 1 D8.5 – Software for multi-omics pipeline optimized for RD discoveries 2

WP9 Real World Data 13 - UKHD 633.00 1 84 D9.1 – Regulatory grade cohort Inventory D9.2 – Individual disease progression models D9.3 – Drug Development Tools

WP10 Clinical Outcome Assessment 15 - VHIR 336.00 1 84 D10.1 – Disease burden and cost of illness framework for RDs D10.2 – Platform for development of regulatorygrade patient-relevant COAs in RD D10.3 – Toolbox on collection of patient selfreported data

WP11 ATMPs 1 - INSERM 210.00 1 84 D11.1 – Result of the PoC studies for ATMP technologies D11.2 – ATMP technology Platform validated in clinical context

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Work packages

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Work Package name Lead Beneficiary Effort (Person- Months)

Start Month

End Month

Deliverables

WP12 N-of-few Approach 16 - LUMC 82.00 1 84 D12.1 – Assessment criteria for patient eligibility to individualised ASO treatment D12.2 – Toolkit on outcome measures for N-of- 1 D12.3 – Standardized platform for timely development of ASO individualized treatments

WP13 RD Virtual Platform (RD-VP) finding and accessing the data ecosystem

1 - INSERM 230.00 1 84 D13.1 – VP specifications and documentation 1 D13.2 – VP specifications and documentation 2 D13.3 – VP specifications and documentation 3

WP14 Data readiness services 16 - LUMC 168.00 1 84 D14.1 – Data Stewardship Wizard with the compilation of data models and data preparation workflows 1 D14.2 – Data Stewardship Wizard with the compilation of data models and data preparation workflows 2 D14.3 – Data Stewardship Wizard with the compilation of data models and data preparation workflows 3

WP15 Data sharing and analysis services 19 - UMCG 224.00 1 84 D15.1 – Portfolio of ERDERA large scale analysis services and pipelines D15.2 – Portfolio of ERDERA federated analysis methods and services D15.3 – Technical guidelines for the implementation of services used in the use cases on federated analysis

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Work packages

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WP16 Knowledge bases and ontologies for RD research 20 - AUMC 250.00 1 84 D16.1 – First standard application of multidimensional knowledge networks (RD maps) addressing an RD case D16.2 – First version of ontologies (antenatal phenotypes and functional impacts) integrated with ORDO D16.3 – Treatabolome demonstrator D16.4 – Knowledge bases and ontologies for PROM/PCOM, disabilities and Treatabolome

WP17 Mentoring and consultancy 22 - EATRIS 38.00 1 84 D17.1 – Recommendations to funders on addressing translatability of projects through funding opportunities D17.2 – Result of feasibility study for consultancy-based expertise services D17.3 – Final report of the mentoring services activity

WP18 Regulatory support service 23 - FGB 88.00 1 84 D18.1 – Set of regulatory recommendations for CRNs D18.2 – Set of Regulatory support tools for preclinical development to facilitate regulatory filings and convergence for RD D18.3 – Roadmap for physical and written standards for quality control of ATMPs D18.4 – Summary report of ERDERA Qualification applications with regulatory Authorities

WP19 Methodological support 24 - APHP 158.00 1 84 D19.1 – First knowledge transfer of

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protocols & tools to CRN and selected JTC projects D19.2 – Final knowledge transfer of protocols & tools to CRN and selected JTC projects D19.3 – First protocol & tools for methodological upscaling of RD research projects D19.4 – Final protocol & tools for methodological upscaling of RD research projects

WP20 Education and Training on RD Research 26 - EURORDIS 318.00 1 84 D20.1 – Report on the identification and fulfilment of RD education and training needs D20.2 – Impact assessment of ERDERA education and training programme

WP21 Technology accelerator 28 - UC 563.00 1 60 D21.1 – Base protocols for research grade rAAVs production D21.2 – Computational tools for of LNPs, EVs and biohybrids formulation development D21.3 – gRNAs for effective and safe gene editing D21.4 – Dataset of Gene therapy Immunogenicity assessment on humanized mouse model D21.5 – Multiomics single cell analysis and bioinformatic pipeline fit-for-purpose qualified to characterize immune response to ATMPs

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D21.6 – Report on standard regulatory quality control parameters of mRNA formulations D21.7 – Regulatory compliant recommendations for rAAV analytics

WP22 Public-Private Collaboration accelerator 29 - FFRD 125.00 1 84 D22.1 – Accelerator Hub inclusion criteria for research projects D22.2 – Impact analysis of Accelerator hub outputs 1 D22.3 – Impact analysis of Accelerator hub outputs 2

WP23 NMGs promotion and national alignment 1 - INSERM 330.00 1 84 D23.1 – Best practice guidelines for creation and promotion of NMGs D23.2 – First report on the impact of NMGs D23.3 – Second report on the impact of NMGs D23.4 – Recommendations on the NMG sustainability

WP24 Fostering engagement of underrepresented countries in ERDERA

32 - AICIB 135.00 1 84 D24.1 – Mapping of UCs specific needs and expectations 1 D24.2 – Mapping of UCs specific needs and expectations 2 D24.3 – Focused guidelines for actions at the National level 1 D24.4 – Focused guidelines for actions at the National level 2 D24.5 – Guidelines to increase UCs integration in ERDERA (including in funding activities) 1

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D24.6 – Guidelines to increase UCs integration in ERDERA (including in funding activities) 2 D24.7 – Guidelines to increase UCs integration in ERDERA (including in funding activities) 3

WP25 ERDERA Global Collaborations 7 - ZonMw 155.00 1 84 D25.1 – White paper on ERNs Living Lab 1 D25.2 – White paper on ERNs Living Lab 2 D25.3 – Sustainability Plan for established strategic alliances and partnerships D25.4 – Publication on the International CRNs

WP26 Ethics requirements 1 - INSERM 0.00 1 84 D26.1 – OEI - Requirement No. 1 D26.2 – OEI - Requirement No. 2 D26.3 – OEI - Requirement No. 3 D26.4 – OEI - Requirement No. 4 D26.5 – OEI - Requirement No. 5 D26.6 – OEI - Requirement No. 6 D26.7 – OEI - Requirement No. 7 D26.8 – OEI - Requirement No. 8

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Work package WP1 – Coordination and management

Work Package Number WP1 Lead Beneficiary 1 - INSERM

Work Package Name Coordination and management

Start Month 1 End Month 84

Objectives

To provide the necessary support for efficient management and coordination of the ERDERA.

Description

T1.1. Day-to-Day management and coordination (M1-M84) TL: INSERM_IT-GGB; Contributors: TEAMIT. Day-to-day management and coordination of the project will be ensured by the coordination team based at INSERM, providing support, and overseeing various aspects, including communication, risk management, and reporting, drawing from the experience of the well-structured EJP RD management office. T1.1. will focus on the planning and reporting related to the contractual obligations included in the Grant Agreement, with the development of the Annual Work Plans and Annual Progress report, including setting up reporting mechanisms, supporting, and training Beneficiaries on reporting procedures and coordinating the production of reports. Risk management is also a priority, identifying, assessing, and following up on threats and opportunities likely to affect the project performance. Additionally, ERDERA will develop a sustainable Data Management Plan ensuring data accountability, reliability, and compliance with IP, ethical, FAIR, and GDPR regulations. It will cover aspects such as data handling, types of data, methodology, sharing and access, curation, preservation, ethical and legal compliance. T1.1. aligns with GO1-3; SO5; OO3,5. T1.2. Governance & strategy (M1-M84) TL: INSERM_IT-GGB; Contributors: TEAMIT, EURORDIS, GA UK*, HRCI, AFM, WDO, RDI. T1.2. aims to establish a governance and a strategy framework for the partnership. The Coordination team (INSERM) will assist all governance and advisory bodies by providing the necessary tools (e.g., secured space for exchange and document storage, voting system), documents (required analyses, working documents, relevant drafts) and prepare the agendas. Additionally, public consultations will be organized, and the Strategic Research and Innovation Agenda (SRIA) will be updated through stakeholder engagement, environmental scanning, including progress and possible synergies with the EU Cancer Mission and the EU Beating Cancer Plan, and SWOT analysis. Patient and Public Involvement & Engagement (PPIE) will be embedded throughout ERDERA, with a core PPIE group guiding engagement practices and ensuring patients' needs are prioritized. T1.2. aligns with GO1-3; SO5; OO3,5. T1.3. Monitoring of the Partnership activities (M1-M84) TL: TEKKARE, CVBF; Contributors: INSERM_ITGGB, ELS,,MSW, CSO-MOH, ANR, FFRD, DLR, ISCIII, ZonMw, LMT, TEAMIT. Monitoring of ERDERA's activities and effectiveness is a key element of T1.3., guaranteed by a multi-level approach, including tracking specific Key Performance Indicators (KPIs), assessing performance at various levels, and collecting data to support decision- making and sustainability planning. This task will also consider criteria on ERDERA Results exploitability by non-rare conditions; application of these criteria to funded research activities will be considered in the funding opportunities, as well as criteria for under-represented countries involvement (widening). ST1.3.1. aims to leverage existing infrastructure and APIs to create a cost-effective, efficient software solution for data collection and monitoring that will constitute a comprehensive monitoring system for the Partnership's needs. ST1.3.2. is about the monitoring of the Partnership's activities across multiple levels: Level 1 - ERDERA as a whole; Level 2 - WPs performance; Level 3 - monitoring of funded projects; Level 4 - governance and strategy. T1.3. aligns with GO1-3; SO5; OO3,5. T1.4. Sustainability strategy (M1-M84) TL: CE; Contributors: INSERM_IT-GGB, TEAMIT. T1.4. is focused on ensuring both the short-term and the long-term vision and viability of the ERDERA through a continuous monitoring and support for IPR and results requiring legal assistance to ensure long-term sustainability; and the development of a plan and procedures for transitioning from a partnership to establishing a self-sustaining institute with its own legal status. It involves managing IP, identifying exploitable results, and preserving consortium interests through a Consortium Agreement. Additionally, it encompasses long-term sustainability planning, emphasizing the value of collective services and data-driven decision-making. The strategy includes defining the vision, mapping business models, and proposing a suitable legal framework while considering costs, financing, and income generation potential, ensuring the Institute's sustained growth within the RD ecosystem. In building the sustainability strategy, the first grant instalment for three years and the output of this period will allow for an in-depth exploration of other sustainability strategies, beyond the institute (for ERDERA as a whole), including sustainability options of the different outputs, fostering ownership of the results by the contributing organisations and the Member States. ERDERA accelerator Hub will also facilitate contacts with potential investors. T1.4. aligns with GO1-3; SO5; OO3,5. T1.5. Ethics compliance (M1-M84) TL: FGB; Contributors: INSERM_IT-GGB. To guarantee and support ethics

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compliance of all project activities, an internal Ethics Advisory Group (EAG) will be set up and composed of ethics experts delegated by beneficiaries. Support will be provided on ethics issues/concerns raised during the implementation of the project and its research activities. This activity will also include the ethics assessment and/or follow-up of funded projects. Different ethics aspects will be addressed according to the Horizon Europe ethics guidance including but not limited to animal welfare, research with humans, data processing activities, involvement of children, AI methods, etc. An Independent Ethics Advisor (IEA) will be appointed to monitor adherence to European ethical and legal frameworks, offering guidance on various aspects, including Data Management Plan. This comprehensive task ensures that ethical standards and compliance are upheld throughout the partnership's lifecycle. T1.5. aligns with GO1-3; SO5; OO3,5.

Work package WP2 – Communication and dissemination

Work Package Number WP2 Lead Beneficiary 2 - TEAMIT

Work Package Name Communication and dissemination

Start Month 1 End Month 84

Objectives

To pave the way for future sustainability of results and outcomes by ensuring broad visibility and understanding that lead to acceptance and uptake, thus, maximising the impact of ERDERA. Specific objectives: (1) Raise awareness about ERDERA vision and mission and continue the process initiated by EJPRD of positioning this ambitious programme as a pivotal actor to advance RD research and build the RD ecosystem in Europe and beyond; (2) Communicate progress, promote resources and services, and disseminate results to build ERDERA’s reputation as an enabler of innovative, standardised, streamlined, and collaborative transnational RD research; (3) In collaboration with all work streams and partners’ comms representatives, maximise outreach and contribute to foster engagement, knowledge exchange, community building, use of services and uptake of results; and (4) Support all WPs in their communication needs, facilitating consistency and alignment with the overall communication strategy, plans, and materials.

Description

T2.1. Communication and dissemination strategy (M1-M84). TL: TEAMIT, INSERM_IT-GGB. T2.1. will design a communication (C) and dissemination (D) strategy for ERDERA by M6, tailored to specific audience groups’ needs and preferences. This strategy will observe Open Science principles and support and enhance ERDERA overall objectives. A comprehensive audience analysis, initially informed by representatives of all work streams and partners’ communication experts, which will constitute the backbone of the C&D strategy. This will be led by the senior communications manager and will include: (1) SWOT analysis that incorporates learnings, achievements, and resources from EJPRD; (2) clear C&D objectives and a set of key messages (generic and specific for each audience group); (3) mapping of audiences, matched with appropriate communication channels and tools (comms. matrix); (4) KPIs to feed into T1.3 for regular evaluation of C&D strategy performance; and (5) explicit roles and responsibilities. T2.1. aligns with GO:3; SO:3,5; OO:3,5. T2.2. C&D operational plans (M6-M84). TL: TEAMIT, INSERM_IT-GGB. The C&D strategy will be implemented through annual work plans designed by T6.2 with the concurrence of WPLs and based on the analysis of the performance evaluation conducted in WP1. These will include SMART objectives, operational pillars, or a pre-emptive calendar of activities, which are audience specific. An editorial plan, led by a scientific writer, will be developed that constructs and supports a coherent ERDERA narrative to drive forward cutting edge ERDERA research, using case studies, personal testimonies, etc. T2.2. will be responsible for the implementation, coordination with partnering organisations and monitoring of annual plans and will collect quarterly KPIs for subsequent evaluation. Finally, specific tactical plans for singled-out tasks will be developed (i.e., coordinated promotion of joint calls) as described in T2.3. A communications officer, with expertise on digital channels, will support the successful delivery of planned activities. T2.2. aligns with GO:3; SO:3; OO:3,5. T2.3. Synergies with work streams and communication tools (M1-M84) TL: TEAMIT. Contributors: INSERM_IT- GGB, EURORDIS, WDO, FFRD, TEDDY. T2.3. will leverage channels, tools, and resources generated by EJP RD to develop ERDERA branding, including communication platforms and materials (website, newsletter, videos, infographics, factsheets, social media assets, etc) to support ERDERA in all communication and dissemination needs. This transversal WP will synergise with all work streams, and will specifically play an active role in: (1) coordinated

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promotion of all funding calls (joint translational, Clinical Trials (CTs) and knowledge sharing), including featuring the progress being made in each funded project and disseminating key results; (2) increase awareness of ERDERA assets and services (data services hub/virtual platform, acceleration hub, knowledge hub, CRNs, training, mentoring); (3) contribute to promote related activities, present case studies and disseminate results; and (4) support outreach and inter(national) capacity alignment activities that includes IRDiRC communications strategy. T2.3. aligns with GO:3; SO:2,3,5; OO:3-5.

Work package WP3 – Joint Transnational Calls for collaborative research projects

Work Package Number WP3 Lead Beneficiary 3 - DLR

Work Package Name Joint Transnational Calls for collaborative research projects

Start Month 1 End Month 84

Objectives

Implementation of seven joint transnational EC co-funded calls through the following tasks: (1) Topics selection and definition of eligibility criteria; (2) JTCs call implementation; and (3) Patient engagement.

Description

T3.1. Call topic selection for JTCs and Clinical Trial (CT) Calls (M1-M84) TL: ANR, DLR, ISCIII, FFRD, FTELE; Contributors: ZonMw, WDO, EURORDIS, all participating funding agencies (BMBF, BNSF, CIHR, CSO-MOH, DFG*, ETAg, F.R.S.-FNRS, FCT, FNR, FRQS, FRRB, FWF, FWO, HRB, IFD, IT-MOH, LMT, LZP, MOSAE, MUR, MZd, NCBR, NKFIH, Rannis, RCN, RIF, RT (TuscReg), SAS, SNSF, SPW, SRC, TUBITAK, UEFISCDI, Vinnova). Identification of topics for the JTCs and CTs calls will be centralised under T3.1. and will include multi-stakeholder input from the RD community inside and outside ERDERA, as well as IRDiRC, and lessons learned from previous E-Rare and EJP RD calls. It is envisioned to formulate a multi-annual call topic strategy with broader themes in consultation with the ERDERA MAB. To maximize synergies, it is expected that the topics for the first three calls will be in line with the CRN themes diagnosis, therapy development, and improved understanding of clinical outcomes. Themes in years 4-7 will take advantage of ATMP technology advancement knowledge derived from WP11 and WP21 as well as facilitate early career researchers and human and social sciences research among other possible themes which will be closely linked to the developments in the CRNs and accelerator hub. To avoid conflicts of interest, a strict firewall between the parties deciding on call topics and partnership beneficiaries will be established. T3.1. aligns with GO:1-3; SO:1-5; OO:1-5. T3.2. JTC implementation (M1-M84) TL: DLR, ANR, FFRD, ISCIII; Contributors: all participating funding agencies (BMBF, BNSF, CIHR, CSO-MOH, DFG*, ETAg, F.R.S.-FNRS, FCT, FNR, FRQS, FRRB, FTELE, FWF, FWO, HRB, IFD, IT-MOH, LMT, LZP, MOSAE, MUR, MZd, NCBR, NKFIH, Rannis, RCN, RIF, RT (TuscReg), SAS, SNSF, SPW, SRC, TUBITAK, UEFISCDI, Vinnova, ZonMW). T3.2. aligns with GO:1-3; SO:1-5; OO:1-5. ST3.2.1. Preparation of the joint transnational calls and establishment of the joint call secretariat: The TL will be responsible for facilitating the definition of eligibility criteria as well as drafting and finalizing all necessary documents including Memorandum of Understanding, Call text, Governance, and evaluation procedures, Guidelines for applicants, Proposal templates. In addition, ST3.2.1. will set up the JCS which will act as central support to the applicants regarding all aspects of proposal drafting and submission. The call for proposals will be published on all relevant channels. The call will be opened for the submission of proposals for at least 60 days. Shortly after publication, a webinar will be held to inform applicants of the call specifics. ST3.2.2. Scientific evaluation of the joint transnational calls: The evaluation will be implemented in a two-step procedure according to the Horizon Europe rules. In the first step, applicants are invited to submit pre-proposals. The eligible applications will first undergo national eligibility check and then be submitted to the transnational peer review Scientific Evaluation Committee (SEC) composed of independent expert scientists chosen for their scientific, technical, patient and disease-specific expertise. The evaluation of proposals will be aligned on the scoring system and criteria of Horizon Europe. The SEC will select the most promising applications for submission of a full proposal. The full proposal step will be used to facilitate inclusion of additional partners from underrepresented countries through a voluntary widening step. Applicants will be able to receive advice on ethical, regulatory, innovation, translational, and data management aspects from ERDERA services and can modify their proposals accordingly. The submitted full proposals will be reviewed by three members of the previously established SEC. These will be complemented by patient experts and methodological experts. The SEC will agree on a final ranking list of excellent proposals recommended for funding. In addition, an

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independent expert will be invited as official observer to the second SEC meeting. The assessment of the observer will be provided in a report to the European Commission. ST3.2.3. Ethics evaluation of projects recommended for funding: An ethics evaluation of the projects selected for funding will be done by independent ethics evaluators following Horizon Europe standards. Only projects that pass the ethics evaluation will be funded. ST3.2.4. Funding decision and kick-off: The Call Steering Committee of participating funders (CSC) will take its final funding decision based strictly on the scientific recommendation of the SEC (ranking list). Each eligible applicant will be funded by the organization of the country/region from which the applicants have applied and according to the national/regional administrative regulations (virtual common pot model). The applicants will be informed about the final funding decision and the national negotiations will commence. An online kick-off meeting will be held for the selected projects. ST3.2.5. Quality management of call procedures: The practicability of administrative and operational mechanisms used for implementation of calls, peer review, funding of projects, and scientific follow-up will be monitored by the JCS with feedback from all participating stakeholders. The lessons learned will feed the call design and implementation of the next joint calls. T3.3. Engagement of patients in research project funding (M1-M84) TL: EURORDIS, FFRD, DLR; Contributors: ANR, ISCIII, ZonMw, RT (TuscReg), WDO, AFM. The working group will focus on improving patient involvement and engagement on all levels of the call process (e.g. call topic definition/eligibility issues for PAOs participation and/or funding/dissemination/evaluation/PPIE-oriented monitoring indicators), but also engagement in research project design. It will facilitate call dissemination and promotion to PAOs. T3.3. aligns with GO:1-3; SO:1,3-5; OO:1,4,5.

Work package WP4 – Clinical trial call management

Work Package Number WP4 Lead Beneficiary 5 - FTELE

Work Package Name Clinical trial call management

Start Month 1 End Month 84

Objectives

(1) Develop the call framework; (2) Open the call and select CTs for funding; (3) Secure proper project implementation, milestones monitoring and financial management; and (4) Leverage the work of the CRN both for the design and the implementation of the studies that will therefore become “demonstrators” of the potential of the CRN itself.

Description

T4.1. Develop the call framework (M1-M18) TL: FTELE, DLR, WDO, QUB-UK*; Contributors: HRB. The partners will define and finalize the text of the call by M18, including timing for proposal submission. The call topic selection and design will be in line with the processes described and in close collaboration with WP3, T3.1. Specifically, this will be an open competitive call, open to researchers, clinical centres, and patient organizations from all Horizon Europe eligible countries. The call text will clarify the eligibility criteria. A minimum of recruiting centres and/or countries will be specified, and the number and role of each centre and partner will need to be defined in the application. The potential roles for companies will be specified and guidelines about their nvolvement (at each stage of the call and of the project) will be provided. The design of the call will consider the model under development by ERA4Health to demonstrate the feasibility of the funding model. The MAB or specific Thematic Groups that could stem from it will be involved in the discussion. The goal is to develop a funding model that overcome bottlenecks encountered under JTC2016 E-Rare-3 and fund multinational RD CTs. In addition, ERDERA will contribute to the Accelerating Clinical Trials in the EU (ACT EU) initiative via ECRIN to integrate their recommendations for multinational CTs in the call text of the ERDERA CTs’ calls. The final call documents will be presented to the Board of Funders for their approval. T4.1. aligns with GO:2; SO:3; OO:1,5. T4.2. Open the call and select trials for funding (M19-M43) TL: FTELE; Contributor: WDO. FTELE will open the first call by M19. The second call will be open by M31. The evaluation process for the applications will include POs patient experts or CABs when available, clinical experts, and experts in trial design and methodology who will provide reports to the group responsible for making the decision. Applicants will be given an opportunity to respond to the peer review after the full proposal evaluation. This will ensure the selection of clinical trial proposals which would adhere to the call requirements, fulfil the eligibility criteria, highest scientific merit, and has potentially a meaningful impact for patients. As a National Research Funding Organisations (RFOs), Fondazione Telethon will establish a call secretariat, and it will manage all the activities related to this task, as well as liaise with other partners if required. Special attention will be

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given to patient-centricity and patient involvement in the proposal and during the project's implementation. Conflict of interest (COI) will be managed for all evaluators. T4.2. aligns with GO:2; SO:3,4; OO:1,4,5. T4.3. Project implementation, project monitoring, and financial management (M38-M84) TL: FTELE; Contributors: IT- MOH, CSO-MOH, WDO. T4.3. will ensure a smooth implementation of the funded trials and follow the achievement of project specific milestones for each funded clinical trial, while it will promote meaningful and impactful research. For each project, funding will be paid in instalments, which will be triggered by the achievement of specific milestones. This is therefore a crucial task to ensure an efficient use of resources. T4.3. aligns with GO:2; SO:1; OO:1,5. This WP comprises two Subtasks: ST 4.3.1. Project implementation and reporting on milestone achievements. ST 4.3.2. Financial management with UPPMD as contributor.

Work package WP5 – Networking to share knowledge

Work Package Number WP5 Lead Beneficiary 6 - LMT

Work Package Name Networking to share knowledge

Start Month 1 End Month 84

Objectives

To share knowledge on research among relevant transnational European and transcontinental RD stakeholders including clinicians, basic researchers, patients’ organisations/RD support groups, as well as PhD students, post-docs and early career researchers, and industry. To implement the networking support scheme for building new European and global research networks or expanding existing research networks on RDs and rare cancers to support patient-centred research, and to include stakeholders of widening countries and young researchers. Realisation of this scheme will address OO5 and will contribute to OO1, OO4, SO3, SO4, SO5, GO1, GO2 and GO3, ensuring knowledge flow and bringing together teams for patient-oriented R&I. Therefore, WP5 will synergize with R&I funding work stream of ERDERA (WP3 and WP4) and could facilitate collaborations (including cross-sectoral) at national, European, and global levels (WP23, WP24 and WP25).

Description

T5.1. Preparation and launching of the funding scheme (M1-M9) TL: LMT, ZonMw; Contributors: FFRD, ANR, EURORDIS, WDO, GA UK*, CSO-MOH, FGB. A call secretariat will be set up and a working group will be formed to prepare call documents (e.g. call text, application template, documents for evaluation procedures and documents for funding of selected networks). The task includes the call launch, building a pool of scientific evaluators (clinicians, researchers, PAO representatives/patients), and preparation of webinar(s) for potential applicants. When the scheme is launched, the funding opportunity will be continuously open with 2 collection dates per year. T5.1. aligns with GO:1-3; SO:3-5; OO:1,4,5. T5.2. Evaluation of the selected proposals after each collection date (M9-M84) TL: LMT, ZonMw; Contributor: FGB. T5.2. will be executed by the call secretariat and comprises of subsequently the following activities: (1) Eligibility check of received applications; (2) Scientific evaluation by the pool of evaluators including patient experts, using the EC Horizon Europe scientific evaluation criteria; (3) Ethics assessment process: This will be performed in the framework of Task 1.6 for the applications that are positively evaluated for funding by the scientific evaluators. The process will consist of evaluating the ethics dimension of the projects and releasing specific ethics requirements to be fulfilled and ethics recommendations to be implemented for each proposal. Attention will be paid to the GDPR compliance for Third Countries (non-EU Member States or EEA countries) on sensitive personal health data. The ethics assessment process will be performed for each round of the call; and (4) Funding decision by the s based on the results of the scientific evaluation and the ethics assessments. T5.2. aligns with GO:1-3; SO:3-5; OO:1,4,5. T5.3 Quality management (M9-M84) TL: LMT, ZonMw; Contributors: FFRD, ANR, EURORDIS, WDO, GA UK*, CSO-MOH, FGB. T5.3 aims to spotlight the practicability of administrative and operational mechanisms used for implementation of the scheme trying to balance the benefit-to-work ratio and with support of WP coordination/ monitoring perform the networks follow-up and monitoring. T5.3 will be performed by the call secretariat with support and feedback from the working group (T5.1. leaders, contributors including the partners responsible for overall ERDERA ethics compliance and scientific monitoring (WP1). The quality of the implementation of the funding scheme will be periodically assessed. Results of this quality management process will be incorporated in the lessons learned reports. The outcomes from each evaluation round will feed the implementation of next collection dates to facilitate the evolution

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of robust and functional administrative and operational practices and ensure a continuous process improvement. T5.3. aligns with GO:1-3; SO:3-5; OO:1,4,5.

Work package WP6 – Diagnostic data availability

Work Package Number WP6 Lead Beneficiary 8 - UT

Work Package Name Diagnostic data availability

Start Month 1 End Month 84

Objectives

(1) Coordinate and consolidate the diagnostic data availability and collation effort in the CRN diagnostic workstream that is based on the existing ERN-led ecosystem, and also comprises national UDPs, national diagnostic centres, programs and national mirror groups; (2) Implement data standardisation, sharing, federation and quality control so that data research re-analysis methodologies can be applied for solving unsolved RD patients and to shorten time to diagnosis; and (3) Ensure access to the data during the project to enable re-analysis of large datasets. This allows a quick return of research results to the clinics, and to RD patients and their families.

Description

T6.1. Coordinate and consolidate the diagnostic research data availability and collation effort in the diagnostic research workstream (M1-M84) TL: UT, CNAG, UTARTU. Contributors: CRG-CERCA, UMCG, CIBER, ISCIII, Aalborg UH, Aarhus UH, AOU Meyer IRCCS, APHP, CCRI GmbH, CHU Dijon, Erasmus MC, IBG, IGC PAN, INSERM_IT-GGB, INSERM_U1112, INSERM_U974, MUS, RCSI, REGIONSYD, UCAM, UNEW, UKCL, UNICAMPANIA, UNISI, UoC, VIB, VULSK, UO, ERN HCPs, diagnostic centres and RD NMGs throughout Europe linked to ERNs and UDPs. T6.1. will focus on coordinating and consolidating the CRN diagnostic research workstream. The following activities will be pursued: (1) Establish and operate a Data Availability Committee to coordinate data availability activities (distributed/ federated approaches); (2) Consolidate research and data policies and agreements in line with 1+MG and GDI; (3) Consolidate standards, minimum quality criteria and processes for omics, phenotypic and meta-data availability and collation in line with GDI and 1+MG; (4) Set up a working group that comprises the different stakeholders (1+MG RD use case, GDI, ERNs and diagnostic centres, ERDERA data service hub) to prepare, pilot, and scale-up the federated data collation approach; (5) Qualify participating ERNs, UDPs, and diagnostic centres in line with 1+MG/GDI, cooperating closely with capacity building in WP20; (6) Establish a network of diagnostic research centres in underrepresented countries based on NMGs, ERNs, Joint Action JARDIN, and 1+MG; and (7) Implement the CRN diagnostic research data management system developed in WP15. T6.1. aligns with GO:1; SO:2,5; OO:3,5. T6.2. Data standardisation, sharing, and federation (M1-M84) TL: CNAG, UTARTU, CAD. Contributors: UT, CRG- CERCA, UMCG, RS, GMS-RS, NKUA, UANTWERPEN, IPG,UO, Aalborg UH, Aarhus UH, AOU Meyer IRCCS, APHP, CCRI GmbH, CHU Dijon, Erasmus MC, IBG, IGC PAN, INSERM_IT-GGB, INSERM_U1112, INSERM_U974, MUS, RCSI, REGIONSYD, UCAM, UNEW, UKCL, UNICAMPANIA, UNISI, UoC, VIB, VULSK, UO ERN HCPs, diagnostic centres throughout Europe linked to ERNs, RD NMGs and UDPs. T6.2. will serve the CRN Diagnostic Research ecosystem by collating REAL (RE-Analysis Logistics) datasets from undiagnosed patients enabling both distributed and federated re-analysis approach. The following activities will be pursued: (1) Establishment of a CRN diagnostic research data availability helpdesk; (2) Making available and federate standardised pheno-clinical information, genomic data (including exomes (Y1-3), short-read (Y1-Y7) and long-read genomes (Y4-Y7) and other omics datasets to serve WP7 and WP8 following standards and quality criteria consolidated in task 6.1.; (3) Ensure all clinical information and metadata is linked to the CRN data management system and files are made available to task 6.3 for distribution across data hubs of the CRN diagnostic research workstream and for returning of research results; (4) Implementation of an automated data quality control system to ensure minimum data quality standards establish in 6.1 are met for delivering reliable results in WP7 and WP8. T6.2. aligns with GO:1; SO:2,5; OO:2. T6.3. Data access and returning research results (M1-M84) TL: UT. Contributors: CNAG, CRG-CERCA, UMCG, CAD, RS, GMS-RS, NKUA, UANTWERPEN, CENTOGENE, IPG, UO, Aalborg UH, Aarhus UH, AOU Meyer IRCCS, APHP, CCRI GmbH, CHU Dijon, Erasmus MC, IBG, IGC PAN, INSERM_IT-GGB, INSERM_U1112, INSERM_U974, MUS, RCSI, REGIONSYD, UCAM, UNEW, UKCL, UNICAMPANIA, UNISI, UoC, VIB, VULSK, UO, ERN HCPs, diagnostic centres throughout Europe linked to ERNs and UDPs, RD NMGs. T6.3. will establish a secure and collaborative data framework that encompasses data access, transfer and distribution as well as returning diagnostic research results for federated and distributed approaches. The following activities will be

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pursued: (1) Alignment with WP14 and WP15 task 15.1 to integrate metadata information and data transfer procedures in the CRN Diagnostic Research management system; (2) Planning data access for research re-analysis, including analysis results (Y1-3); (3) Data transfer from the CRN diagnostic workstream entry points (task 6.2) to points of data analysis according to the needs of WP7 and WP8; (4) Utilising the CRN diagnostic research data management system to return research results following GA4GH policy recommendations in a timely manner including monitoring time-todiagnosis; (5) Periodic submission of relevant variant results information to international public databases like ClinVar. With WP14, we will promote a federated data sustainability model relying on GDI national nodes (e.g. Federated EGA), although central EGA in Barcelona will provide central sustainability until federated model is available (and for partners that cannot submit to a national node). T6.3. aligns with GO:1; SO:2,5; OO:2.

Work package WP7 – Genome re-analysis research pipeline

Work Package Number WP7 Lead Beneficiary 10 - CNAG

Work Package Name Genome re-analysis research pipeline

Start Month 1 End Month 84

Objectives

(1) Coordinate a pan-European genomics diagnostic research pipeline beyond the state-of-the-art diagnostic pipeline; (2) Re-analyse existing data moving from a distributed to a federated one to discover new molecular diagnoses from known, and novel, disease-gene associations; (3) Leverage knowledge and develop best practices to improve variant interpretation across disease groups and ERNs and discover new gene-disease associations; and (4) Facilitate the adoption of knowledge, prepare and share best practice guidelines across disease groups and ERNs.

Description

T7.1. Exome and genome re-analysis pipeline coordination and monitoring (M1-M84) TL: UT, CNAG, SRUMC; Contributors: CHU DIJON, UNEW*, UGENT, ISCIII-IIER, CAD, UU, CENTOGENE, CHEO-RI*, UMCG, EURORDIS, CIBER. T7.1. will establish one Data Analysis Task Force (DATF) and three super-Data Interpretation Task Forces (DITFs) to bring together the expertise from data analysis and clinical researchers. The committee will jointly coordinate and monitor the continuously revised CRN standard diagnostic research pipeline for REAL data sets (e.g. phenotype, pedigree and genetic data), which should be applicable in both a distributed and federated manner (based on Solve-RD, using GRCh38). The committee will also coordinate and monitor the strategy (e.g., data freezes, prioritization and monitoring of re-analysis projects, interpretation strategy, participating groups, and timelines, summarizing and reporting of results and findings), engage with other WPs (e.g., WP6, WP8, Data Services Hub) for data transfer, and align with B1+MG/GDI initiatives. T7.1. aligns with GO:1; SO:5; OO:2,3,5. T7.2. Standardised exome and genome re-analysis beyond state-of-the-art diagnosis (M1-M84) TL: CNAG,CAD; Contributors: UT, SRUMC, UU, CENTOGENE, CHEO-RI*, UMCG, Inserm_Orphanet, CIBER. In Y1-3, standardized re-analysis of REAL data sets will be organized through distributed analysis, the RD-Connect GPAP and other platforms (coordinated via T7.1). In parallel, a federated model will be prepared, in collaboration with WP15, to ensure pipeline portability and implementation, and with those partners having capacity (INSERMCAD, UU). Federated analysis will be favoured as soon as operational and deployment aspects are resolved. The first 18M will focus on exome re-analysis, moving to srWGS, and LRS later (informed by WP8). Analyses will include all variant types, for which consensus algorithms are determined via ‘Data Challenges’. Initially, focus will be on coding regions, shifting to non-coding regions on par with analysing genome REAL data. Variants will be transferred to DITFs (T7.3) for interpretation. T7.2. aligns with GO:1; SO:1,4,5; OO:1,3. T7.3. Innovative exome and genome re-analysis (M7-M84) TL: SRUMC, ISCIII; Contributors: CHU Dijon, UGENT, UNEW*, CENTOGENE, RS(GMS-GMCK), GMS-RS, IMAGINE, CHEO-RI*. Interpretation of variants (from T7.2) will be coordinated by 3 super-DITFs for efficiency and uniformity across all disease groups/ERNs. Super-DITFs are responsible for multiple disease groups/ERNs, combined based on overlapping clinical spectra, or joined through national undiagnosed disease networks. Involvement of local HCPs is achieved via data submission (WP6) and yearly ‘Solvathons’ (years 2-6). First, we will focus on interpretation of existing genomic datasets, and the coding sequence, moving towards non-coding sequence with the availability of genome data and other -omics datasets (informed by WP8). In addition to analysis of known RD disease-genes, focus will include novel disease-gene/mechanism discovery. New diagnoses will be communicated to the patients via the local HCPs/submitters. T7.3. aligns with GO:1; SO:1,4,5; OO:4,5.

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T7.4. Propagation of knowledge and best practice guidelines (M1-M84) TL: KU Leuven, INSERM_U974; Contributors: CAD, IBG, SRUMC, EURORDIS, CNAG. To shorten the pathway to diagnosis and to treatment, two translation tools will be targeted. First, a molecular diagnosis may provide (in)direct leads for therapeutic strategies in n=1 or n=few trials (link to WP12), requiring links with repositories to Treatabolome (link to WP16) and/or other actionable variants for immediate identification. We will provide implementation guidelines to promote implementation in local health care services. Second, the usefulness of knowledge base and ontologies (WP16) will be promoted and evaluated for their performance in clinical context based on their yield. T7.4. aligns with GO:1; SO:1,2; OO:1.

Work package WP8 – Innovation to shorten time to RD diagnosis

Work Package Number WP8 Lead Beneficiary 11 - SRUMC

Work Package Name Innovation to shorten time to RD diagnosis

Start Month 1 End Month 84

Objectives

To establish the most innovative diagnostic approaches for RD to enable a significantly shorter time to diagnosis by full genome analysis, omics integration, and interpretation. These innovations will be crucial to make more RD families amenable to actionability, therapy, and improved care. Several WP8 objectives can be later utilized by joint transnational calls. Specific objectives: (1) Enable complete long-read genome analysis for RD diagnoses as a truly generic and fully complete test; and (2) Develop new genomics and integrated multi-omics approaches to interpret genetic variation enabling improved RD diagnoses and that can be implemented across Europe.

Description

T8.1. Enable inclusion of underrepresented countries into innovative RD diagnostic research (M1-M48) TL: MUH, VULSK, Contributors: SRUMC, TUM, CNAG, UT. T8 will connect RD experts across Europe and let RD clinicians, scientists, and patients from underrepresented countries participate in the diagnostic innovations and train a RD-expert network in selecting RD patients for the T8.2-3 use cases. This capacity building (with WP20.3) leads to optimized RD case selection, increased and faster diagnoses. T8.1. aligns with GO:1; SO:1,2,4; OO:1-4. T8.2. Enable complete long-read genome sequencing and analysis for RD to shorten time to diagnosis (M1- M84) TL: SRUMC, UT; Contributors: UMCG, CHEO-RI*, TUM, CNAG. We will provide RD-optimized longread sequencing (LRS) pipelines. These shall be tested on selected use cases (Task 8.1) to assess diagnostic rate and time to diagnosis. Our LRS experts will analyse and interpret data jointly with disease experts. Innovations will lead to a truly complete and generic diagnostic test, with best practices translated across Europe. T8.2. aligns with GO:1; SO:1,2,4; OO:1-4. T8.3. Enable complete genome mapping for RD to shorten time to diagnosis (M13-M48) TL: SRUMC, UCL*; Contributors: TUM, CNAG, UT. As in T8.2, we will provide RD-optimized ultralong-read optical genome mapping (OGM) pipelines to the RD community, however, with a focus on other use cases, leading to more complete RD diagnostic tests, best practices, and translation across Europe. T8.3. aligns with GO:1; SO:1,2,4; OO:1-4. T8.4. New genomics (transcriptomics) analysis capabilities to understand genetic variation in RD (M1-M84) TL: TUM, CCRI GmbH; Contributors: CUH*, IBG, CNAG, SRUMC, UT. RNA-seq reveals aberrations in expression and splicing missed by genome analyses, leading to increased diagnostic rates and mechanistic insights. Together with national efforts, we will develop algorithms, interpretation guidelines, and interfaces to jointly analyse DNA and bulk and single-cell RNA data. It will include variant effect predictors with (sc)RNAseq-based tools and variant phasing from long-read sequencing. We will record diagnostic rates by aberration type and time each step from the first visit to diagnostic. T8.4. aligns with GO:1; SO:1,2,4; OO:1-4. T8.5. Multi-omics data integration to shorten time to diagnosis in RD (M13-M84) TL: TUM, AMU; Contributors: CCRI GmbH, OPBG, REGIONH, SRUMC, CNAG, UT. T8.1-4 focus on pinpointing the effects of variants on the gene they are located in. However, disease-causing variants can also affect in cascade the function of further genes. These consequences can be used to identify the culprit and understand disease mechanisms. T8.5 will do so by implementing new AI techniques integrating multiscale omics data layers and phenotypic data, with a focus on epigenomics. Every step, data modality, and analysis strategy will be monitored for time and diagnostic rate. T8.5. aligns with GO:1; SO:1,2,4; OO:1-4.

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Work package WP9 – Real World Data

Work Package Number WP9 Lead Beneficiary 13 - UKHD

Work Package Name Real World Data

Start Month 1 End Month 84

Objectives

To augment the trial readiness of real-world data available to the CRN partners. Specific objectives: (1) To develop an efficient system to retrieve primary healthcare data for RD outcome research; (2) To demonstrate the utilization of population-based data in RD outcome research; (3) To generate powerful reference patient cohorts by integration of heterogeneous data sources; (4) To collect and make available regulatory-grade natural history reference data; (5) To develop innovative models to predict disease progression in rare and ultrarare diseases; (6) To develop a platform for clinical trial simulation of RD populations. (7) To demonstrate the usefulness of the ERDERA Data and Expertise Hubs for enhancing the clinical trial readiness and scientific value of RD RWD.

Description

T9.1. Use of primary healthcare data for RD outcome research (M1-M60) TL: SRUMC; Contributors: VHIR, APHP, Erasmus MC, UPM, LMU, KUM. Structured and unstructured EHR data will be extracted and prepared for re-use in registries and outcome research projects. The following disease groups will be studied: Urogenital and craniofacial anomalies, neuromuscular disorders, epileptic encephalopathies, mitochondrial diseases, and rare anemias. Each subproject will involve EHRs at several HCPs, with different platforms (commercial and in-house) and different languages. Different solutions will be benchmarked on a common set of manually annotated EHRs in different languages. In parallel, we will demonstrate for each site how automated extraction from EHRs can be realized. This involves the following steps: (1) Obtain institutional approvals and data use conditions for EHR data extraction from HCPs; (2) Data pseudonymization and determining level of anonymity; (3) Compile structured and/or unstructured data items to be extracted; (4) Implement mappings to existing ontologies and semantic models, starting with common data elements (CDE), semantic model for CDEs and mappings to OMOP; and (5) Create structured output forms from unstructured text using NLP methods and evaluate their utility by clinical domain experts. T9.1. aligns with GO:2,3; SO:2,5; OO:1,2. T9.2. Use of population-based data for RD outcome research (M1-48) TL: APHP; Contributor: Sciensano, UKHD. Innovative approaches to empower population-based outcome research will be developed in T9.2., including the development of a generic minimal interoperable dataset for population-based studies based on linked ERN and social security data. Three case studies will cover different care pathways, including one mostly surgical, one mostly based on successive drug regimens, and one based on neonatal screening: In ST1, exploring Dravet syndrome, the French National RD Registry (BNDMR), and National Health Data System (SNDS) will be combined with the Italian national Dravet registry to study the impact of drug therapies on life expectancy and healthcare costs. Both cohorts will be shared with the EpiCare registry. In ST2, national health insurance data of Hirschsprung disease patients will be linked with outcome data collected nationwide in Belgium, combined with equivalent national healthcare data from BNDMR and SNDS in France, analysed for the impact of phenotype and care pathways on health outcomes, and shared with the ERNICA registry. ST3 will develop a pathway for evaluation of the long-term impact of population-based newborn screening (NBS). A generic NBS module with controlled vocabularies and ontologies will be developed, enabling AI/ ML data readiness. The module will be adapted for metabolic diseases and implemented in MetabERN’s U-IMD registry, which contains longitudinal RWD from patients diagnosed either pre-symptomatically by NBS or after manifestation of symptoms. This will enable evaluation of NBS programmes across various screened diseases and EU countries and involve interactions with the Screen4Rare/ERN multistakeholder platform and ongoing EU initiatives towards genomic NBS (Screen4Care, NGS4NBS). T9.2. aligns with GO:3; SO:2; OO:2. T9.3. Integration of patient cohorts for natural history/standard-of-care reference studies (M1-M60) TL: UKHD; Contributors: APHP, UT, LUMC, SRUMC. T9.3. aims to demonstrate the usefulness of the ERDERA Data Hub services to integrate existing RD registry data into large high-quality reference cohorts for future clinical trials. Four case studies will be performed: (1) Three regional registries from Italy, Germany and France will be joined with the ILIAD registry, and a national French cohort to create a single natural-history cohort of “RASopathies”, a disease group for which molecular therapeutics are becoming available; (2) The international PodoNet Registry of >2,000 children with congenital and steroid resistant nephrotic syndrome will be integrated with the respective sub-cohort in the ERKReg Registry and local databases to form reference populations for emerging first-in-human gene therapy studies in monogenic podocytopathies; (3) Rare endocrine and bone disease cohorts followed in the EuRRECa/EURR- Bone registries will be pooled with institutional and national databases to generate reference outcome data for surgical

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interventions, e.g., in pituitary adenoma or osteogenesis imperfecta; (4) The TreatHSP (hereditary spastic paraplegia), PROSPAX (spastic ataxias) and ARCA (autosomal recessive cerebellar ataxia) registries will be integrated to >5,000 data sets including clinical information, digital mobility measures, molecular biomarkers, and genetic markers to develop advanced disease progression models (task 9.5) and patient-relevant outcome concepts (task 10.1). All integrated cohorts will be FAIRified, onboarded to the ERDERA Virtual Platform and made available in clinical-trial ready formats (tasks 9.4, 13.3, 14.2). The metadata of all cohorts will be deposited in ERDRI. A concept for future integration in the EHDS will be developed. T9.3. aligns with GO:1,3; SO:2; OO:2,3. T9.4. Development of a blueprint and inventory of regulatory-grade natural history cohort data (M1-M84) TL: LMU, KUM; Contributors: UKHD, INSERM_U1112, C-Path. T9.4. aims to establish the procedures for academiadriven registries and cohort studies to achieve EMA qualification to serve as external comparator arms in CTs, post-authorisation safety surveillance and efficacy studies. For such EMA qualification, RWD must fulfil numerous requirements and must undergo extensive and iterative regulatory discussions. We plan to develop a procedural blueprint based on the exemplary processing of three European RD registries and cohort studies (on mitochondrial disorders, hereditary spastic paraparesis and C3 glomerulopathy). From these pilot projects, an inventory and point-by-point guidance will be developed, and comprehensive support provided for registries/cohort studies aiming to achieve regulatory-grade status. The project will be performed in collaboration with EMA’s Data Analysis and Real-World Interrogation Network and coordinated with the IHI consortium for innovative RD therapies. T9.4. aligns with GO:2; SO:2; OO:2. T9.5. Disease progression modelling and prognostic biomarker research (M13-M60) TL: UT; Contributors: VHIR, UPM, UKLFR, UKHD. T9.5. aligns with GO:2,3; SO:1,2; OO:1. T9.5. will develop and apply innovative RD progression modelling methodology in three paradigmatic disease groups: ST9.5.1. Focuses on spastic ataxia diseases (SPAX), a group of >300 ultrarare diseases which share progressive damage to the spinocerebellar and pyramidal tracts. Multimodal longitudinal outcome datasets established in task 9.3 will be utilized for disease progression modelling. An innovative statistical toolbox for individual and n-offew diseases course prediction based on clinical and biomarker profiles (digital- motor datasets, molecular and imaging biomarkers, genetic stratifiers) will be developed and validated. ST9.5.2. Address sickle cell disease, aiming to develop individual risk models to improve disease characterization and health status monitoring, personalize therapeutic approaches and ultimately optimize health outcomes. Further to the longitudinal dataset of the rare anemia ERN registry with >600 variables covering clinical manifestations, organ damage, treatments, and laboratory data mapped in OMOP, multimodal information including metabolomic, genomic (SNP microarrays) and radiomic data (>500 cerebral MRIs) is available for analysis. ST9.5.3. Explore spinal muscular atrophy, using the SMArtCARE registry with longitudinal data from >2000 patients treated with one of the three drugs approved for this disorder. This high-quality longitudinal dataset, which is part of the EURO-NMD registry hub and currently used for regulatory and HTA purposes, will be used to model SMA disease trajectories and to test the reproducibility of clinical trial results in a real-world setting. The projects will receive extensive support from ERDERA modelling experts (WP19). Prior EJP-RD work on individual disease prediction, quantification of uncertainty and simulation of treatment-induced deviations will be developed further towards multimodal outcome progression modelling and use for trial-readiness. The simulation of treatment-induced deviations from predicted individual progression trajectories for ultra-rare neurological diseases will set the stage for the innovative n-of-1 and n- of-few treatment trials envisaged in WP12. T9.6. Development of a model-based clinical trial simulation platform for RDs (M7-M54) TL: C-Path; Contributors: DDF, UKHD. T9.6. will use existing and create new model-building technology to demonstrate the creation of Drug Development Tools (DDTs) that will improve the understanding of RD progression patterns and help predicting the effectiveness and safety of new drugs faster, with more certainty, and at lower costs. Natural history and previous clinical trial data will be used to develop prediction models that allow to simulate therapeutic intervention effects on clinical endpoints and trial sample size requirements, using both conventional and smallpopulation trial designs. Data modelling will be performed on the C-Path RDCA-DAP platform. A strategy for the formal review and potential regulatory endorsement of the quantitative and/or AI based disease progression models as DDTs will be developed. Two therapeutic areas have been selected for DDT development based on their advanced stage of regulatory milestone achievement and initial modelling experience: Duchenne Muscular Dystrophy (DMD) and Autosomal Dominant Polycystic Kidney Disease (ADPKD). The experience of DDF in novel machine learning (ML) methods and C-Path's expertise in developing quantitative solutions will be joined to generate an enhanced CTS tool for DMD. For ADPKD, ERKNet with its registry cohort of >3,000 patients, CPath and the US PKD Outcomes Consortium will undertake a transcontinental collaboration to develop a globally applicable PKD progression model towards regulatory maturity as a DDT. Based on the modelling expertise generated in the ADPKD project, modelling of the eGFR loss endpoint will be considered also for other progressive rare kidney diseases with a therapeutic perspective (e.g. SRNS, IGA nephropathy, complement mediated diseases) for which high-quality RWD is available. T9.6. aligns with GO:2; SO:2; OO:1,2.

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Work package WP10 – Clinical Outcome Assessment

Work Package Number WP10 Lead Beneficiary 15 - VHIR

Work Package Name Clinical Outcome Assessment

Start Month 1 End Month 84

Objectives

WP10 aims to promote the development and integration of PCOMs (PROMs and other COA types) together with PREMs and socioeconomic information for monitoring clinical research (CTs, clinical studies, patient registries) and assess its efficacy and safety for patients together with the evaluation of disease progression: (1) To develop a platform for the development and validation of regulatory-grade PCOMs for RDs which are suitable for establishing truly patient-centred clinical trial endpoints, based on core disease/group of diseases impact sets on the patient as well as societal level; (2) To develop and implement digital tools allowing efficient and broad collection of PCOMs patient self-reported data (PROMs/PREMs, socio-economic information etc) in RD patient cohorts and integration with patient databases and research data; and (3) To systematically explore the societal impact and personal burden of RDs across Europe.

Description

T10.1. Platform for regulatory-grade patient-centred COA development and validation (M1-M84) TL: UKHD; Contributors: VHIR, LMU, KUM, UT, APHP, Erasmus MC, MRT, EURORDIS. In a multistakeholder patient-centred process, we will develop patient-reported outcome assessments and assess patient-relevance of top-ranked COA candidates for five demonstrator RDs (HSP/ataxia [ERN-RND], RASopathies [ERN-Ithaca], mitochondrial disease [5 ERNs], rare anemia disorders [ERN-EuroBloodNet], and craniofacial anomalies [ERN CRANIO]. We will: (1) establish ICF-coded core impact sets for each of the RDs, (2) match ICF-coded functional impacts with suitable existing PROMs (Coll ERICA), and (3) validate PROMs in at least 3 European languages, establishing sensitivity to change, meaningful within-patient change and crosslanguage equivalence. Additionally, anchoring studies for top-ranked COA candidates in the four demonstrator diseases will be performed to establish their patient-relevance. T10.1. aligns with GO:2,3; SO:1-4; OO:1,4,5. T10.2. Development and Implementation of Clinical Outcome Assessment Tools (M1-M84) TL: VHIR; Contributors: UT, APHP, UKHD, UPM, EURORDIS. In T10.2., methodologies to collect and implement PCOMs data linked to patient registries will be developed, considering legal, technological, and regulatory requirements, and validated in 4 case studies using mobile health solutions and devices on rare neurological, neuromuscular, kidney, and haematological disorders. (1) Development of study protocols including the collection of end-user clinical and technical requirement and data standardization (Coll WP13); (2) Identification of available mobile Apps and devices and their readiness (Coll WP14); (3) Clarification of regulatory aspects: mHealth privacy and security protections (Coll WP18); (4) Development/ adaptation of mHealth solutions linked to patient registries; (5) Clinical studies execution; (6) Validation of patient generated data for data quality; and (7) Development of a data visualization framework. T10.2. aligns with GO:2,3; SO:1-4; OO:1,2,5. T10.3. Unveiling the Hidden Burden: Estimating the Socioeconomic Impact of RDs for Informed Decision Making and Resource Allocation (M13-M72) TL: Sciensano; Contributors: Erasmus MC, RDI, EURORDIS, APHP, WDO, CVBF, QUB-UK*. T10.3. will establish and implement a framework for estimating the socioeconomic impact of RDs in Europe based on real-world data through data mapping, Disability Adjusted Life Years (DALY) calculation, and cost estimation in ten RDs and related available registries. 1) Protocol based on harmonized and improved methods for calculating RD DALYs. It will include consensus disease models, outlining the key health states and Disability Weights. 2) Protocol based on harmonized and improved methods for calculating Cost of Illness (COI) associated with RDs, including caregivers. It includes assessing direct medical costs and assessing indirect costs by monetizing Years of Life Lost (YLL). Protocols will include methods to visualize the impact of different scenarios, e.g. impact of new treatment on the DALY and COI estimates. A dedicated stakeholder strategy targeting patient registries owners will foster uptake at national level, aiming for routine quantification of the socioeconomic impact of RDs. T10.3. aligns with GO:2,3; SO:3,4; OO:1,4,5.

Work package WP11 – ATMPs

Work Package Number WP11 Lead Beneficiary 1 - INSERM

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Work Package Name ATMPs

Start Month 1 End Month 84

Objectives

To demonstrate the applications of established innovative, scalable technology platforms of ATMPs developed in the Acceleration hub for prioritized RDs. This WP will collaborate with the two WPs that focus on diagnosis and natural history studies, which are essential for therapy development. In the first year of the project, the objective will be to identify the most urgent and suitable disease groups for ATMP therapies. For following years, objectives will be: (1) To match technology platforms to diseases with greatest need for and benefit from ATMPs; (2) To design proof of concept studies to pressure test selected technology platforms; (3) To assess readiness to enter a clinical development based on available data and technology platform; and (4) To promote ATMP clinical development by establishing a network of qualified clinical trial sites.

Description

T11.1. Identify and rank disease indications requiring ATMPs (M1-M12) TL: UKHD; Contributors: EURORDIS, CVBF. A multistakeholder survey will be performed across the ERDERA. First, general criteria for ATMP treatment suitability will be pre-defined by experts. The main parameters and relative weight in the algorithm will include disease demographics, morbidity/disability, potential for therapeutic efficacy based on mechanistic considerations, availability of established intervention endpoints, natural history studies, efficacy and safety of current therapies, availability of European study cohorts. Priority lists of suitable candidate conditions will be generated, and the most promising candidates will be chosen for demonstrator projects. From the collected information, priority lists of suitable candidate conditions with the greatest anticipated potential benefit from ATMP therapies will be derived for each ATMP class and the most promising candidates will be chosen for demonstrator projects. T11.1. aligns with GO:2; SO:1,2,4,5. T.11.2. Select and adapt the technical platforms with prioritised need (M12-M36) TL: GNT; Contributors: Inserm_ART- ARNm, Pfizer*. Technical platforms with high performance identified among those developed in WP21 will be selected to meet the requirements for the disease list developed in T11.1. The ATMPs experts committee including multiple stakeholders created during T11.2 will prioritize the disease list, minimize the risks associated to the project implementation and identify three diseases for proof of concept. The criteria used for disease selection and the different views brought from the members of the selection committee will be used for the development of a specific algorithm based on AI (fitting gene sequence, gene size, organ/cell type and type of genetic modification and delivery system) that will help the future generations in such prioritisation strategies. T11.2. aligns with GO:2; SO:1,4; OO:2. T11.3. Design the proof-of-concept studies to evaluate the selected pipelines (M36-M72) TL: INSERM_ART-ARNm, INSERM_IT-GGB; Contributors: FHG, GNT. Proof of concept studies will be designed to test the available technology platforms identified in T.11.2 on disease prioritized in T11.1. Human-based disease models of high clinical biomimicry including patient-specific disease models will be privileged whenever available. In case of metabolic diseases, the objective is to evaluate the efficacy of liver targeting in the presence of an underlying diseases, e.g., fatty or fibrotic liver so to better resemble the conditions encountered in western countries. Neurological disorders requiring central nervous system, muscle or dual CNS/muscle targeting represents the current frontier of gene transfer will be a second target. Technologies targeting HSCs after systemic delivery will allow for gene replacement and gene editing thus addressing a large part of rare genetic diseases. The task will be performed with a close interaction between the Accelerator hub and clinical development partners to better implement the clinical translation. T11.3. aligns with GO:2; SO:1,4; OO:2. T11.4. Evaluate the selected platforms for CTs requirement and joint transnational call (M48-M84) Contributors: CVBF, UC, GNT, INSERM_ART-ARNm. The most effective combinations of innovative ATMP and RD in terms of therapeutic efficacy as well as manufacturing capacity and cost-effectiveness will be selected for CTs by a dedicated Committee of ATMP expert (M36-72) and incorporate inputs from experts in WP21 Innovative Therapies Technology Accelerator. Criteria and assets for the selection of platforms/disease combination will be: (1) The possibility to scale-out to GMP manufacturing of the Innovative ATMP; (2) Existence of sufficient data for the authorization of a Clinical Trial Application including a proof-of-concept in an accepted disease model; (3) A positive risk/benefit assessment for the patients; and (4) The existence of a clinical network able to perform the clinical trial with access to the required patients. To this end, we will be launching internal call for partnership to build a regulatory and clinical network supporting pre-IND studies (M24-48). ATMP based CTs will be funded through the JTCs within the WP11 based on the priorities identified in Task 11.1 and the technology readiness assessed by the ATMPs experts’ committee. In addition, based on the advancement status of the preclinical phase, support to identify alternative fundings for the clinical translation will be provided by the WP11 and the Acceleration Hub. T11.4. aligns with GO:2; SO:1,4; OO:2,3.

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Work package WP12 – N-of-few Approach

Work Package Number WP12 Lead Beneficiary 16 - LUMC

Work Package Name N-of-few Approach

Start Month 1 End Month 84

Objectives

(1) Develop an academic platform to develop and test tailored antisense oligonucleotide treatments; and (2) Four use cases to pressure test the platform.

Description

T12.1. Academic platform development (M1-84) TL: LUMC; Contributors: UKHD, UT, SRUMC, UKLFR, WDO, RCC*. T12.1. aims to establish an innovative, scalable platform for academic development and implementation of mutation-specific antisense oligonucleotide (ASO) treatments for individuals with nanorare RD mutations. Along this pipeline will create a combined preclinical/clinical/regulatory framework for tailored n-of-1/n-of-few treatments; and implement, standardize, and optimize each of its modules, processes, and levels. The platform will cover the following aspects: (1) Patient identification (link to diagnostic registries in ERNs and treatment board); (2) ASO design and synthesis (consensus on processes); (3) Efficacy assessment (consensus on processes); (4) Toxicity and safety assessment (consensus on process and reducing the need of animals); (5) Identification of patient relevant outcomes in an n-of-few setting and run-in natural history studies (task 12.2); (6) Treatment/study design and analysis (task 12.3); (7) A registry to capture individualized patient relevant efficacy and safety outcomes before and after treatment; (8) Implementation of first in human treatment infrastructure in n-of-few setting (task 12.4); (9) Regulatory; and (10) Ethical framework (built from pieces generated for each of the aspects. In M1-36, we will build the components of the platform and roll out to one underrepresented country. In M37-84, we will roll out the platform across other centres that will treat patients with individualized therapies and iteratively optimize components based on feedback from the case studies (T12.5) and participating centres. T12.1. aligns with GO:1; SO:1; OO:3,5. T12.2. Identification of patient relevant-outcomes (n-of-1/few) and run in natural history study (M1-84) TL: UT; Contributors: UKHD, LUMC, FSJD-CERCA. T12.2. aims to (1) Develop adapted processes for patient-relevant outcome selection (CRN – Outcome Research) to fit the n-of-1/n-of-few setting; (2) Identify a toolbox of diseasespecific and generic outcomes coordinated by the WP manager during consensus multistakeholder meetings including participants from across Europe (at least 35% underrepresented countries); (3) Implement run-in natural history studies to determine individual sensitivity to change of candidate outcomes selected by patient and clinician and pre-treatment progression trajectories. T12.2. aligns with GO:2; SO:1; OO:3,5. T12.3. Treatment/study design and analysis (M1-84) TL: UT; Contributors: UKHD, UKLFR, LUMC. T12.3. aims to (1) Achieve consensus on strategies to evaluate treatment efficacy on an n-of-1 level (e.g., implementation of staged evaluation milestones: safety – target engagement – efficacy on surrogate parameters – clinical efficacy); and (2) Develop platform trial design that allow to assess efficacy of the platform approach across diseases and treatments, coordinated by the WP manager. T12.3. aligns with GO:2; SO:1; OO:3,5. T12.4. Implementation of first in human treatment infrastructure (M1-84) TL: UKHD; Contributors: UT, LUMC. T12.4. aims to (1) Develop criteria and a process for site qualification; and (2) establish a network of qualified of clinical sites in European countries that can carry out n-of-1 treatments. Criteria will be set, and pressure tested by first use cases by centres with lead experience in implementing this human treatment infrastructure (see Task 12.5) and during consensus meetings, coordinated by the WP manager. The infrastructure will be rolled out to the first underrepresented country in M1-36. T12.4. aligns with GO:2; SO:1; OO:3,5. T12.5. Case studies (M13-M84) TL: UT, UKHD; Contributor: LUMC. The power of the platform generated in Task 12.1 will be shown by 4 use cases, selected to represent different challenging scenarios (e.g. n-of-1; n-of-few; childhood/adult onset; target tissue e.g. brain/eye/liver) to pressure test the platform. We will start with 2 use cases using ASOs for ataxia telangiectasia and POL3RA-related neurodegeneration in M13-36. In M50-84, 2 other use cases will be selected from within the network. These use cases can be either ASOs (using the whole platform) or ATMPs (using aspect 5-9 of the platform). Selection will be facilitated by the selection criteria set in the treatment board (task 12.1) and in collaboration with WP21. Note that for each use case a contribution towards development costs will be provided. T12.5. aligns with GO:2; SO:5; OO:1,3.

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Work package WP13 – RD Virtual Platform (RD-VP) finding and accessing the data ecosystem

Work Package Number WP13 Lead Beneficiary 1 - INSERM

Work Package Name RD Virtual Platform (RD-VP) finding and accessing the data ecosystem

Start Month 1 End Month 84

Objectives

(1) To scale up the federated infrastructure built in EJP RD (RD-VP) by evolving a service-oriented architecture (SOA) in which data, knowledge, and analysis methods from CRN/JTC/CT projects can contribute to the ERDERA data hub as value proposition, at annually increasing rates: To expand the adoption of semantic and technical standards to new sources of data, in order to increase the interoperability and usefulness of the ecosystem for RD research (VP compliance); to steer the expansion (make new resources, including CRN/JTC/CT results, automatically findable within the SOA-based ecosystem) and reusability (allow for querying and reusing data within the connected resources) of the ecosystem in relation to ERDERA workstreams needs, and to support their development; (2) To 40organize and technically lead the teams of Data Hub + CRN/JTC/CT researchers and developers to effectively complete projects that result in increasing numbers of stakeholders contributing to the Data Hub as value proposition (effectuate the CTO role); (3) To support WP14-16 in their collaborations with projects contributing data, analysis, and knowledge to the ERDERA data hub as value proposition; and (4) To ensure the interactions with other ecosystems in Europe and at the international level as far as they respond to ERDERA needs. This WP impacts all GO:1-3; SO:1-5; OO2.

Description

T13.1. VP/data service hub steering (M1-M84) TL: INSERM_Orphanet; Contributors: LUMC;AUMC, UTWENTE, Pfizer*, ROCHE*. Data Service Hub Task leaders, CRNs WS leaders, Funding research WS leaders, National and international alignment WS leaders. The extension and evolution of the RD VP will follow a steering and prioritization process, at the strategic and operational level. The VP is expected to evolve into an intelligent learning system in which newly added resources contribute to statistical, AI, and ML applications. The strategic prioritization of the DSH activities will be done in cooperation with the global ERDERA strategy (task 1.2), to ensure that developments and services in the DSH remain aligned with the SRIA specific objectives, and with the ERDERA overarching prioritization. Special attention will be given to the cooperation with other European and international initiatives. Crosstalk with the ERDERA ELSI, IPR and regulatory services in relation to aspects relevant for the VP will be ensured. The operational decision- making process will be designed according to Agile methods in monthly development cycles. T13.1. aligns with GO:1-3; SO:1-5; OO:2. T13.2. RD-VP evolution and scaling up (M1-M84) TL: UTWENTE, AIT; Contributors: INSERM_Orphanet, BBMRI- ERIC, UPM, GUF, UMCG, LUMC, ULEIC*, CNAG, UKLFR, QUB-UK*. This task has two main responsibilities: (1) Maintain and evolve the RD-VP’s architecture and specifications, and (2) Overlook the developments to guarantee that they conform with the VP’s architecture. T13.2. main activities include: (1) Curation and evolution of the Virtual Platform’s architecture and specifications in collaboration with all involved partners and Tasks 13.1 and 13.3; and (2) Overarching technical developments management of the Virtual Platform to guarantee compliance of the developments with the VP’s architecture and specifications in collaboration with the stakeholders responsible for the developments. Service-oriented evolution of components, including VP Index evolutive maintenance, metadata and data models updates, AAI and consent, PPRL interoperability, core Orphanet services, content discovery services based on Beacon framework, distributed analysis services and VP Portal. T13.2. aligns with GO:1-3; SO:1-5; OO:2. T13.3. VP onboarding services (M1-M84) TL: UMCG, UPM; Participants: SRUMC, LUMC, AUMC. T13.3. is responsible for the maintenance and evolution of the onboarding documentation, services, operations, and support. Based on the steering and prioritization of new resources to be connected to the RD-VP conducted by Task 13.1, Task 13.3 engages with these resources to offer support for the onboarding process, including automation approaches. Onboarding of these resources will track closely with the requirements of WP14 and WP15, to aid in the rapid establishment of analytical pipelines through improved resource discovery. T13.3 will implement practical tools, scripts, and templates to expediate the onboarding process, using ERDERA prioritized software platforms and standards, including assistance with hosting of connected services where needed, providing documentation/SOPs, a support channel and sessions into ERDERA training WPs. Attention will be given to the appropriate capture of legal and IP expectations and constraints, in relation with Task 1.6 – ELSI support service and WP18 Regulatory Support Service, to ensure proper usage, and credit, of the onboarded resources. The onboarding task will closely interact with the mentoring and consultancy service that integrates all ERDERA components and is designed to scale with the number of projects funded through JTC/CT

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and CRN projects. In a direct feedback loop the consultancy will apply onboarding services for new resource types and provide feedback for their evolution. Conversely, the added value of onboarding will be directly demonstrable by analyses that run on onboarded resources. T13.3. aligns with GO:1-3; SO:1-5; OO:2.

Work package WP14 – Data readiness services

Work Package Number WP14 Lead Beneficiary 16 - LUMC

Work Package Name Data readiness services

Start Month 1 End Month 84

Objectives

To expand the use of services by which RD partners contribute to ERDERA’s robust infrastructure of data, resources, and expertise (SRIA OO2) to accelerate the automated ethical and legal exploitation of the healthcare and research data from their projects (based on guidance from WP1 and WP17-19), boosting scientific and regulatory evaluation and healthcare delivery (SRIA SO2). Specific objectives are: (1) To provide a workflow for RD partners to efficiently organise interdisciplinary data readiness teams executing FAIR data creation and FAIR data processing customised to their project, accelerating the expansion of the ERDERA-Data Hub and supporting in-project analysis, federated learning and AI across projects; (2) To provide tools, semantic data models, and specifications that enable RD partners to make data records that they manage ready for automated use if and when accessed, and where needed initiate and monitor the development of missing tools, models and specifications. The enrichment resulting from WP14 pertains to machine actionability and interoperability of data records. The container of the records (e.g. a database or catalogue) must also be FAIR for the records to be findable, accessible, and reusable within the VP (see WP13); (3) To provide tools and services for RD partners to prepare data for the regulatory pathway in drug development, in collaboration with WP9 and WP18; and (4) To contribute to the design and privacy-preserving use of personal data that guarantees that individuals can access data records derived from them, be informed of derivate use of their data, data use conditions can be set at record or subset level, and partitioned data from the same individual can be linked.

Description

T14.1. Services for making data findable, accessible, interoperable, reusable for automated applications (M1-M84) TL: SRUMC, AUMC; Contributors: LUMC, UTWENTE, UPM, WDO, ISS, CNAG, FGB. T14.1. focuses on providing a working model by which RD partners can increase the exploitability of the data that they manage for their project, and for ERDERA, focusing on accelerating the adoption of instructions and tools (T14.2, T14.3) by the ERDERA projects, complying the strategic objectives of the ERDERA (e.g. SRIA SO2 and the GOs it serves)  and in collaboration with NMGs. Following a landscape analysis, it helps organise co-creation teams and workshops, via which a data readiness plan is made and executed. The results are tested by the ERDERA projects, and stakeholders proving that the ERDERA-DH serves multiple stakeholders. The role of PLWRD in viewing and managing their own data is monitored. T14.1. aligns with GO:1-3; SO:2,4,5; OO:1-5. T14.2. Data ingestion tools (M1-M84) TL: UMCG, UPM; Contributors: AUMC, LUMC, SRUMC, AIT, CNAG, CRG- CERCA. T14.2. focuses on providing generic tools that the data readiness teams use to accelerate the usability of data. Data are made usable for responsible federated analysis on connected computing and analysis facilities. It builds on tools and standards previously developed, such as by Solve-RD and the EJP RD. They in turn have built on international collaborations and European projects that investigated implementation of FAIR data principles. Tools for making data ready are identified and developed by co-creation teams, populating the ERDERA-DH. It achieves two goals: (i) make data from CRN/funded projects more exploitable, (ii) evolve data ingestion tools for the ERDERA-DH, particularly focusing on scalability. T14.2. aligns with GO:3; SO:1-3,5; OO:1-3,5. T14.3. Services for advancing the regulatory pathway in drug development (M1-M84) TL: C-PATH, DDF; Contributors: WDO, LUMC. Projects at all stages of the development need to contribute to regulatory approvals to address the unmet medical needs of the RD community in a timely manner and in a way that is meaningful to patients. The development of existing and new predictive tools and methods that qualify as Drug Development Tools (DDTs) require regulatory and data science strategies at design and planning stage to accelerate innovation and the use of Real-World Evidence (RWE) in study design and medicines developments. This task maximises the use of data by tools, accessible to the community, for optimising and accelerating drug development for RDs. The C-Path Rare Disease Cures Accelerator- Data and Analytics Platform (RDCA-DAP®) serves as an accelerator and target platform. It contributes best practices to

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scale up the usability of RD data in the regulatory process (WP9, WP17, WP18). It connects to the patient and regulatory community through the patient advocacy groups of UPPMD and DDF. T14.3. aligns with GO:2; SO:2,4,5; OO:1-5.

Work package WP15 – Data sharing and analysis services

Work Package Number WP15 Lead Beneficiary 19 - UMCG

Work Package Name Data sharing and analysis services

Start Month 1 End Month 84

Objectives

(1) Provide analysis ready reference deployments of the ERDERA analysis infrastructure, including support and interoperability with WP13-14 with ability to scale to cloud; (2) Provide protocols, software and pipelines for large data sets and high-performance computing (HPC) analyses to the infrastructure; and (3) Provide protocols, software and systems that enable federated data analysis use cases and pilots.

Description

T15.1. Data analysis platform deployment (M1-M84) TL: UMCG, ULEIC*; Contributor: CNAG, AIT. T15.1. aims to deliver ready to use analysis infrastructure, integrating output of T15.2-3 and key resources, i.e., high performance compute ‘cloud’ from EJP-RD, Solve-RD, CINECA, EOSC-life; federated analysis by Genomed4All, EUCAN-connect/ DataSHIELD; and user facing analysis services such as the RD-Connect GPAP and MOLGENIS. The infrastructure consists of portable and interoperable packages that can be installed and scaled-up by ERDERA partners on-site or on external (cloud) providers, including a system to track data, metadata, analyses, and the genomic variants interpretation workflow. T15.1. aligns with GO:1-3; SO:1,2,4,5; OO:1-5. T15.2. High performance compute pipeline and ‘cloud’ services (M1-M84) TL: UMCG, CAD; Contributor: CNAG. T15.2. aims to integrate and maintain best practice analysis pipelines that require ‘high performance’ such as genomics, AI and radiomics, building on key resources from RD-Connect GPAP/CNAG, MOLGENIS/UMCG VIP, PFMG2025, Clinical Genomics Sweden, and analysis services such as C-PATH and Orphascape-phenotypic (see task 15.3), and aligned with EOSC, EHDS and ESFRI developments (e.g. LS AAI via GDI project). Main work is to integrate methods prioritized by CRN and funded projects into portable and interoperable HPC analysis pipelines, bundled in a ‘HPC’ in a box template (‘starter kit’) to be installed by research partners to enable first distributed and later federated analysis between the ERDERA partners. T15.2. aligns with GO:1-3; SO:1,2,4,5; OO:1-5. T15.3. Federated analysis (M1-M84) TL: UPM, UU, CNAG; Contributor: UMCG, CAD, AIT, IMAGINE, CHEO-RI*. T15.3. aims to upscale the analysis infrastructure in a federated fashion, leveraging infrastructure from 1+MG/GDI and enabling distributed deployment of T15.1 and T15.2 assets to enable use cases and pilots in Subtask 15.3.1 (Federated analysis and federated learning infrastructure) and Subtask 15.3.2 (Technical support for international federated genome- phenome analysis for research) to realize intra-European and cross-continental analysis capability, starting with large datasets relevant to CRN and JTC projects, aiming to a sustainable approach. T15.3. aligns with GO:1-3; SO:1,2,4,5; OO:1-5.

Work package WP16 – Knowledge bases and ontologies for RD research

Work Package Number WP16 Lead Beneficiary 20 - AUMC

Work Package Name Knowledge bases and ontologies for RD research

Start Month 1 End Month 84

Objectives

(1) Ensure the evolution, harmonisation, extend and adaptation of pre-existing knowledge repositories and ontologies; (2) Address the developing needs of RD researchers and clinicians regarding the use of knowledge resources; (3) Fill identified gaps in knowledge bases; and (4) Demonstrate the utilisation of these resources in supporting diagnosis and treatment research. This WP encompasses the following components: (1) PCOMs repository deployment; (2) Knowledge

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base of prenatal phenotypic features of RDs for early diagnosis; (3) System linking molecular diagnosis to treatment (Treatabolome and RD maps); (4) RD maps – multidimensional knowledge networks – spanning from genetic causes to observed phenotypes and treatment pathways; (5) Ontology of functional impacts of RDs; and (6) HOOM (HPO-ORDO Ontological Module). Orphanet Functional Consequences Ontology (OFCO) covers various aspects related to RDs: (1) Incorporates ontologies like ORDO and HPO used in the RD domain; (2) Includes semantic models such as the EJP-RD semantic model; and (3) Integrates enriched versions of models like OMOP-CDM and CDISC. All resources are linked to the VP and to WS and WPs throughout the ERDERA: (1) Integrated use cases are demonstrated; (2) Tutorials and training offered; and (3) Made accessible for CRNs and funded projects in the form of services and collaborations.

Description

T16.1. Evolving a repository of FAIR Patient-Cantered Outcome Measures (PCOMs)/ Patient-Reported Outcome Measures (PROMs) (M1-M84) TL: INSERM_Orphanet, MRT; Contributor: AUMC. T16.1. aims to expand the PCOMs repository for RD developed in ERICA following the expansion of the annotations of functional impacts of RD performed by Orphanet in collaboration with patient organisations and RD experts, by statistically clustering RD around their functional impacts, to allow for repurposing of existing PCOMs. WP10 will use this repository for PCOMs validation and will feed this repository with newly validated PCOMs. Clustering will be facilitated by the creation of an ontology of functional impacts of RD in T16.5. T16.1. aligns with GO:3; SO:1-3; OO:1. T16.2. Antenatal echographic and pathologic RD phenotypes knowledge base and ontology (M1-M84) TL: INSERM_Orphanet, Sonio. T16.2. aims to leverage on the knowledge being generated by the software and AI for prenatal diagnosis of RDs developed by Sonio, based on academic foetal medicine research, and to create a reference ontology of phenotypic annotations corresponding to detectable RD antenatal abnormalities, by proposing an extension of the HPO and Orphanet content (HOOM), in collaboration with foetal medicine and RD experts. This innovative, openly reusable content will allow for its integration into diagnostic pipelines for early detection and action-taking on RD, including guiding early genetic diagnosis, in the frame of CRN Diagnostics. T16.2. aligns with GO:1; SO:2; OO:1. T.16.3. Treatabolome (M1-M84) TL: CNAG, INSERM_U974. The Treatabolome (http://treatabolome.org/) is a unique, FAIR, resource initially developed in Solve-RD that provides evidence-based links from genes and variants to published treatments with the objective that clinicians diagnosing patients can more easily find the published evidence they need to make appropriate treatment decisions for their newly diagnosed patients. The Treatabolome is encoded using ontologies and standards and the information is available through a user-friendly interface and an API to provide interoperability with other tools such as clinical decision support software. This task will engage with the ERNs to seek expertise on new disease groups and will develop a system to facilitate the entry of new data through literature mining and the curation and update of knowledge through a crowd-sourcing approach. T16.3. aligns with GO:2; SO:1-3; OO:2. T16.4. RD maps (M1-M84) TL: UM, LUMC; Contributor: AMU. T16.4. is dedicated to building tools and services around RD maps connecting essential knowledgebases with each other, to provide models for identification and prioritization of biomarkers for diagnosis and treatment. These RD maps are multi-dimensional models of RDs, starting from molecular interactions – molecular machine-readable pathways developed in previously EJP RD – and connect with established and newly developed knowledgebases from Task 1, 2 and 3 for phenotype and genotype connections and will be annotated with RD ontologies from T5. Additionally, the RD maps will support concrete research questions within the CRNs and funded projects. Within the project we will identify several concrete questions to solve using the RD maps and provide workflows and demonstrators how to create and analyse them, e.g. for ATMP or drug prioritization. The goal for the first 3 years is to establish workflows and interoperability between the resources in how to create the maps, the next 4 years are used for identifying and solving concrete research questions together with the CRNs and “ready” data from WP14 and WP9. T16.4. aligns with GO:1-2; SO:1,2; OO:2. T16.5. Improving, creating, and expanding RD Ontologies and semantic models (M1-M84) TL: AUMC, INSERM_Orphanet; Contributor: LUMC, UM. T16.5. aims to contribute semantically enriched information models and ontologies to the graph-based repository emerging from this WP. On the one hand, existing RD ontologies will be enhanced to allow for better integration with other data into knowledge models, and novel ontologies will be created (antenatal phenotypes of D, T16.2., and functional impact of RD) and integrated with the Orphanet RD ontology (ORDO). On the other hand, semantic models for disease-specific data elements, including PCOMs elements, will be extended or developed, and semantic version of standard information models, including CDISC, OMOP-CDM, and Phenopackets, will be produced. These standardised reference data will be reusable in the frame of CRNs and JTCs research, as well as by Data readiness (WP14) and Data analysis (WP16) activities. T16.5. aligns with GO:1; SO:1,2,5; OO:2.

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Work package WP17 – Mentoring and consultancy

Work Package Number WP17 Lead Beneficiary 22 - EATRIS

Work Package Name Mentoring and consultancy

Start Month 1 End Month 84

Objectives

(1) Continue and further expand the successful EJP RD mentoring programme; (2) Mentoring will be implemented as an ERDERA strategy for projects funded by the alliance (WP3 and WP4) starting at the design phase and throughout the entire life of the project. By adopting the successful light-touch mentoring support scheme from EJP RD, research teams of the funded projects will benefit from a low threshold assessment of the critical milestones that are key for a successful translational development with the objective of reaching the clinic as soon as possible; (3) Perform a feasibility study of a potential workflow of additional expert support based on a consultancy services-based model. This study will analyse the long-term sustainability for this potential service provision. The assessment of this study will be determinant for including this additional service workflow in ERDERA; and (4) Provide feedback to ERDERA partners on the critical barriers for project progression based on the interaction with the research teams and recommendations for profiling the scope of the funding calls.

Description

T17.1. Execution of the Mentoring Program (M1-M84) TL: EATRIS; Contributors: FGB, VHIR. Coordinated and managed by EATRIS, mentoring support will be provided to eligible applicants of the funded projects (WP3 and WP4), who will be informed of the opportunity in the call text of the project calls, as well as through dissemination activities, such as webinars and presentations at suitable meetings and conferences. Selected projects will be offered the support of a dedicated Mentor (or mentors) who will utilize the Innovation Management Toolbox created in EJP RD and the EATRIS database of mentoring experts (also including experts from the partners involved in ERDERA and other institutions (including but not limited to ECRIN, CBVF, c4c, AFM Telethon and ISCIII. Experts will be trained in the mentoring workflow and a comprehensive database will be updated and maintained to cover all the expertise of the RD research value chain. Periodic reports of the mentoring activity will be generated that will be the base for the self-assessment of the process and establish the rationale for eventual adaptations of both at the strategic and operational levels. The mentoring process will also identify potential knowledge and methodology gaps that may require consultancy and/or methodological support. The outcome of the process will be periodic reports of the mentoring activity that will be the base for the self-assessment of the process and establish the rationale for eventual adaptations on both strategic and operational levels. T17.1. aligns with GO:1,2; SO:2,4; OO:2. T17.2. Consultancy service (M1-M84) TL: FGB; Contributor: EATRIS, VHIR. With the aim of providing the best support to the development of projects funded by ERDERA, and in general to the RD community, FGB will lead, during the first funding cycle, a task with the aim of exploring the need and the interest of providing additional expert support in the framework of a consultancy-based support service. Direct feedback from the mentoring service, as well as surveys and interviews with RD research teams will be the basis for a feasibility study that will assess the interest of creating a consultancy-based service as an additional workflow in ERDERA activities. This study will analyse the priorities, workflows, and financial sustainability of these potential service provision approach. A pilot test for assessing the efficiency and implementation of consultancy services will be conducted by FGB during the second funding and identify the feasibility of its implementation (including operational and financial considerations) as an additional service of ERDERA. This task will be developed in 2 subtasks. T17.2. aligns with GO:1,2; SO:4; OO:2.

Work package WP18 – Regulatory support service

Work Package Number WP18 Lead Beneficiary 23 - FGB

Work Package Name Regulatory support service

Start Month 1 End Month 84

Objectives

To provide beneficiaries with a multi-specialistic regulatory support service, ensuring that consortiumderived tools,

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methods, data, and sources are in line with up-to-dated requirements for regulatory filings including qualification. Specific objectives are: (1) To create a dedicated project Regulatory Support Group; (2) To develop new regulatory support tools outlined in tasks 18.1 and 18.2 to facilitate more effective translation of new therapies to the clinic; (3) To provide advice on opportunities, strategy, and timings to engage with regulatory agencies at the appropriate time during the project; (4) To facilitate the applications for regulatory procedures to regulatory procedures to the European Medicines Agency (EMA) or other regulatory bodies; and (5) To support efficient, regulatory-compliant first in man CTs. Regulatory support will be also delivered to researchers from funded projects through consultancy services (Task 17.2).

Description

T18.1. Regulatory support to preclinical research (M1-M84) TL: EATRIS, IABS-EU, Miltenyi*; Contributors: Pfizer*, RJF*, AZ*, UCB*, HIPRA*, LAB HIPRA*, ROCHE*, FDB*. A one stop point to support preclinical research to develop therapeutics, including ATMPs, for RD, in addition to the support outlined in task 18.2 will be created, to facilitate a more accessible regulatory process by helping to implement new standards and tools that help developers meet regulatory requirements early on. To this aim, this ERDERA regulatory support group will: (1) Facilitate successful delivery and qualification of new tools and methodologies, appropriate resources, and databases that support efficient ATMP development for RD; (2) Assess current Pharmacopeia chapters/monographs related to ATMPs for RD for relevance and recommend necessary additions and modifications to be implemented based on the technology platforms and new data generated within ERDERA and beyond, through close interaction with the European Directorate for the Quality of Medicines & Health Care (EDQM); (3) Liaise with regulatory agencies including EMA and FDA; and (4) Develop new SOPs for nonclinical and clinical development in close collaboration with ATMP developers. T18.1. aligns with GO:2; SO:1,2,4,5; OO:2-5. T18.2. Regulatory support to clinical research (M1-M84) TL: FGB, AZ*; Contributors: TEDDY. Task 18.2. will provide advice on regulatory requirements with clinical research and regulatory procedures. This support will encompass different types of clinical studies and regulatory procedures. The research specificities will be addressed: paediatric provisions across all paediatric age groups in case of paediatric studies, provisions for genetic, clinical studies with ATMPs according to Reg. 1394/2007/EC. The Regulatory support group will provide beneficiaries with sources, guidance, recommendations, and webinars on the use of data and samples collected during both routine care and clinical studies for regulatory purposes, e.g. dossiers for marketing authorisation applications of medicines and medical devices, clinical studies. Other aspects will be addressed based on the beneficiaries needs. The Regulatory support group will work with the EAB, the Data hub, the Consultancy service, the CRNs, and the Accelerator Hub. It will facilitate engagement with regulatory agencies, including the EMA, to receive the proper support/advice. The group will also help to identify the most suitable regulatory procedures where the research activities need to be included. Whenever relevant, other regulatory agencies/authorities will be addressed as well. T18.2. aligns with GO:2; SO:1,2,4,5; OO:2-5.

Work package WP19 – Methodological support

Work Package Number WP19 Lead Beneficiary 24 - APHP

Work Package Name Methodological support

Start Month 1 End Month 84

Objectives

To contribute to the knowledge transfer of (bio)statistical, data scientific, epidemiological, AI, and ML expertise with the CRNs and other partners. The emphasis is on CTs, epidemiological studies, registries, RWD, and combinations thereof. This rests on state-of-the art methodology and its implementation. When the clinical trial topics have been consolidated, methodology will be further refined and updated. We will contribute to the WP17 Mentoring and Consultancy within the broad competence of the participating partners. A strong interaction will be foreseen with the WP Data Readiness, the WP Real World Data, the WP Finding and Accessing the Data Ecosystem, the WP Data Readiness Services, and the WP Data Sharing and Analysis Services.

Description

T19.1. Methodological knowledge transfer towards local clinical trial teams (M1-M84) TL: APHP, INSERM_U1138,

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SFU; Contributors: UHASSELT, UKA. Innovative CT designs tailored to small sample sizes are needed for RDs. Often, CT units in the field do not dispose of adequate knowledge or experts to plan CTs using designs for RDs (tailored to patient’s heterogeneity, small sample setting, non-standard endpoints, etc.). We will transfer knowledge in terms of (1) proposing suitable designs for RD clinical questions, (2) providing help in the protocol writing, (3) discussing computational code or statistical package, (4) providing support during the conduct of the trial and in the trial analysis, and (5) participating to the scientific manuscript writing. The idea is not to replace the work of the local teams but to transfer the expert’s knowledge, tools, and confidence as a learning process. Local team should be able to use the first trial as an opportunity to develop new skills to be used in future studies. T19.1. aligns with GO:2; SO:1,2,4,5; OO:2,3. T19.2. Novel methodology for the use of all available knowledge, including RWD (M1-M84) TL: INSERM_1138, UHASSELT, APHP; Contributors: PLUS. T19.2. aims to better characterise and understand patient’s disease trajectories and treatments, in RD, one uses multiple data source coming from patients (i.e., real-world data, registries, clinical trial data, patient reported outcomes, etc.) and preclinical trials findings on the disease pathway and mechanism. Incorporating this knowledge into the design of a clinical trial or RWE is not straightforward and it requires specific methodological considerations. We will propose statistical and ML approaches to maximize the use of existing knowledge. We will propose multi-modal approaches, allowing modelling and analysing multisource (preclinical and clinical) and multidimensional (omics, etc.) data. This will benefit clinical trialists and, evidently patients, as the methodology will enrich information available from trials and therefore enable reducing their length and size. T19.2. aligns with GO:2; SO:1,2,4,5; OO:2-5. T19.3. Data analysis methodology when data are multivariate, hierarchical, incomplete, and of differing data types (M1- M84) TL: UHASSELT, PLUS, APHP; Contributors: UU. In CTs, real-world studies, and other supporting data, data structures are complex (e.g., data of different outcome types, longitudinally collected), yet using the full complexity increases the chances of successful, shorter, and cheaper trials. Adequate models are needed. Their computational feasibility needs to be ascertained, in view of small sample sizes. The use of surrogate marker methodology will further enhance the methodology. Whereas in common diseases it allows to reduce sample size, in the RD context, it can ensure that the maximally available sample size provides enough information to test a clinically meaningful hypothesis. When data are incomplete, the primary analysis strategy should be chosen so that the assumption of Missingness at Random applies. Then, sensitivity analyses should be done to investigate the stability of the results. This has set in motion an entirely new strand of currently ongoing research, ranging from new modelling strategies for incomplete data (e.g., based on multiple imputation), over sensitivity analysis, to computational strategies. This will improve the design and conduct of CTs in RD. T19.3. aligns with GO:1,2; SO:1,4,5; OO:2-5. T19.4. Non-parametric, pharmacometrics and randomization-based methodology (M1-M84) TL: UKA, UU, APHP; Contributors: PLUS, UHASSELT. Methods such as generalized pairwise comparisons have been developed for multiple outcomes, either when a hierarchy in the outcomes is present, or when this is not the case. Their place in the context of RDs should be carefully considered. On the one hand, they are computationally convenient, on the other, their power properties need to be examined for a given trial, such as considered within a CRN. Simulation based evaluation, tailored to the needs of a specific research question, in the context of a specific RD, should be undertaken. Pharmacometrics models based on nonlinear mixed effects analysis of longitudinal data are particularly suitable for RD, given their ability of making use of all relevant data in a structure that allows the use of prior information. T19.4. aligns with GO:2; SO:1,2,4,5; OO:2,3.

Work package WP20 – Education and Training on RD Research

Work Package Number WP20 Lead Beneficiary 26 - EURORDIS

Work Package Name Education and Training on RD Research

Start Month 1 End Month 84

Objectives

To develop a comprehensive programme of education and training on RD research serving the needs across career stages, the R&I pipeline and multistakeholder communities. Specific objectives: (1) Empower patient representatives to enable their full-scale involvement into RD research (T20.1); (2) Support the RD-focused career development of young researchers (T20.1 and T20.4); (3) Empower the RD community for participation in ERDERA and beyond (T20.2 and T20.3); (4) Enable wide dissemination of education and training into national systems (T20.2 and T20.3); and (5) Enable continuous identification and fulfilment of novel, arising cutting-edge education and training needs in tight interaction with all ERDERA workstreams (T20.2.).

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Description

T20.1. Patients and young researchers’ trainings (M1-M84) TL: EURORDIS; Contributors: WDO, DDF, GA UK*, c4c-S, TEDDY, IMAGINE, LUMC. T20.1. aims to train patients and young researchers about the fundamentals of RD research. Training topics will be aligned with ERDERA needs and work streams, specifically the CRNs as they constitute a new component of the RD ecosystem. The core part will rely on the existing EURORDIS Open Academy and will be a scalable approach of training patients and young researchers through a 3-step programme: i) beginner level, ii) intermediate level, iii) advanced level. A strong emphasis will be put on mutual learning from patients and researchers. The training will be delivered through a blended, dynamic, and interactive approach, considering the learning specificities of each target audience. 2-3000 people a year (30% outside of EU) will be trained through the beginner level; up to 100 through the intermediate level (specialised pathways, face-to-face) and at least 30 through the advanced level (online, “Master Classes”). Patient experts participating in ERDERA calls’ evaluation (WP3-4-5) would have completed at least the first two levels. An annual workshop will also be organized for up to 20 young patients (12-18 years old) building upon EJP RD paediatric trainings. T20.1. aligns with GO:3; SO:3,4; OO:1,3,4. T20.2. Identification and fulfilment of training needs (M1-M84) TL: VULSK, ASU FC; Contributors: EURORDIS, FFRD, UCSC. T20.2. aims to ensure a continuous process for identification of education and training needs and to create a strong basis for the introduction of new trainings. T20.2. aligns with GO:3; SO:4; OO:2,3,4. ST20.2.1 Continuous identification of training needs (M1-M84) will be performed using different sources: ERDERA workstreams; NMGs (with WP23); interaction with the global RD ecosystem (with WP25). ST20.2.2 Implementation of novel horizon- scanning trainings (M13-M84). For novel arising needs, expert-level horizon-scanning trainings will be organized with the engagement of the most prominent experts in the field. ST20.2.3. Implementation of the train-the-trainer principle (M1-M84). National training needs will be identified and implemented in collaboration with WP23 applying the train- the-trainer principle to achieve a “snowball effect”: inclusion and dedicated support for national representatives in WP20 and implementation of adapted national trainings in WP23. Providers of education and training will define standardization rules for national training programmes and will support and supervise national trainers. At year 3, evaluation of the state-of-the-art of the ERDERA research and education programme will be accomplished to inform further developments. T20.3. RD research trainings for multistakeholder communities (M1-M84) TL: CNAG, VULSK; Contributors: UT, SRUMC, UTARTU, TUM, EATRIS, GA UK*, Fraunhofer UK*, ECRIN, SFU, UHASSELT, ISS, LUMC, UMCG, AUMC, WDO, DDF, UTWENTE. T20.3. aims to equip the RD-research-related multistakeholder community with the knowledge and skills needed for effective participation in ERDERA activities and beyond. Five subtasks are arranged to reflect targeted training blocks benefiting the main ERDERA Work streams to empower: (1) RD diagnostic research; (2) RD clinical research; (3) knowledge on research methodologies; (4) RD clinical trial methodologies and management; and (5) data research. A graded scalable approach will be implemented: beginner-level (mostly online trainings), followed by advanced-level, and/or nationally adapted online or face-to-face trainings for selected highly motivated learners who accomplished the beginner-level trainings and reached a certain level of expertise. Up to several hundreds of trainees per year will benefit from each beginner-level training and 20-30 selected trainees will be trained on an advanced course (for each of the 5 training tracks). The most effective training methodologies will be applied and measures for broad dissemination and inclusion will be invoked (incl. rotation of courses and fellowships for participants from underrepresented countries). T20.3. aligns with GO:1,2,3; SO:2,3,4,5; OO:2,3,4. T20.4. European Diploma on RD research (M1-M84) TL: UCSC, IBG; Contributors: c4c-S, ISS, SU, UNIROMA1, ISCIII, ACU, UCD, RSU, VULSK, SFU. T20.4. aims to establish a higher education program culminating in an academic diploma with credits, through an inter-university consortium of academic and non-academic partners with track record in RD training activities. T20.4. aligns with GO:1; SO:4,5; OO:3,4. ST20.4.1. Establish a cross ERDERA expert consortium and setup of training modules (M1-M12). Multilateral agreements will be made among academic and non-academic partners and connections with European university alliances, to ensure policy alignment and improve geographical representation as well as to ensure the smooth integration of young RD researchers into the EU R&I ecosystem. ST20.4.2. Pilot program launch (M13-24). This will run as a short-time pilot program (min 10 ECTS). ST20.4.3. Optimization and scale-up of the program (M25-36). The study plan will be refined and potentially upgraded to longer-term education programs to address emerging needs and provide recognized diplomas. ST20.4.4. Running the first systematic EU diploma curriculum (M30-60). Enrolling the first cohort of students, the curriculum will include a transnational mobility schedule and teaching modules from different partners. ST20.4.5. Optimization and updating of study plan (M52-M84). Continuing adaptation of the course. ST20.4.6. Pursue future sustainability of the established curricula (M6-84). Collaboration with stakeholders will be established to secure the program’s sustainability (incl. opportunities offered through Erasmus Mundus programme).

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Work package WP21 – Technology accelerator

Work Package Number WP21 Lead Beneficiary 28 - UC

Work Package Name Technology accelerator

Start Month 1 End Month 60

Objectives

To increase the success of in vivo ATMP therapies by improving state of the art technologies, processes, standards, and tools that are critical for efficacious and safe in vivo ATMPs. An open access portfolio of solutions and a network of expertise will be created in Europe which will decrease the number of therapies that fail in development and contribute to SO5, delivering new effective therapies for rare diseases approved in the EU and beyond (GO2). This will be achieved through cost-effective and harmonized AAV gene therapy; advancing RNAs for therapeutics; novel types of RNA nanoparticles (NP); comparative evaluation of gene editing technologies and delivery in relevant model systems for RDs; and development of platforms to characterize therapy response.

Description

T21.1. Streamlining chemistry manufacturing and controls in AAV vector production (M1-M60) TL: KU Leuven, UCB*, GNT. Contributors: VHIR, UC, UAB, INSERM_Target(Nantes), FHG, PEI, UKHD, REI*, Miltenyi*, FDB*, ROCHE*. Cost-effective and harmonized rAAV gene therapy vector development will be enabled through the development of a publicly available base manufacturing process to support next generation rAAV development and validation of new methodologies to ensure high quality and safety of newly standardized vectors. Fit-for-purpose analytical assays for determining CQA-related rAAV characteristics will be defined and validated. New technologies to develop more accurate analytical methods will be developed. T21.1. aligns with GO:2; SO:1,4,5; OO:1,2,5. T21.2. Advancing RNAs for therapeutics (M1-M60) TL: UC, Pfizer*, FHG. Contributors: INSERM_ART-ARNm, CHARITE, UEF, PEI, FSJD-CERCA, GNT, RJF*, AZ*, Miltenyi*, HIPRA*, LAB HIPRA*, FDB*. Approaches for RNA manufacturing will be optimized to advance the state of the art for mRNA therapeutics and ensure higher stability, translatability, cell-specificity, and lower immunogenicity. This will involve improving RNA design, production and purification, analytical characterization, and screening of optimal RNA molecules. A head-to-head comparison of RNA sequences will be conducted in in reporter cell lines to assess and compare the efficacy of selected RNA sequences (e.g., delivery of gene editing tools for stable gene therapy and gene replacement). Selected RNAs will be used in subtasks T21.3 and T21.4. T21.2. aligns with GO:2; SO:1,4,5; OO:1,2,5. T21.3. Advancing of novel types of mRNA nanoparticles (NP) including LNP, extracellular vesicles (EVs) and biohybrids (M1-M60) TL: INSERM_ART-ARNm, Pfizer*, CNRS. Contributors: FHG, PEI, UC, CHARITE, CIBER, VHIR, PLUS, UEF, Miltenyi*, AZ*, RJF*, HIPRA*, LAB HIPRA*, FDB*, ROCHE*. A better mechanistic understanding of extra and intracellular barriers to mRNA delivery via NP/EVs will allow a more rational design of novel mRNA formulations, including targeting tissue types along with improved and sustained release. In accordance, tools to implement formulation design, efficiency, production of standardized protocols will be developed. Different mRNA formulations will be generated with optimal mRNA structures produced in T21.2 and will be scaled-up for use for RNA-based gene editing, and evaluation of therapy response. T21.3. aligns with GO:2; SO:1,4; OO:1,2. T21.4. Gene editing approaches towards therapy of RDs (M1-M60) TL: OSR, Miltenyi*, IMAGINE. Contributors: UC, CIBER, GNT, UKLFR, BIU, UAB, FSJD-CERCA, FTELE, AZ*, Pfizer*, FDB*, ROCHE*. Comparison of state-of- the-art gene editing technologies and capitalize on viral and non-viral gene delivery modalities optimized in T21.1-3 to develop novel, more effective and safer therapeutic approaches for paradigmatic RDs affecting the blood, the central nervous system, the striated muscle, the retina, and the liver. We will complement these in vitro and in vivo gene therapy studies with an in-dept characterization of editing specificity and treatment-related toxicities, including immune responses and strategies to avoid them (T21.5), thus providing robust pre-clinical packages exploitable for the nomination of diseases suitable for clinical development. T21.4. aligns with GO:2; SO:1; OO:1. T21.5. Assessment of therapy response and immunogenicity (M1-M60) TL: CHARITE, Pfizer*; Contributors: CHI, Miltenyi*, ROCHE*, FSJD-CERCA, UAB, UC, OSR, INSERM_Target(Nantes), AZ*, UCB*. T21.5. aims to develop, qualify, and validate complex platforms used to characterize and monitor immune reactions directed to the developed ATMP. In vivo and in vitro tissue model systems will be used to verify the feasibility of test platforms developed. Mitigation strategies to prevent/overcome unwanted immune response will be developed. T21.5. aligns with GO:2; SO:1,4; OO:1,2.

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Work package WP22 – Public-Private Collaboration accelerator

Work Package Number WP22 Lead Beneficiary 29 - FFRD

Work Package Name Public-Private Collaboration accelerator

Start Month 1 End Month 84

Objectives

To create and operate an innovation marketplace, supporting and connecting promising RD projects with R&D experts, patient groups, and public and private investors, to enable further development. WP22 is dedicated to GO2, SO1. Specifically: (1) Engage third party investors, pharma industry and other funders of innovation; (2) Create workflows and legal documentation necessary to structurally identify, evaluate and select projects; (3) Support selected projects to become investment ready through business planning, coaching, nonclinical development, also in collaboration with other relevant synergistic initiatives; and (5) Actively match projects to external investment opportunities.

Description

T22.1. Setting up the marketplace (M1-M12) TL: ISCIII; Contributors: AFM, EATRIS, FFRD, SRUMC, EURORDIS. ISCIII will define the process workflows and create the legal documentation (working with RUMC and EATRIS) to facilitate sharing of information in the accelerator. Of particular importance are confidentiality and conflict of interest declarations, also with external collaborators. Experts from Expertise Service Hub, as well as external experts, e.g. from REMEDi4ALL, C-Path and C4C will be utilized as needed. FFRD will liaise with prospective investors and onboard them (using letters of intent to express interest) into the marketplace. AFM (with input from FFRD and EURORDIS) will define the project selection process, connecting with WP17 mentoring and consultancy creating workflows to be able to utilize their expertise, WP4 CRN and WP3 JTC to set up the outreach and project identification process within JTCs and the CRNs. T22.1. aligns with GO:2; SO:4,5; OO:3,5. T22.2. Acceleration readiness (M1-M72) TL: AFM; Contributors: EATRIS, FFRD, EURORDIS. During JTC calls, information sessions will be integrated in the call publication strategy, as well as monitoring process of JTC projects to ensure all applicants and Principal investigators are fully aware of the requirements for further therapeutic development and the opportunities provided by the Acceleration Hub, Expertise Hub, and infrastructures. Annual meetings will be organized in liaison with JTC and monitoring WPs to identify JTC projects with potential for development into new intervention. Identified projects will then be proposed to enter profiling and development process in the Acceleration Hub (T22.3). Close collaboration with all WP22 partners will be sought for the selection of projects, with structural involvement of patient representatives in the selection process. During JTC calls: integrate information sessions in the monitoring process of JTC projects (and projects from ERA4HEALTH) to ensure investigators are fully aware of the requirements for further development and the opportunities provided by the Acceleration Hub, Expertise Hub, and infrastructures. T22.2. aligns with GO:2; SO:1,4; OO:1,3,5. T22.3. Asset profiling and development (M12-M80) TL: EATRIS; Contributors: AFM, FFRD, EURORDIS, ISCIII. Selected projects will receive support defining an IPR, exploitation and patient access strategy, designing a critical path to proof of concept including stage gates and milestones, seeking funding for technical development. Expertise from CRNs, Consulting and Mentoring WPs will be accessed, as well as other initiatives such as C-Path, C4C, REMEDi4ALL, EU Lead Factory. It is expected that 5-10 projects per year can be supported, with first projects receiving support from M18. T22.3. aligns with GO:2; SO:1,4; OO:1,3, 5. T22.4. Matchmaking and marketing (M18-M84) TL: FFRD; Contributors: AFM, EATRIS. Regular interactions with pharma and other potential funders will be scheduled to pitch projects and facilitate discussions and negotiations. A range of different formats will be used including webinars of pitches (presentations + Q/A sessions), networking events, speed dating events between researchers and funders, and set-up of F2F meetings to discuss potential collaboration. Coaching of researchers, creation of pitch decks and support in pitching to third party funders, based on the creation of a grid including key elements such as identified and validated target, PI, mechanism of action and competitive landscape analysis. Marketing of the accelerator program and the projects in the portfolio will be conducted on an ongoing basis, with direct outreach to investors. T22.4. aligns with GO:2; SO:1, 4,5; OO:1,3,5.

Work package WP23 – NMGs promotion and national alignment

Work Package Number WP23 Lead Beneficiary 1 - INSERM

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Work Package Name NMGs promotion and national alignment

Start Month 1 End Month 84

Objectives

The main objective of this WP is to ensure the alignment of European and national RD research policies and activities by supporting the deployment, the operations, and the synergies of and between the National Mirror Groups (NMGs) in all participating countries. An NMG is a group created – or designated - in each country, benefiting from the ERDERA to bring together the expertise and knowledge of the RD community of a specific country. The objectives of a NMG are to identify national needs that should be discussed and addressed, if possible, within the framework of the ERDERA activities; to coordinate national stakeholders and promote national alignment with the European RD research strategy, to revitalize the national RD plans/strategies and to collect and share lessons learned and good practices. NMGs will also be solicited to help with liaising with the national stakeholders, including but not limited to funders, to manage the lessons learned from the cancer field and organise, when relevant, joint activities (like common or complementary funding opportunities).

Description

T23.1. Fostering creation of NMGs (M1-M84) TL: INSERM_IT-GGB; Contributors: ISCIII, UNEW*, CCRI GmbH, UTARTU, IBG, CCUH, INSERM_U974, MUH, TIF, MICYRN*, VULSK, ZonMw, INSA, HRB, MoH SR, RDG, JSI, UO. T23.1. aligns with GO:1-3, SO:5, OO:3 and will consist of two subtasks. ST23.1.1. Development of NMG models and guidelines. In the first 4 months of the project, several models of NMGs will be developed based on the study of the national groups created during EJP RD. These different models, which will differ in several aspects, will be used on the NMGs development process on the Task 23.1.2. ST23.1.2. Developing & maintaining NMGs through a national multi-stakeholder engagement. The NMGs will be set up and supported to organize coordinated interaction between the ERDERA and national and regional stakeholders. To provide successful action of NMG, financial support will be provided to each NMG. T23.2. Deployment and operation of NMGs (M1-M84) TL: UNEW*; Contributor: INSERM_IT-GGB, ISCIII, CCRI GmbH, UTARTU, IBG, CCUH, INSERM_U974, MUH, TIF, MICYRN*, VULSK, ZonMw, INSA, HRB, MoH SR, RDG, JSI, UO. T23.2. aligns with GO:1-3; SO:4,5; OO:3,4 and will consist of four subtasks. ST23.2.1. NMG responsibilities. Each NMG should fulfil their responsibilities to ensure optimal collaboration with ERDERA. Fulfilling responsibilities will help advocate national interests and provide bidirectional information flow from and to partnership. During the first 3 years of the project, WP leads will support the deployment and operation of new and existing NMGs. This will include supporting NMGs to develop a three-year plan for their NMG. In the following four years of the project, NMG leads will continue to maintain and develop their NMG with support from WP leads. ST23.2.2. Country-Specific multidirectional Engagement. The WP leads will ensure regular communication with NMG teams to support them in aligning their national activities with those delivered under the ERDERA. During the first three years of the project, communication channels will be established WP leads and NMGs, as well between NMGs to support multi-directional engagement. WP leads will work with other WPs to communicate the sharing of tools, resources and knowledge developed by other WP to the NMGS. ST23.2.3. Country Data Collection. A number (minimum one per year) of questionnaires will be designed to collect RD data on national activities and international collaborations. Outputs will be dedicated to annual reports and guideline development. This will continue throughout the project. ST23.2.4. Trainings activities through the ‘Train the trainer model’. A series of training activities for national stakeholders is planned. This subtask will collaborate with ERDERA Workstream ‘Education & training activities. Each NMG will identify a national training provider and will be trained on the topic preselected by NMGs. Once trained, they will then be able to share what they have learned at national level to national stakeholders. T23.3. Animation of NMGs synergies (M6-M84) TL: UNEW*, CCUH; Contributors: INSERM_IT-GGB, UTARTU, CCRI GmbH, ISCIII, VULSK, ZonMw, INSA, MICYRN*, INSERM_U974, MUH, TIF, IBG, HRB, MoH SR, RDG, JSI, UO. T23.3. aligns with GO:1-3; SO:5; OO:3,5 and will consist of three subtasks. ST23.3.1. Organization of annual meeting of the National Alignment Board (NAB), foreseen back-to-back with the annual General Assembly. NAB will be composed of Representatives from each NMG and Representatives of the EC Directorates. During these meetings, NMG leads will share progress on the creation and operations of NMGs, and steps taken to align national and ERDERA level activity. These meetings will also serve as a basis for writing of best practice guidelines on NMG creation and promotion in first three years of partnership. During the next four years, the recommendations for sustainability of NMGs will be developed. ST23.3.2. Fostering interaction between NMGs, to enhance cross-country collaboration. Three workshops will be

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organized during first three years, followed by four in the last four years, topics of which will be identified during International NAB meeting, but foreseeably include subjects such as creation/adaptation/federation of national registries for RD, linking national diagnostics structures with ERDERA resources, optimising clinical trial planning and delivery, national drug reimbursement programs, NBS etc. During this subtask intensive collaboration and communication in between various sets of NMGs on monthly or even weekly basis will be coordinated and facilitated using some platform for example SharePoint. ST23.3.3. Sharing achievements of NMGs. During the first three years of partnership using the outputs of T23.2.3, an overview report will be developed every year, arranged by topic (e.g. showcasing the situation and trends around live national plans for RD, national registries for RD, etc). During the following four years four more reports will be developed and two peer-reviewed publications in open access journals will be published during project with a purpose to reach out more health care specialists, for example GPs, clinical specialists, and scientists from other fields.

Work package WP24 – Fostering engagement of underrepresented countries in ERDERA

Work Package Number WP24 Lead Beneficiary 32 - AICIB

Work Package Name Fostering engagement of underrepresented countries in ERDERA

Start Month 1 End Month 84

Objectives

(1) Address specific needs and expectations of underrepresented countries (UCs) in RD; (2) Foster collaboration between UCs and Research & Innovation (R&I) high performing countries at European and Global level; (3) Boost inclusion of UCs in ERDERA funded programmes; (4) Promote transversal, coordinated efforts in mapping capabilities and assets, capacity development, advocacy, awareness-raising, and leveraging the knowledge and experiences of UCs with R&I high performing countries; (5) Create a more inclusive and impactful R&I ecosystem in RD; and (6) De-emphasize the inequalities between R&I high and low performing countries and unlock the potential present within UCs and its added value to the ERDERA.

Description

T24.1. Promoting capacity development actions (M1-M84) TL: VULSK, REUH; Contributors: EURORDIS, IBG, CVBF, GERAD, UCD. T24.1. aims to (1) Develop a mapping exercise to identify specific needs and expectations, perceived by UCs as limiting their full integration in RD R&I, aligned with ERDERA workstreams; (2) Assess the conditions for their full integration across all ERDERA activities (JTCs, CRNs, Acceleration Hub, Data Services Hub, Support Services), including potential barriers to participation in CTs; (3) Implement knowledge exchange and mutual learning exercises between UCs (research actors, patient organisations) for comparative assessment and promotion of activities, at National level, to foster participation in ERDERA. Complementation of efforts for the coordination of the National Mirror Group representatives; (4) Benchmark high performing countries in R&I to integrate learnings and expertise; and (5) Develop focused guidelines for potential actions at the National level (e.g., national calls to support specific lines of research aligned with CRNs objectives). T24.1. aligns with GO:3, SO:4-5. T24.2. Undertaking advocacy and awareness efforts to UCs added value (M1-M84) TL: FFRD, IBG, CAU; Contributors: EURORDIS, RSU, RDB. T24.2. aims to (1) Detect latent and unknown RDrelated assets in UCs countries, and additional patient populations, to convert it into added value for the ERDERA; (2) Implement measures to identify RDs with particular (geographic) incidence, distinctive critical mass, and infrastructural resources; (3) Establish synergies with JARDIN, EURORDIS, HNN3.0 (Health NCPs), NFP4EU (Health NFPs), and NCP_WIDERA.NET, to get further support for the identification of UCs added value; (4) Collaborate with national entities, National Mirror Groups and scientific societies for the assessment of distinctive features of UCs; (5) Pursuance of advocacy and awareness actions (to be undertaken with the coordination of ERDERA and of the workstreams) to promote their inclusion in the ERDERA activities; and (6) Alignment of academic, clinical, and patient organisations, at the national level, and promotion of the geographical regions (North Africa, Eastern Europe, and Southern Europe countries). T24.2. aligns with GO:3; SO:3,5; OO:3. T24.3. Support actions to improve UC participation in all ERDERA activities (M1-M84) TL: AICIB, IMGGE; Contributors: TUBITAK, RIF, ZonMw, VULSK, EURORDIS, CVBF, HRB. T24.3. aims to (1) Identify actions to increase the participation of UCs RD community in ERDERA, and of the funds available for both UCs and ERDERA partners; (2) Develop guidelines for promoting the inclusion of UCs in ERDERA funded actions (e.g., JTCs, Networking Calls, and others) in coordination with the WS Leads; (3) Propose new measures to increase UCs participation using

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the outputs of the mapping of needs, lessons learnt from EJPRD, EURORDIS surveys, and other relevant initiatives; (4) Establish baseline figures, and proposal of targets for levels of participation in these activities; (5) Analysis and adaptation of measures and guidelines used in the Widening programmes (e.g., Twinning, Excellence Hubs, Hop-on), and their application to ERDERA activities (when appropriate); and (5) Exploitation of access to additional financial support (e.g. EU Widening programmes, structural funds) to increase UCs participation in ERDERA and leverage available funds, and propose measures to be implemented (also to be highlighted to the National Mirror Groups). T24.3. aligns with GO:3; SO:5.

Work package WP25 – ERDERA Global Collaborations

Work Package Number WP25 Lead Beneficiary 7 - ZonMw

Work Package Name ERDERA Global Collaborations

Start Month 1 End Month 84

Objectives

To establish international partnerships that will benefit ERDERA and promote sharing of resources, expertise, and research findings which will address issues of resource fragmentation, harness collective knowledge and expertise, and facilitate exchange of information and data, ultimately leading to improved diagnosis and the development of new and innovative treatments for RDs.

Description

T25.1. Strategic Alliances (M1-M84) TL: INSERM_IT-GGB, SU, ZonMw; Contributors: RCC*, CIHR*, RDI, EURORDIS, FFRD, ISCIII, AICIB, CAU, AUMC, ASU-FC, HSJD. In collaboration with WP1 and other ERDERA WPs, T25.1. aims to foster effective international collaboration and maximize outreach of the results of ERDERA through strategic alliances. T25.1. will be subdivided in four subtasks. INSERM_IT-GGB and Sorbonne University will start in parallel the mapping of potential international strategic alliances encompassing universities, programs, projects (including EJP RD and ERDERA funded projects), and initiatives that have not yet been identified in the SRIA/EJP RD/ concept Partnership. Recommendations from IRDiRC members will be sought to augment this process. Following this initial mapping, a dedicated Working Group composed of contributors, and ERDERA partners will further develop the collated list of identified international strategic partners and will facilitate partner matchmaking by identifying the specific topics and opportunities for collaboration across different ERDERA WPs. Throughout the duration of ERDERA, regular virtual meetings will be organised to discuss progress, share updates and outcomes, and address any challenges or opportunities that may arise during the existence of ERDERA. The identification of additional strategic partners and opportunities will be continuously performed and in collaboration with WP1, a sustainability plan of the established partnerships will be developed. T25.1. aligns with GO:1-3; SO:4,5; OO:2,3,5. T25.2 Support to IRDiRC Scientific Secretariat (M1-M84) TL: FFRD; Contributors: TEAMIT. FFRD will secure the IRDiRC Scientific Secretariat (SciSec), which plays a vital role in the day-to-day operations and management of the IRDiRC Consortium. As key administrative and scientific support unit, SciSec ensures the fulfilment of IRDiRC's needs and requests, assisting various bodies such as the Consortium Assembly, Constituent Committees, Scientific Committees, Operating Committee, Task Forces, and Working Groups, optimizing their complementary activities. SciSec will continue to organize and coordinate Consortium Assembly in-person and online meetings, including joint meetings with Scientific Committees, Task Force workshops, and IRDiRC Congress. SciSec will coordinate the Consortium’s communication activities and delivers comprehensive meeting reports that capture the outcomes and discussions of these events and activities, including participation in the writing of white paper/recommendations of IRDiRC Task Forces. To ensure liaison between ERDERA and IRDiRC stakeholders, SciSec members will present IRDiRC updates to ERDERA Executive Committee and organize dedicated IRDiRC sessions during ERDERA General Assembly meetings. In addition, ERDERA beneficiaries' involvement in IRDiRC activities will be promoted, and the IRDiRC Chair and Vice-Chair will be invited to join the ERDERA MAB. T25.2. aligns with GO:1-3; SO:4-5; OO:3,5. T25.3. Promote the International Dimension of the CRN of ERDERA by building global networks among CRNs and patient organizations (M1-M84) TL: RDI, INSERM_IT-GGB; Contributors: AICIB, AUMC, c4c-S, UKHD, UT, CHEO- RI*, RCC*. T25.3. aims to perform networking, matchmaking, and identifying similar initiatives as the CRNs, that exist in other geographies. In collaboration with the Workstream on CRN, international CRN conferences will be organised to facilitate networking between various international CRNs, jointly organized with IRDiRC, RDI, and ERDERA. The

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conferences will be organised on biannual basis and may be organised in conjunction with the IRDiRC-ERDERA Congress. T25.3. aligns with GO:2,3; SO:3-5; OO:2,3,5. T25.4 Alignment with the Research strategies of the European Reference Networks (M1-M84) TL: ASU-FC, HSJD; Contributor: IOR. T25.4 aims to align research strategies within the ERNs and with ERDERA. A platform "ERNs Living Lab" will be created to foster new cross-fertilising communication and avenues across ERNs and stakeholders to accelerate patient-relevant advancements and discuss the alignment of research strategies and implementation of the acquired knowledge in activities of ERDERA and beyond. T25.4. aligns with GO:3; SO:1,4,5; OO:3-5.

Work package WP26 – Ethics requirements

Work Package Number WP26 Lead Beneficiary 1 - INSERM

Work Package Name Ethics requirements

Start Month 1 End Month 84

Objectives

The objective is to ensure compliance with the 'ethics requirements' set out in this work package.

Description

This work package sets out the 'ethics requirements' that the project must comply with.

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STAFF EFFORT

Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

1 - INSERM 288.00 125.00 12.00 6.00 828.00

1.1 - CAD 6.00 22.00 72.00

2 - TEAMIT 36.00 72.00 126.00

3 - DLR 3.00 20.00 8.00 31.00

4 - ANR 3.00 19.00 1.00 23.00

5 - FTELE 2.00 17.00 66.00

6 - LMT 2.00 4.00 6.00

7 - ZonMw 3.00 6.00 10.00 44.00

8 - UT 78.00 66.00 33.00 57.00 30.00 300.00

9 - UTARTU 42.00 56.00

10 - CNAG 72.00 105.00 6.00 278.00

11 - SRUMC 6.00 153.00 121.00 34.00 356.00

12 - TUM 6.00 76.00 84.00

13 - UKHD 152.00 76.00 279.00

14 - C-PATH 80.00 107.00

15 - VHIR 27.00 76.00 134.00

16 - LUMC 6.00 27.00 118.00

17 - UTWENTE 38.00

18 - UPM 27.00 28.00 149.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

19 - UMCG 29.00 15.00 166.00

20 - AUMC 3.00 67.00

21 - UM 36.00

22 - EATRIS 86.00

23 - FGB 36.00 2.00 93.00

24 - APHP 6.00 89.00 42.00 169.00

25 - UHASSELT 35.00

26 - EURORDIS 12.00 3.00 1.00 8.00 124.00

27 - VULSK 3.00 6.00 18.00 100.00

28 - UC 57.00

29 - FFRD 5.00 2.00 146.00

30 - AFM 8.00 44.00

31 - CCUH 30.00

32 - AICIB 3.00 23.00

33 - IMGGE 15.00

34 - RDI 5.00 2.00 19.00

35 - ACU 2.00

36 - AIT 23.00

37 - AMU 12.00 26.00

38 - AOU Meyer IRCCS 9.00 9.00

39 - ASU-FC 35.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

40 - BIU 3.00

42 - CCRI GmbH 6.00 18.00 36.00

43 - CENTOGENE 7.00 11.00 18.00

44 - CHARITE 61.00

45 - CHI 20.00

46 - CHU Dijon 9.00 18.00 27.00

47 - CE 54.00 54.00

48 - REGIONH 6.00 12.00 18.00

49 - CSO-MOH 18.00 2.00 1.00 21.00

50 - CVBF 18.00 2.00 31.00

51 - DDF 26.00 44.00

53 - Erasmus MC 9.00 15.00 44.00 68.00

54 - F.R.S.-FNRS 2.00 2.00

55 - FHG 62.00

56 - FRRB 2.00 2.00

57 - ECRIN 5.00

58 - FWF 2.00 2.00

59 - IFD 2.00 2.00

60 - FSJD-CERCA 23.00

61 - FCT 2.00 2.00

62.1 - AZV 2.00 2.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

63 - GNT 102.00

64 - GERAD 5.00

65 - RS 3.00 1.00 4.00

66 - GUF 11.00

67 - HRB 2.00 18.00

68 - İBG 6.00 7.00 6.00 56.00

69 - Rannis 2.00 2.00

70 - IMAGINE 12.00 49.00

71 - IGC PAN 6.00 6.00

72 - ISCIII 17.00 36.00 76.00

73 - INSA 12.00

74 - IOR 3.00

75 - ISS 11.00

76 - IT-MOH 2.00 2.00

77 - KU Leuven 6.00 10.00 53.00

78 - LZP 2.00 2.00

79 - MRT 27.00

80 - MUS 6.00 6.00

81 - MUH 6.00 18.00 36.00

82 - MUR 2.00 2.00

83 - NCBR 2.00 2.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

84 - NKUA 6.00 6.00

85 - MoH SR 12.00

86 - RDG 8.00

87 - REGIONSYD 18.00 18.00

88 - OPBG 9.00 12.00 21.00

89 - OSR 43.00

90 - JSI 8.00

91 - RCN 2.00 2.00

92 - FWO 2.00 2.00

93 - REUH 9.00 21.00

94 - RIF 2.00 6.00

95 - RCSI 9.00 9.00

96 - RSU 5.00

97 - Sciensano 30.00 18.00 48.00

98 - SPW 2.00 2.00

99 - SFU 26.00

100 - ETAg 2.00 2.00

101 - SAS 2.00 2.00

102 - Sonio 27.00

103 - MOSAE 2.00 2.00

104 - SRC 2.00 2.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

105 - TEDDY 6.00

106 - TEKKARE 66.00 66.00

107 - TIF 12.00

108 - TUBITAK 2.00 6.00

109 - RT (TuscReg) 3.00 3.00

110 - UAB 20.00

111 - UCD 7.00

112 - UCSC 16.00

113 - UEF 27.00

114 - UKA 22.00

115 - UKLFR 30.00 38.00

116 - UNICAMPANIA 9.00 9.00

117 - UoC 6.00 6.00

118 - UGENT 6.00 18.00 24.00

119 - UKCL 9.00 9.00

120 - WDO 5.00 1.00 3.00 1.00 2.00 21.00

121 - UU 18.00 62.00

122 - c4c-S 10.00

123 - CAU 8.00

124 - CNRS 20.00

125 - CIBER 9.00 27.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

126 - UEFISCDI 2.00 2.00

127 - UNIROMA1 9.00

128 - UANTWERPEN 6.00 6.00

129 - BNSF 2.00 2.00

130 - Aalborg UH 6.00 6.00

131 - RDB 3.00

132 - BBMRI-ERIC 8.00

133 - AarhusUH 9.00 9.00

134 - IABS-EU 12.00

135 - HRCI 5.00 5.00

136.1 - KUM 33.00 8.00 41.00

137 - MSW 38.00 38.00

138 - FNR 2.00 2.00

139 - NKFIH 2.00 2.00

140 - PEI 36.00

141 - PLUS 37.00

142 - SU 3.00

143 - UNISI 9.00 9.00

144 - Vinnova 2.00 2.00

145 - CRG-CERCA 15.00 18.00

146 - GMS-RS 3.00 1.00 4.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP1 WP2 WP3 WP4 WP5 WP6 WP7 WP8 WP9 WP10 Total Person-Months

147 - VIB 3.00 3.00

148 - IPG 6.00 6.00

149 - UO 6.00 6.00

150 - HSJD 7.00

Total Person-Months 607.00 72.00 132.00 28.00 22.00 614.00 490.00 347.00 633.00 336.00 6355.00

Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

1 - INSERM 84.00 40.00 86.00 23.00 828.00

1.1 - CAD 44.00 72.00

2 - TEAMIT 126.00

3 - DLR 31.00

4 - ANR 23.00

5 - FTELE 36.00 66.00

6 - LMT 6.00

7 - ZonMw 44.00

8 - UT 30.00 6.00 300.00

9 - UTARTU 2.00 56.00

10 - CNAG 5.00 41.00 27.00 22.00 278.00

11 - SRUMC 16.00 15.00 8.00 356.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

12 - TUM 2.00 84.00

13 - UKHD 18.00 30.00 279.00

14 - C-PATH 27.00 107.00

15 - VHIR 18.00 134.00

16 - LUMC 22.00 31.00 25.00 5.00 2.00 118.00

17 - UTWENTE 26.00 10.00 2.00 38.00

18 - UPM 32.00 37.00 25.00 149.00

19 - UMCG 32.00 10.00 78.00 2.00 166.00

20 - AUMC 18.00 15.00 28.00 2.00 67.00

21 - UM 36.00 36.00

22 - EATRIS 15.00 26.00 9.00 86.00

23 - FGB 5.00 50.00 93.00

24 - APHP 32.00 169.00

25 - UHASSELT 32.00 3.00 35.00

26 - EURORDIS 18.00 64.00 124.00

27 - VULSK 46.00 100.00

28 - UC 57.00

29 - FFRD 6.00 146.00

30 - AFM 44.00

31 - CCUH 30.00

32 - AICIB 23.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

33 - IMGGE 15.00

34 - RDI 19.00

35 - ACU 2.00 2.00

36 - AIT 16.00 7.00 23.00

37 - AMU 14.00 26.00

38 - AOU Meyer IRCCS 9.00

39 - ASU-FC 25.00 35.00

40 - BIU 3.00

42 - CCRI GmbH 36.00

43 - CENTOGENE 18.00

44 - CHARITE 61.00

45 - CHI 20.00

46 - CHU Dijon 27.00

47 - CE 54.00

48 - REGIONH 18.00

49 - CSO-MOH 21.00

50 - CVBF 6.00 31.00

51 - DDF 12.00 6.00 44.00

53 - Erasmus MC 68.00

54 - F.R.S.-FNRS 2.00

55 - FHG 62.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

56 - FRRB 2.00

57 - ECRIN 5.00 5.00

58 - FWF 2.00

59 - IFD 2.00

60 - FSJD-CERCA 23.00

61 - FCT 2.00

62.1 - AZV 2.00

63 - GNT 48.00 102.00

64 - GERAD 5.00

65 - RS 4.00

66 - GUF 11.00 11.00

67 - HRB 18.00

68 - İBG 13.00 56.00

69 - Rannis 2.00

70 - IMAGINE 14.00 3.00 49.00

71 - IGC PAN 6.00

72 - ISCIII 2.00 76.00

73 - INSA 12.00

74 - IOR 3.00

75 - ISS 2.00 9.00 11.00

76 - IT-MOH 2.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

77 - KU Leuven 53.00

78 - LZP 2.00

79 - MRT 27.00 27.00

80 - MUS 6.00

81 - MUH 36.00

82 - MUR 2.00

83 - NCBR 2.00

84 - NKUA 6.00

85 - MoH SR 12.00

86 - RDG 8.00

87 - REGIONSYD 18.00

88 - OPBG 21.00

89 - OSR 43.00

90 - JSI 8.00

91 - RCN 2.00

92 - FWO 2.00

93 - REUH 21.00

94 - RIF 6.00

95 - RCSI 9.00

96 - RSU 2.00 5.00

97 - Sciensano 48.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

98 - SPW 2.00

99 - SFU 5.00 21.00 26.00

100 - ETAg 2.00

101 - SAS 2.00

102 - Sonio 27.00 27.00

103 - MOSAE 2.00

104 - SRC 2.00

105 - TEDDY 6.00 6.00

106 - TEKKARE 66.00

107 - TIF 12.00

108 - TUBITAK 6.00

109 - RT (TuscReg) 3.00

110 - UAB 20.00

111 - UCD 2.00 7.00

112 - UCSC 16.00 16.00

113 - UEF 27.00

114 - UKA 22.00 22.00

115 - UKLFR 38.00

116 - UNICAMPANIA 9.00

117 - UoC 6.00

118 - UGENT 24.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

119 - UKCL 9.00

120 - WDO 9.00 21.00

121 - UU 22.00 22.00 62.00

122 - c4c-S 10.00 10.00

123 - CAU 8.00

124 - CNRS 20.00

125 - CIBER 27.00

126 - UEFISCDI 2.00

127 - UNIROMA1 9.00 9.00

128 - UANTWERPEN 6.00

129 - BNSF 2.00

130 - Aalborg UH 6.00

131 - RDB 3.00

132 - BBMRI-ERIC 8.00 8.00

133 - AarhusUH 9.00

134 - IABS-EU 12.00 12.00

135 - HRCI 5.00

136.1 - KUM 41.00

137 - MSW 38.00

138 - FNR 2.00

139 - NKFIH 2.00

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP11 WP12 WP13 WP14 WP15 WP16 WP17 WP18 WP19 WP20 Total Person-Months

140 - PEI 36.00

141 - PLUS 22.00 37.00

142 - SU 2.00 3.00

143 - UNISI 9.00

144 - Vinnova 2.00

145 - CRG-CERCA 3.00 18.00

146 - GMS-RS 4.00

147 - VIB 3.00

148 - IPG 6.00

149 - UO 6.00

150 - HSJD 7.00

Total Person-Months 210.00 82.00 230.00 168.00 224.00 250.00 38.00 88.00 158.00 318.00 6355.00

Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

1 - INSERM 12.00 152.00 828.00

1.1 - CAD 72.00

2 - TEAMIT 18.00 126.00

3 - DLR 31.00

4 - ANR 23.00

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Staff effort per participant

Grant Preparation (Work packages - Effort screen) — Enter the info.

Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

5 - FTELE 11.00 66.00

6 - LMT 6.00

7 - ZonMw 12.00 4.00 9.00 44.00

8 - UT 300.00

9 - UTARTU 12.00 56.00

10 - CNAG 278.00

11 - SRUMC 3.00 356.00

12 - TUM 84.00

13 - UKHD 3.00 279.00

14 - C-PATH 107.00

15 - VHIR 13.00 134.00

16 - LUMC 118.00

17 - UTWENTE 38.00

18 - UPM 149.00

19 - UMCG 166.00

20 - AUMC 1.00 67.00

21 - UM 36.00

22 - EATRIS 36.00 86.00

23 - FGB 93.00

24 - APHP 169.00

25 - UHASSELT 35.00

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Staff effort per participant

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Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

26 - EURORDIS 6.00 11.00 1.00 124.00

27 - VULSK 12.00 15.00 100.00

28 - UC 57.00 57.00

29 - FFRD 36.00 7.00 90.00 146.00

30 - AFM 36.00 44.00

31 - CCUH 30.00 30.00

32 - AICIB 19.00 1.00 23.00

33 - IMGGE 15.00 15.00

34 - RDI 12.00 19.00

35 - ACU 2.00

36 - AIT 23.00

37 - AMU 26.00

38 - AOU Meyer IRCCS 9.00

39 - ASU-FC 10.00 35.00

40 - BIU 3.00 3.00

42 - CCRI GmbH 12.00 36.00

43 - CENTOGENE 18.00

44 - CHARITE 61.00 61.00

45 - CHI 20.00 20.00

46 - CHU Dijon 27.00

47 - CE 54.00

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Staff effort per participant

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Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

48 - REGIONH 18.00

49 - CSO-MOH 21.00

50 - CVBF 5.00 31.00

51 - DDF 44.00

53 - Erasmus MC 68.00

54 - F.R.S.-FNRS 2.00

55 - FHG 62.00 62.00

56 - FRRB 2.00

57 - ECRIN 5.00

58 - FWF 2.00

59 - IFD 2.00

60 - FSJD-CERCA 23.00 23.00

61 - FCT 2.00

62.1 - AZV 2.00

63 - GNT 54.00 102.00

64 - GERAD 5.00 5.00

65 - RS 4.00

66 - GUF 11.00

67 - HRB 12.00 4.00 18.00

68 - İBG 12.00 12.00 56.00

69 - Rannis 2.00

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Staff effort per participant

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Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

70 - IMAGINE 20.00 49.00

71 - IGC PAN 6.00

72 - ISCIII 8.00 12.00 1.00 76.00

73 - INSA 12.00 12.00

74 - IOR 3.00 3.00

75 - ISS 11.00

76 - IT-MOH 2.00

77 - KU Leuven 37.00 53.00

78 - LZP 2.00

79 - MRT 27.00

80 - MUS 6.00

81 - MUH 12.00 36.00

82 - MUR 2.00

83 - NCBR 2.00

84 - NKUA 6.00

85 - MoH SR 12.00 12.00

86 - RDG 8.00 8.00

87 - REGIONSYD 18.00

88 - OPBG 21.00

89 - OSR 43.00 43.00

90 - JSI 8.00 8.00

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Staff effort per participant

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Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

91 - RCN 2.00

92 - FWO 2.00

93 - REUH 12.00 21.00

94 - RIF 4.00 6.00

95 - RCSI 9.00

96 - RSU 3.00 5.00

97 - Sciensano 48.00

98 - SPW 2.00

99 - SFU 26.00

100 - ETAg 2.00

101 - SAS 2.00

102 - Sonio 27.00

103 - MOSAE 2.00

104 - SRC 2.00

105 - TEDDY 6.00

106 - TEKKARE 66.00

107 - TIF 12.00 12.00

108 - TUBITAK 4.00 6.00

109 - RT (TuscReg) 3.00

110 - UAB 20.00 20.00

111 - UCD 5.00 7.00

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Staff effort per participant

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Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

112 - UCSC 16.00

113 - UEF 27.00 27.00

114 - UKA 22.00

115 - UKLFR 8.00 38.00

116 - UNICAMPANIA 9.00

117 - UoC 6.00

118 - UGENT 24.00

119 - UKCL 9.00

120 - WDO 21.00

121 - UU 62.00

122 - c4c-S 10.00

123 - CAU 7.00 1.00 8.00

124 - CNRS 20.00 20.00

125 - CIBER 18.00 27.00

126 - UEFISCDI 2.00

127 - UNIROMA1 9.00

128 - UANTWERPEN 6.00

129 - BNSF 2.00

130 - Aalborg UH 6.00

131 - RDB 3.00 3.00

132 - BBMRI-ERIC 8.00

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Staff effort per participant

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Participant WP21 WP22 WP23 WP24 WP25 WP26 Total Person-Months

133 - AarhusUH 9.00

134 - IABS-EU 12.00

135 - HRCI 5.00

136.1 - KUM 41.00

137 - MSW 38.00

138 - FNR 2.00

139 - NKFIH 2.00

140 - PEI 36.00 36.00

141 - PLUS 15.00 37.00

142 - SU 1.00 3.00

143 - UNISI 9.00

144 - Vinnova 2.00

145 - CRG-CERCA 18.00

146 - GMS-RS 4.00

147 - VIB 3.00

148 - IPG 6.00

149 - UO 6.00

150 - HSJD 7.00 7.00

Total Person-Months 563.00 125.00 330.00 135.00 155.00 0.00 6355.00

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LIST OF DELIVERABLES

Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D1.1 Data Management Plan WP1 1 - INSERM R — Document, report SEN - Sensitive 6

D1.2 Annual Work Plan Y2 WP1 1 - INSERM R — Document, report SEN - Sensitive 9

D1.3 Annual Work Plan Y3 WP1 1 - INSERM R — Document, report SEN - Sensitive 21

D1.4 Annual Work Plan Y4 WP1 1 - INSERM R — Document, report SEN - Sensitive 33

D1.5 Annual Work Plan Y5 WP1 1 - INSERM R — Document, report SEN - Sensitive 45

D1.6 Annual Work Plan Y6 WP1 1 - INSERM R — Document, report SEN - Sensitive 57

D1.7 Annual Work Plan Y7 WP1 1 - INSERM R — Document, report SEN - Sensitive 69

D1.8 Periodic Progress Report 1 WP1 1 - INSERM R — Document, report SEN - Sensitive 26

D1.9 Periodic Progress Report 2 WP1 1 - INSERM R — Document, report SEN - Sensitive 44

D1.10 Periodic Progress Report 3 WP1 1 - INSERM R — Document, report SEN - Sensitive 62

D1.11 Periodic Progress Report 4 WP1 1 - INSERM R — Document, report SEN - Sensitive 74

D1.12 Periodic Progress Report 5 WP1 1 - INSERM R — Document, report SEN - Sensitive 84

D1.13 RDP SRIA Update WP1 1 - INSERM R — Document, report PU - Public 36

D1.14 Report on cumulative expenditure incurred WP1 1 - INSERM R — Document, report SEN - Sensitive 12

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D2.1 Project website WP2 2 - TEAMIT DEC —Websites, patent filings, videos, etc

PU - Public 3

D2.2 Communication and dissemination strategy WP2 2 - TEAMIT R — Document, report SEN - Sensitive 6

D3.1 Joint Transnational Call documents 1 WP3 3 - DLR R — Document, report SEN - Sensitive 3

D3.2 Joint Transnational Call documents 2 WP3 3 - DLR R — Document, report SEN - Sensitive 15

D3.3 Joint Transnational Call documents 3 WP3 3 - DLR R — Document, report SEN - Sensitive 27

D3.4 Joint Transnational Call documents 4 WP3 3 - DLR R — Document, report SEN - Sensitive 39

D3.5 Joint Transnational Call documents 5 WP3 3 - DLR R — Document, report SEN - Sensitive 51

D3.6 Joint Transnational Call documents 6 WP3 3 - DLR R — Document, report SEN - Sensitive 63

D3.7 Joint Transnational Call documents 7 WP3 3 - DLR R — Document, report SEN - Sensitive 75

D3.8 Evaluation Report of the JTCs 1 WP3 3 - DLR R — Document, report SEN - Sensitive 15

D3.9 Evaluation Report of the JTCs 2 WP3 3 - DLR R — Document, report SEN - Sensitive 27

D3.10 Evaluation Report of the JTCs 3 WP3 3 - DLR R — Document, report SEN - Sensitive 39

D3.11 Evaluation Report of the JTCs 4 WP3 3 - DLR R — Document, report SEN - Sensitive 51

D3.12 Evaluation Report of the JTCs 5 WP3 3 - DLR R — Document, report SEN - Sensitive 63

D3.13 Evaluation Report of the JTCs 6 WP3 3 - DLR R — Document, report SEN - Sensitive 75

D3.14 Evaluation Report of the JTCs 7 WP3 3 - DLR R — Document, report SEN - Sensitive 84

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D4.1 Clinical Trial Call documents WP4 5 - FTELE R — Document, report PU - Public 18

D4.2 Evaluation Report of the Clinical Trial Call WP4 5 - FTELE R — Document, report SEN - Sensitive 36

D5.1 NSS Call documents WP5 7 - ZonMw R — Document, report SEN - Sensitive 9

D5.2 List of funded networks 1 WP5 7 - ZonMw R — Document, report PU - Public 36

D5.3 List of funded networks 2 WP5 7 - ZonMw R — Document, report PU - Public 84

D6.1 Roadmap for genomic federated data analysis approach

WP6 8 - UT R — Document, report PU - Public 6

D6.2 Research dataset on returned diagnostic results 1

WP6 8 - UT R — Document, report PU - Public 36

D6.3 Research dataset on returned diagnostic results 2

WP6 8 - UT R — Document, report PU - Public 84

D7.1 Coordinated re-analysis diagnostic pipeline WP7 10 - CNAG R — Document, report PU - Public 12

D7.2 Best practice guidelines for diagnostic re- analysis & reinterpretation strategies

WP7 77 - KU Leuven R — Document, report PU - Public 30

D8.1 Software for long-read pipelines optimized for RD diagnoses

WP8 11 - SRUMC OTHER PU - Public 36

D8.2 Software update for integrated DNARNA pipeline optimized for RD discoveries 1

WP8 12 - TUM OTHER PU - Public 24

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D8.3 Software update for integrated DNARNA pipeline optimized for RD discoveries 2

WP8 12 - TUM OTHER PU - Public 60

D8.4 Software for multi-omics pipeline optimized for RD discoveries 1

WP8 37 - AMU OTHER PU - Public 36

D8.5 Software for multi-omics pipeline optimized for RD discoveries 2

WP8 37 - AMU OTHER PU - Public 72

D9.1 Regulatory grade cohort Inventory WP9 136 - LMU R — Document, report PU - Public 36

D9.2 Individual disease progression models WP9 8 - UT R — Document, report PU - Public 36

D9.3 Drug Development Tools WP9 14 - C-PATH R — Document, report PU - Public 72

D10.1 Disease burden and cost of illness framework for RDs

WP10 97 - Sciensano R — Document, report PU - Public 36

D10.2 Platform for development of regulatorygrade patient-relevant COAs in RD

WP10 13 - UKHD R — Document, report PU - Public 84

D10.3 Toolbox on collection of patient selfreported data

WP10 15 - VHIR R — Document, report PU - Public 84

D11.1 Result of the PoC studies for ATMP technologies

WP11 1 - INSERM DEM — Demonstrator, pilot, prototype

SEN - Sensitive 60

D11.2 ATMP technology Platform validated in clinical context

WP11 5 - FTELE DEM — Demonstrator, pilot, prototype

PU - Public 84

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D12.1 Assessment criteria for patient eligibility to individualised ASO treatment

WP12 16 - LUMC R — Document, report SEN - Sensitive 36

D12.2 Toolkit on outcome measures for N-of- 1 WP12 8 - UT DEC —Websites, patent filings, videos, etc

PU - Public 56

D12.3 Standardized platform for timely development of ASO individualized treatments

WP12 16 - LUMC DEM — Demonstrator, pilot, prototype

PU - Public 84

D13.1 VP specifications and documentation 1 WP13 1 - INSERM R — Document, report PU - Public 12

D13.2 VP specifications and documentation 2 WP13 1 - INSERM R — Document, report PU - Public 36

D13.3 VP specifications and documentation 3 WP13 1 - INSERM R — Document, report PU - Public 84

D14.1 Data Stewardship Wizard with the compilation of data models and data preparation workflows 1

WP14 16 - LUMC OTHER PU - Public 12

D14.2 Data Stewardship Wizard with the compilation of data models and data preparation workflows 2

WP14 16 - LUMC OTHER PU - Public 36

D14.3 Data Stewardship Wizard with the compilation of data models and data preparation workflows 3

WP14 16 - LUMC OTHER PU - Public 84

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D15.1 Portfolio of ERDERA large scale analysis services and pipelines

WP15 19 - UMCG DEC —Websites, patent filings, videos, etc

PU - Public 12

D15.2 Portfolio of ERDERA federated analysis methods and services

WP15 18 - UPM DEC —Websites, patent filings, videos, etc

PU - Public 24

D15.3 Technical guidelines for the implementation of services used in the use cases on federated analysis

WP15 10 - CNAG OTHER PU - Public 30

D16.1 First standard application of multidimensional knowledge networks (RD maps) addressing an RD case

WP16 21 - UM DEM — Demonstrator, pilot, prototype

PU - Public 36

D16.2 First version of ontologies (antenatal phenotypes and functional impacts) integrated with ORDO

WP16 1 - INSERM DEM — Demonstrator, pilot, prototype

PU - Public 36

D16.3 Treatabolome demonstrator WP16 1 - INSERM DEM — Demonstrator, pilot, prototype

PU - Public 72

D16.4 Knowledge bases and ontologies for PROM/ PCOM, disabilities and Treatabolome

WP16 1 - INSERM DEM — Demonstrator, pilot, prototype

PU - Public 84

D17.1 Recommendations to funders on addressing translatability of projects through funding opportunities

WP17 22 - EATRIS R — Document, report SEN - Sensitive 36

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D17.2 Result of feasibility study for consultancy- based expertise services

WP17 23 - FGB R — Document, report SEN - Sensitive 36

D17.3 Final report of the mentoring services activity

WP17 22 - EATRIS R — Document, report SEN - Sensitive 84

D18.1 Set of regulatory recommendations for CRNs

WP18 23 - FGB R — Document, report SEN - Sensitive 6

D18.2 Set of Regulatory support tools for preclinical development to facilitate regulatory filings and convergence for RD

WP18 22 - EATRIS R — Document, report PU - Public 36

D18.3 Roadmap for physical and written standards for quality control of ATMPs

WP18 22 - EATRIS R — Document, report SEN - Sensitive 60

D18.4 Summary report of ERDERA Qualification applications with regulatory Authorities

WP18 23 - FGB R — Document, report PU - Public 80

D19.1 First knowledge transfer of protocols & tools to CRN and selected JTC projects

WP19 24 - APHP OTHER PU - Public 36

D19.2 Final knowledge transfer of protocols & tools to CRN and selected JTC projects

WP19 24 - APHP OTHER PU - Public 84

D19.3 First protocol & tools for methodological upscaling of RD research projects

WP19 25 - UHASSELT OTHER PU - Public 36

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Deliverables

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Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D19.4 Final protocol & tools for methodological upscaling of RD research projects

WP19 25 - UHASSELT OTHER PU - Public 84

D20.1 Report on the identification and fulfilment of RD education and training needs

WP20 27 - VULSK R — Document, report PU - Public 36

D20.2 Impact assessment of ERDERA education and training programme

WP20 27 - VULSK R — Document, report PU - Public 84

D21.1 Base protocols for research grade rAAVs production

WP21 63 - GNT R — Document, report PU - Public 25

D21.2 Computational tools for of LNPs, EVs and biohybrids formulation development

WP21 164 - RJF OTHER SEN - Sensitive 30

D21.3 gRNAs for effective and safe gene editing WP21 70 - IMAGINE R — Document, report SEN - Sensitive 36

D21.4 Dataset of Gene therapy Immunogenicity assessment on humanized mouse model

WP21 1 - INSERM DATA — data sets, microdata, etc

PU - Public 36

D21.5 Multiomics single cell analysis and bioinformatic pipeline fit-for-purpose qualified to characterize immune response to ATMPs

WP21 45 - CHI DATA — data sets, microdata, etc

SEN - Sensitive 36

D21.6 Report on standard regulatory quality control parameters of mRNA formulations

WP21 161 - Pfizer R — Document, report PU - Public 50

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Deliverables

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Public — fully open ( automatically posted online) Sensitive — limited under the conditions of the Grant Agreement EU classified —RESTREINT-UE/EU-RESTRICTED, CONFIDENTIEL-UE/EU-CONFIDENTIAL, SECRET-UE/EU-SECRET under Decision 2015/444

Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D21.7 Regulatory compliant recommendations for rAAV analytics

WP21 77 - KU Leuven R — Document, report PU - Public 58

D22.1 Accelerator Hub inclusion criteria for research projects

WP22 30 - AFM R — Document, report SEN - Sensitive 12

D22.2 Impact analysis of Accelerator hub outputs 1 WP22 22 - EATRIS R — Document, report PU - Public 36

D22.3 Impact analysis of Accelerator hub outputs 2 WP22 22 - EATRIS R — Document, report PU - Public 84

D23.1 Best practice guidelines for creation and promotion of NMGs

WP23 1 - INSERM R — Document, report PU - Public 6

D23.2 First report on the impact of NMGs WP23 170 - UNEW R — Document, report PU - Public 36

D23.3 Second report on the impact of NMGs WP23 170 - UNEW R — Document, report PU - Public 80

D23.4 Recommendations on the NMG sustainability

WP23 31 - CCUH R — Document, report PU - Public 77

D24.1 Mapping of UCs specific needs and expectations 1

WP24 27 - VULSK R — Document, report SEN - Sensitive 12

D24.2 Mapping of UCs specific needs and expectations 2

WP24 27 - VULSK R — Document, report SEN - Sensitive 48

D24.3 Focused guidelines for actions at the National level 1

WP24 93 - REUH R — Document, report PU - Public 24

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Deliverables

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The labels used mean:

Public — fully open ( automatically posted online) Sensitive — limited under the conditions of the Grant Agreement EU classified —RESTREINT-UE/EU-RESTRICTED, CONFIDENTIEL-UE/EU-CONFIDENTIAL, SECRET-UE/EU-SECRET under Decision 2015/444

Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D24.4 Focused guidelines for actions at the National level 2

WP24 93 - REUH R — Document, report PU - Public 48

D24.5 Guidelines to increase UCs integration in ERDERA (including in funding activities) 1

WP24 32 - AICIB R — Document, report PU - Public 12

D24.6 Guidelines to increase UCs integration in ERDERA (including in funding activities) 2

WP24 32 - AICIB R — Document, report PU - Public 48

D24.7 Guidelines to increase UCs integration in ERDERA (including in funding activities) 3

WP24 32 - AICIB R — Document, report PU - Public 72

D25.1 White paper on ERNs Living Lab 1 WP25 39 - ASU-FC R — Document, report PU - Public 36

D25.2 White paper on ERNs Living Lab 2 WP25 39 - ASU-FC R — Document, report PU - Public 84

D25.3 Sustainability Plan for established strategic alliances and partnerships

WP25 7 - ZonMw DEM — Demonstrator, pilot, prototype

SEN - Sensitive 75

D25.4 Publication on the International CRNs WP25 34 - RDI R — Document, report PU - Public 84

D26.1 OEI - Requirement No. 1 WP26 1 - INSERM ETHICS SEN - Sensitive 1

D26.2 OEI - Requirement No. 2 WP26 1 - INSERM ETHICS SEN - Sensitive 12

D26.3 OEI - Requirement No. 3 WP26 1 - INSERM ETHICS SEN - Sensitive 24

D26.4 OEI - Requirement No. 4 WP26 1 - INSERM ETHICS SEN - Sensitive 36

D26.5 OEI - Requirement No. 5 WP26 1 - INSERM ETHICS SEN - Sensitive 48

77

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Deliverables

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Public — fully open ( automatically posted online) Sensitive — limited under the conditions of the Grant Agreement EU classified —RESTREINT-UE/EU-RESTRICTED, CONFIDENTIEL-UE/EU-CONFIDENTIAL, SECRET-UE/EU-SECRET under Decision 2015/444

Deliverable No

Deliverable Name Work Package No

Lead Beneficiary Type Dissemination Level Due Date (month)

D26.6 OEI - Requirement No. 6 WP26 1 - INSERM ETHICS SEN - Sensitive 60

D26.7 OEI - Requirement No. 7 WP26 1 - INSERM ETHICS SEN - Sensitive 72

D26.8 OEI - Requirement No. 8 WP26 1 - INSERM ETHICS SEN - Sensitive 84

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Deliverable D1.1 – Data Management Plan

Deliverable Number D1.1 Lead Beneficiary 1 - INSERM

Deliverable Name Data Management Plan

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 6 Work Package No WP1

Description

Detailed data management plan. To be constantly updated, and the resulting version will be submitted at each reporting period.

Deliverable D1.2 – Annual Work Plan Y2

Deliverable Number D1.2 Lead Beneficiary 1 - INSERM

Deliverable Name Annual Work Plan Y2

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 9 Work Package No WP1

Description

Detailing the planned activities and resources.

Deliverable D1.3 – Annual Work Plan Y3

Deliverable Number D1.3 Lead Beneficiary 1 - INSERM

Deliverable Name Annual Work Plan Y3

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 21 Work Package No WP1

Description

Detailing the planned activities and resources.

Deliverable D1.4 – Annual Work Plan Y4

Deliverable Number D1.4 Lead Beneficiary 1 - INSERM

Deliverable Name Annual Work Plan Y4

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 33 Work Package No WP1

Description

Detailing the planned activities and resources.

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Deliverable D1.5 – Annual Work Plan Y5

Deliverable Number D1.5 Lead Beneficiary 1 - INSERM

Deliverable Name Annual Work Plan Y5

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 45 Work Package No WP1

Description

Detailing the planned activities and resources.

Deliverable D1.6 – Annual Work Plan Y6

Deliverable Number D1.6 Lead Beneficiary 1 - INSERM

Deliverable Name Annual Work Plan Y6

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 57 Work Package No WP1

Description

Detailing the planned activities and resources.

Deliverable D1.7 – Annual Work Plan Y7

Deliverable Number D1.7 Lead Beneficiary 1 - INSERM

Deliverable Name Annual Work Plan Y7

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 69 Work Package No WP1

Description

Detailing the planned activities and resources.

Deliverable D1.8 – Periodic Progress Report 1

Deliverable Number D1.8 Lead Beneficiary 1 - INSERM

Deliverable Name Periodic Progress Report 1

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 26 Work Package No WP1

Description

ERDERA progress report.

80

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Deliverable D1.9 – Periodic Progress Report 2

Deliverable Number D1.9 Lead Beneficiary 1 - INSERM

Deliverable Name Periodic Progress Report 2

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 44 Work Package No WP1

Description

ERDERA progress report.

Deliverable D1.10 – Periodic Progress Report 3

Deliverable Number D1.10 Lead Beneficiary 1 - INSERM

Deliverable Name Periodic Progress Report 3

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 62 Work Package No WP1

Description

ERDERA progress report.

Deliverable D1.11 – Periodic Progress Report 4

Deliverable Number D1.11 Lead Beneficiary 1 - INSERM

Deliverable Name Periodic Progress Report 4

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 74 Work Package No WP1

Description

ERDERA progress report.

Deliverable D1.12 – Periodic Progress Report 5

Deliverable Number D1.12 Lead Beneficiary 1 - INSERM

Deliverable Name Periodic Progress Report 5

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 84 Work Package No WP1

Description

ERDERA progress report.

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Deliverable D1.13 – RDP SRIA Update

Deliverable Number D1.13 Lead Beneficiary 1 - INSERM

Deliverable Name RDP SRIA Update

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP1

Description

ERDERA SRIA updated

Deliverable D1.14 – Report on cumulative expenditure incurred

Deliverable Number D1.14 Lead Beneficiary 1 - INSERM

Deliverable Name Report on cumulative expenditure incurred

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 12 Work Package No WP1

Description

Report on cumulative expenditure incurred.

Deliverable D2.1 – Project website

Deliverable Number D2.1 Lead Beneficiary 2 - TEAMIT

Deliverable Name Project website

Type DEC —Websites, patent filings, videos, etc

Dissemination Level PU - Public

Due Date (month) 3 Work Package No WP2

Description

Report on ERDERA visual identity manual and website development.

Deliverable D2.2 – Communication and dissemination strategy

Deliverable Number D2.2 Lead Beneficiary 2 - TEAMIT

Deliverable Name Communication and dissemination strategy

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 6 Work Package No WP2

Description

Audience analysis, objectives; key messages; channels & tools; KPIs, initial plan.

82

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Deliverable D3.1 – Joint Transnational Call documents 1

Deliverable Number D3.1 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 1

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 3 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

Deliverable D3.2 – Joint Transnational Call documents 2

Deliverable Number D3.2 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 2

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 15 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

Deliverable D3.3 – Joint Transnational Call documents 3

Deliverable Number D3.3 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 3

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 27 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

Deliverable D3.4 – Joint Transnational Call documents 4

Deliverable Number D3.4 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 4

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 39 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

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Deliverable D3.5 – Joint Transnational Call documents 5

Deliverable Number D3.5 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 5

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 51 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

Deliverable D3.6 – Joint Transnational Call documents 6

Deliverable Number D3.6 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 6

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 63 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

Deliverable D3.7 – Joint Transnational Call documents 7

Deliverable Number D3.7 Lead Beneficiary 3 - DLR

Deliverable Name Joint Transnational Call documents 7

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 75 Work Package No WP3

Description

Report including MoU, call text, guidelines, application’s form templates.

Deliverable D3.8 – Evaluation Report of the JTCs 1

Deliverable Number D3.8 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 1

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 15 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

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Deliverable D3.9 – Evaluation Report of the JTCs 2

Deliverable Number D3.9 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 2

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 27 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

Deliverable D3.10 – Evaluation Report of the JTCs 3

Deliverable Number D3.10 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 3

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 39 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

Deliverable D3.11 – Evaluation Report of the JTCs 4

Deliverable Number D3.11 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 4

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 51 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

Deliverable D3.12 – Evaluation Report of the JTCs 5

Deliverable Number D3.12 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 5

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 63 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

85

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Deliverable D3.13 – Evaluation Report of the JTCs 6

Deliverable Number D3.13 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 6

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 75 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

Deliverable D3.14 – Evaluation Report of the JTCs 7

Deliverable Number D3.14 Lead Beneficiary 3 - DLR

Deliverable Name Evaluation Report of the JTCs 7

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 84 Work Package No WP3

Description

Report including the observers' report on the evaluation, the joint selection list of the projects to be funded.

Deliverable D4.1 – Clinical Trial Call documents

Deliverable Number D4.1 Lead Beneficiary 5 - FTELE

Deliverable Name Clinical Trial Call documents

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 18 Work Package No WP4

Description

Supporting documentation, application form, guidelines for applicants, data, privacy and regulations

Deliverable D4.2 – Evaluation Report of the Clinical Trial Call

Deliverable Number D4.2 Lead Beneficiary 5 - FTELE

Deliverable Name Evaluation Report of the Clinical Trial Call

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP4

Description

Report including the observers' report on the evaluation, the list of the projects to be funded

86

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Deliverable D5.1 – NSS Call documents

Deliverable Number D5.1 Lead Beneficiary 7 - ZonMw

Deliverable Name NSS Call documents

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 9 Work Package No WP5

Description

Supporting documentation (call text, application template, confidential document for evaluators)

Deliverable D5.2 – List of funded networks 1

Deliverable Number D5.2 Lead Beneficiary 7 - ZonMw

Deliverable Name List of funded networks 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP5

Description

List of the networks selected for funding, including summaries

Deliverable D5.3 – List of funded networks 2

Deliverable Number D5.3 Lead Beneficiary 7 - ZonMw

Deliverable Name List of funded networks 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP5

Description

List of the networks selected for funding, including summaries

Deliverable D6.1 – Roadmap for genomic federated data analysis approach

Deliverable Number D6.1 Lead Beneficiary 8 - UT

Deliverable Name Roadmap for genomic federated data analysis approach

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 6 Work Package No WP6

Description

Strategy agreed with 1+MG/GDI from the joint workshop

87

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Deliverable D6.2 – Research dataset on returned diagnostic results 1

Deliverable Number D6.2 Lead Beneficiary 8 - UT

Deliverable Name Research dataset on returned diagnostic results 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP6

Description

Aggregated data processed to generate returned diagnostic results

Deliverable D6.3 – Research dataset on returned diagnostic results 2

Deliverable Number D6.3 Lead Beneficiary 8 - UT

Deliverable Name Research dataset on returned diagnostic results 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP6

Description

Aggregated data processed to generate returned diagnostic results

Deliverable D7.1 – Coordinated re-analysis diagnostic pipeline

Deliverable Number D7.1 Lead Beneficiary 10 - CNAG

Deliverable Name Coordinated re-analysis diagnostic pipeline

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 12 Work Package No WP7

Description

Description of the revised coordinated re-analysis research diagnostic pipeline

Deliverable D7.2 – Best practice guidelines for diagnostic re-analysis & reinterpretation strategies

Deliverable Number D7.2 Lead Beneficiary 77 - KU Leuven

Deliverable Name Best practice guidelines for diagnostic re-analysis & reinterpretation strategies

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 30 Work Package No WP7

Description

Translation of successful diagnostic reanalysis strategies into best practice guidelines, including the propagation and evaluation of phenotypic and ontology usage

88

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Deliverable D8.1 – Software for long-read pipelines optimized for RD diagnoses

Deliverable Number D8.1 Lead Beneficiary 11 - SRUMC

Deliverable Name Software for long-read pipelines optimized for RD diagnoses

Type OTHER Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP8

Description

Software for ONT and PacBio sequencing to call most variant types released, tested and optimized for RD use

Deliverable D8.2 – Software update for integrated DNARNA pipeline optimized for RD discoveries 1

Deliverable Number D8.2 Lead Beneficiary 12 - TUM

Deliverable Name Software update for integrated DNARNA pipeline optimized for RD discoveries 1

Type OTHER Dissemination Level PU - Public

Due Date (month) 24 Work Package No WP8

Description

Software (update) released for an integrated DNA-RNA analysis of RD data released, tested and optimized for RD use

Deliverable D8.3 – Software update for integrated DNARNA pipeline optimized for RD discoveries 2

Deliverable Number D8.3 Lead Beneficiary 12 - TUM

Deliverable Name Software update for integrated DNARNA pipeline optimized for RD discoveries 2

Type OTHER Dissemination Level PU - Public

Due Date (month) 60 Work Package No WP8

Description

Software (update) released for an integrated DNA-RNA analysis of RD data released, tested and optimized for RD use

Deliverable D8.4 – Software for multi-omics pipeline optimized for RD discoveries 1

Deliverable Number D8.4 Lead Beneficiary 37 - AMU

Deliverable Name Software for multi-omics pipeline optimized for RD discoveries 1

Type OTHER Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP8

Description

Software released for an integrated multi-omics RD data released, tested and optimized for RD use

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Deliverable D8.5 – Software for multi-omics pipeline optimized for RD discoveries 2

Deliverable Number D8.5 Lead Beneficiary 37 - AMU

Deliverable Name Software for multi-omics pipeline optimized for RD discoveries 2

Type OTHER Dissemination Level PU - Public

Due Date (month) 72 Work Package No WP8

Description

Software released for an integrated multi-omics RD data released, tested and optimized for RD use

Deliverable D9.1 – Regulatory grade cohort Inventory

Deliverable Number D9.1 Lead Beneficiary 136 - LMU

Deliverable Name Regulatory grade cohort Inventory

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP9

Description

Online list of EMA approved RD cohort data sets

Deliverable D9.2 – Individual disease progression models

Deliverable Number D9.2 Lead Beneficiary 8 - UT

Deliverable Name Individual disease progression models

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP9

Description

4 pilot models for individual-subject progression modelling and prediction

Deliverable D9.3 – Drug Development Tools

Deliverable Number D9.3 Lead Beneficiary 14 - C-PATH

Deliverable Name Drug Development Tools

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 72 Work Package No WP9

Description

Two drug development tools established and accepted by EMA

90

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Deliverable D10.1 – Disease burden and cost of illness framework for RDs

Deliverable Number D10.1 Lead Beneficiary 97 - Sciensano

Deliverable Name Disease burden and cost of illness framework for RDs

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP10

Description

Framework to estimate cost of illness and burden of disease (BoD) in case data for other RDs becomes available

Deliverable D10.2 – Platform for development of regulatorygrade patient-relevant COAs in RD

Deliverable Number D10.2 Lead Beneficiary 13 - UKHD

Deliverable Name Platform for development of regulatorygrade patient-relevant COAs in RD

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP10

Description

Step-by-step description of COAs development process

Deliverable D10.3 – Toolbox on collection of patient selfreported data

Deliverable Number D10.3 Lead Beneficiary 15 - VHIR

Deliverable Name Toolbox on collection of patient selfreported data

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP10

Description

Pragmatic guidance on data standardization, regulatory and technological aspects for implementing patient selfreported data in clinical studies

Deliverable D11.1 – Result of the PoC studies for ATMP technologies

Deliverable Number D11.1 Lead Beneficiary 1 - INSERM

Deliverable Name Result of the PoC studies for ATMP technologies

Type DEM — Demonstrator, pilot, prototype

Dissemination Level SEN - Sensitive

Due Date (month) 60 Work Package No WP11

Description

Proof-of-concept studies to support efficacy of the chosen technology on relevant preclinical models of the identified diseases

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Deliverable D11.2 – ATMP technology Platform validated in clinical context

Deliverable Number D11.2 Lead Beneficiary 5 - FTELE

Deliverable Name ATMP technology Platform validated in clinical context

Type DEM — Demonstrator, pilot, prototype

Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP11

Description

Evaluation of the clinical translatability of the technology platform in the context of the selected disease

Deliverable D12.1 – Assessment criteria for patient eligibility to individualised ASO treatment

Deliverable Number D12.1 Lead Beneficiary 16 - LUMC

Deliverable Name Assessment criteria for patient eligibility to individualised ASO treatment

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP12

Description

Report on how to assess patient eligibility for individualized treatment (mutation, disease, patient and other considerations)

Deliverable D12.2 – Toolkit on outcome measures for N-of- 1

Deliverable Number D12.2 Lead Beneficiary 8 - UT

Deliverable Name Toolkit on outcome measures for N-of- 1

Type DEC —Websites, patent filings, videos, etc

Dissemination Level PU - Public

Due Date (month) 56 Work Package No WP12

Description

Catalogue of outcome measures available online

Deliverable D12.3 – Standardized platform for timely development of ASO individualized treatments

Deliverable Number D12.3 Lead Beneficiary 16 - LUMC

Deliverable Name Standardized platform for timely development of ASO individualized treatments

Type DEM — Demonstrator, pilot, prototype

Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP12

Description

92

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Project: 101156595 — ERDERA — HORIZON-HLTH-2023-DISEASE-07

Operational platform for patient identification, ASO design and synthesis, safety assessment in cells and animals and ethics management

Deliverable D13.1 – VP specifications and documentation 1

Deliverable Number D13.1 Lead Beneficiary 1 - INSERM

Deliverable Name VP specifications and documentation 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 12 Work Package No WP13

Description

First issue of a compilation of specifications, requirements, and documentations of the RD-VP, including onboarding guidance

Deliverable D13.2 – VP specifications and documentation 2

Deliverable Number D13.2 Lead Beneficiary 1 - INSERM

Deliverable Name VP specifications and documentation 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP13

Description

Intermediate issue of a compilation of specifications, requirements, and documentations of the RD-VP, including onboarding guidance

Deliverable D13.3 – VP specifications and documentation 3

Deliverable Number D13.3 Lead Beneficiary 1 - INSERM

Deliverable Name VP specifications and documentation 3

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP13

Description

Final issue of a compilation of specifications, requirements, and documentations of the RD-VP, including onboarding guidance

Deliverable D14.1 – Data Stewardship Wizard with the compilation of data models and data preparation workflows 1

Deliverable Number D14.1 Lead Beneficiary 16 - LUMC

Deliverable Name Data Stewardship Wizard with the compilation of data models and data preparation workflows 1

Type OTHER Dissemination Level PU - Public

93

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Due Date (month) 12 Work Package No WP14

Description

First issue of a compilation of data models and data ingestions pipelines (conversions, transformations, deposition options) disseminated as an updated version of the Data Stewardship Wizard

Deliverable D14.2 – Data Stewardship Wizard with the compilation of data models and data preparation workflows 2

Deliverable Number D14.2 Lead Beneficiary 16 - LUMC

Deliverable Name Data Stewardship Wizard with the compilation of data models and data preparation workflows 2

Type OTHER Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP14

Description

Intermediate issue of a compilation of data models and data ingestions pipelines (conversions, transformations, deposition options) disseminated as an updated version of the Data Stewardship Wizard

Deliverable D14.3 – Data Stewardship Wizard with the compilation of data models and data preparation workflows 3

Deliverable Number D14.3 Lead Beneficiary 16 - LUMC

Deliverable Name Data Stewardship Wizard with the compilation of data models and data preparation workflows 3

Type OTHER Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP14

Description

Final issue of a compilation of data models and data ingestions pipelines (conversions, transformations, deposition options) disseminated as an updated version of the Data Stewardship Wizard

Deliverable D15.1 – Portfolio of ERDERA large scale analysis services and pipelines

Deliverable Number D15.1 Lead Beneficiary 19 - UMCG

Deliverable Name Portfolio of ERDERA large scale analysis services and pipelines

Type DEC —Websites, patent filings, videos, etc

Dissemination Level PU - Public

Due Date (month) 12 Work Package No WP15

Description

Practical documentation for analysis service developers, operators and end-users on SOPs, templates, pipelines and services. It will be updated as necessary during the project

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Deliverable D15.2 – Portfolio of ERDERA federated analysis methods and services

Deliverable Number D15.2 Lead Beneficiary 18 - UPM

Deliverable Name Portfolio of ERDERA federated analysis methods and services

Type DEC —Websites, patent filings, videos, etc

Dissemination Level PU - Public

Due Date (month) 24 Work Package No WP15

Description

Practical documentation for federated analysis service developers, operators and end-users

Deliverable D15.3 – Technical guidelines for the implementation of services used in the use cases on federated analysis

Deliverable Number D15.3 Lead Beneficiary 10 - CNAG

Deliverable Name Technical guidelines for the implementation of services used in the use cases on federated analysis

Type OTHER Dissemination Level PU - Public

Due Date (month) 30 Work Package No WP15

Description

Description of the tools and methods used in the federated analyses use cases. It will be updated as necessary during the project

Deliverable D16.1 – First standard application of multidimensional knowledge networks (RD maps) addressing an RD case

Deliverable Number D16.1 Lead Beneficiary 21 - UM

Deliverable Name First standard application of multidimensional knowledge networks (RD maps) addressing an RD case

Type DEM — Demonstrator, pilot, prototype

Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP16

Description

First multidimensional knowledge networks - spanning from genetic causes to observed phenotypes and treatment pathways developed and implemented in an RD case/application

Deliverable D16.2 – First version of ontologies (antenatal phenotypes and functional impacts) integrated with ORDO

Deliverable Number D16.2 Lead Beneficiary 1 - INSERM

Deliverable Name First version of ontologies (antenatal phenotypes and functional impacts) integrated with ORDO

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Type DEM — Demonstrator, pilot, prototype

Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP16

Description

Ontological models produced and populated with curated content and published in Orphadata

Deliverable D16.3 – Treatabolome demonstrator

Deliverable Number D16.3 Lead Beneficiary 1 - INSERM

Deliverable Name Treatabolome demonstrator

Type DEM — Demonstrator, pilot, prototype

Dissemination Level PU - Public

Due Date (month) 72 Work Package No WP16

Description

Upscaled and expanded Treatabolome with additional disease groups and functionalities to facilitate data entry and data curation

Deliverable D16.4 – Knowledge bases and ontologies for PROM/PCOM, disabilities and Treatabolome

Deliverable Number D16.4 Lead Beneficiary 1 - INSERM

Deliverable Name Knowledge bases and ontologies for PROM/PCOM, disabilities and Treatabolome

Type DEM — Demonstrator, pilot, prototype

Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP16

Description

Completed knowledgebases – including interoperability (in RD maps) and inclusion in virtual platform

Deliverable D17.1 – Recommendations to funders on addressing translatability of projects through funding opportunities

Deliverable Number D17.1 Lead Beneficiary 22 - EATRIS

Deliverable Name Recommendations to funders on addressing translatability of projects through funding opportunities

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP17

Description

A document providing recommendations for the design of the calls based on the feedback of experts who provided support to the projects funded by ERDERA

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Deliverable D17.2 – Result of feasibility study for consultancy-based expertise services

Deliverable Number D17.2 Lead Beneficiary 23 - FGB

Deliverable Name Result of feasibility study for consultancy-based expertise services

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP17

Description

Report describing the outcomes from the feasibility study on the proposed management and operations of the consultancy service

Deliverable D17.3 – Final report of the mentoring services activity

Deliverable Number D17.3 Lead Beneficiary 22 - EATRIS

Deliverable Name Final report of the mentoring services activity

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 84 Work Package No WP17

Description

A report describing the result of the mentoring process

Deliverable D18.1 – Set of regulatory recommendations for CRNs

Deliverable Number D18.1 Lead Beneficiary 23 - FGB

Deliverable Name Set of regulatory recommendations for CRNs

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 6 Work Package No WP18

Description

Set of regulatory recommendations processing of heath data and biosamples handling in clinical studies to prepare CRNs on a regulatory-compliant clinical research

Deliverable D18.2 – Set of Regulatory support tools for preclinical development to facilitate regulatory filings and convergence for RD

Deliverable Number D18.2 Lead Beneficiary 22 - EATRIS

Deliverable Name Set of Regulatory support tools for preclinical development to facilitate regulatory filings and convergence for RD

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP18

Description

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Report on prospective tools, standards methods, and resources, including a regulatory database to support regulatory filings and convergence for rare disease

Deliverable D18.3 – Roadmap for physical and written standards for quality control of ATMPs

Deliverable Number D18.3 Lead Beneficiary 22 - EATRIS

Deliverable Name Roadmap for physical and written standards for quality control of ATMPs

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 60 Work Package No WP18

Description

Roadmap on how to realize the prioritized list of physical or written quality standards needed to support the platform technologies of the project

Deliverable D18.4 – Summary report of ERDERA Qualification applications with regulatory Authorities

Deliverable Number D18.4 Lead Beneficiary 23 - FGB

Deliverable Name Summary report of ERDERA Qualification applications with regulatory Authorities

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 80 Work Package No WP18

Description

Final overview of status and required future steps, of qualification applications with Regulatory authorities for novel therapeutic approaches developed within the project

Deliverable D19.1 – First knowledge transfer of protocols & tools to CRN and selected JTC projects

Deliverable Number D19.1 Lead Beneficiary 24 - APHP

Deliverable Name First knowledge transfer of protocols & tools to CRN and selected JTC projects

Type OTHER Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP19

Description

First reports & tools (including software) describing the State-of-the art methodology and recommendations transferred to ERDERA funded and inhouse research project, including biostatistical, epidemiological, data science, artificial intelligence and machine learning dimensions

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Deliverable D19.2 – Final knowledge transfer of protocols & tools to CRN and selected JTC projects

Deliverable Number D19.2 Lead Beneficiary 24 - APHP

Deliverable Name Final knowledge transfer of protocols & tools to CRN and selected JTC projects

Type OTHER Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP19

Description

Final reports & tools (including software) describing the State-of-the art methodology and recommendations transferred to ERDERA funded and inhouse research project, including biostatistical, epidemiological, data science, artificial intelligence and machine learning dimensions

Deliverable D19.3 – First protocol & tools for methodological upscaling of RD research projects

Deliverable Number D19.3 Lead Beneficiary 25 - UHASSELT

Deliverable Name First protocol & tools for methodological upscaling of RD research projects

Type OTHER Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP19

Description

First report & tools (including software) on the learnings and applications resulting from at least three conducted methodological projects

Deliverable D19.4 – Final protocol & tools for methodological upscaling of RD research projects

Deliverable Number D19.4 Lead Beneficiary 25 - UHASSELT

Deliverable Name Final protocol & tools for methodological upscaling of RD research projects

Type OTHER Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP19

Description

Final report & tools (including software) on the learnings and applications resulting from at least three conducted methodological projects

Deliverable D20.1 – Report on the identification and fulfilment of RD education and training needs

Deliverable Number D20.1 Lead Beneficiary 27 - VULSK

Deliverable Name Report on the identification and fulfilment of RD education and training needs

Type R — Document, report Dissemination Level PU - Public

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Due Date (month) 36 Work Package No WP20

Description

Evaluation of state-of-the-art of RD education and training in ERDERA and beyond

Deliverable D20.2 – Impact assessment of ERDERA education and training programme

Deliverable Number D20.2 Lead Beneficiary 27 - VULSK

Deliverable Name Impact assessment of ERDERA education and training programme

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP20

Description

Impact assessment of ERDERA education and training programme and further recommendations for continuous improvement

Deliverable D21.1 – Base protocols for research grade rAAVs production

Deliverable Number D21.1 Lead Beneficiary 63 - GNT

Deliverable Name Base protocols for research grade rAAVs production

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 25 Work Package No WP21

Description

Development of base protocols for rAAV production based on publicly available, state-of-the-art knowledge & methodologies

Deliverable D21.2 – Computational tools for of LNPs, EVs and biohybrids formulation development

Deliverable Number D21.2 Lead Beneficiary 164 - RJF

Deliverable Name Computational tools for of LNPs, EVs and biohybrids formulation development

Type OTHER Dissemination Level SEN - Sensitive

Due Date (month) 30 Work Package No WP21

Description

Development of computational tools for formulation development and running first test set

Deliverable D21.3 – gRNAs for effective and safe gene editing

Deliverable Number D21.3 Lead Beneficiary 70 - IMAGINE

Deliverable Name gRNAs for effective and safe gene editing

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Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP21

Description

In vitro nomination of an effective and specific gene editing reagent for each of the selected rare diseases

Deliverable D21.4 – Dataset of Gene therapy Immunogenicity assessment on humanized mouse model

Deliverable Number D21.4 Lead Beneficiary 1 - INSERM

Deliverable Name Dataset of Gene therapy Immunogenicity assessment on humanized mouse model

Type DATA — data sets, microdata, etc

Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP21

Description

Data set of complex immunogenicity results to at least four gene therapy tools in NSG-mice engrafted with human immune system

Deliverable D21.5 – Multiomics single cell analysis and bioinformatic pipeline fit-for-purpose qualified to characterize immune response to ATMPs

Deliverable Number D21.5 Lead Beneficiary 45 - CHI

Deliverable Name Multiomics single cell analysis and bioinformatic pipeline fit-for-purpose qualified to characterize immune response to ATMPs

Type DATA — data sets, microdata, etc

Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP21

Description

Methodical and Bioinformatics workflow on multiomics analysis of immune therapy response

Deliverable D21.6 – Report on standard regulatory quality control parameters of mRNA formulations

Deliverable Number D21.6 Lead Beneficiary 161 - Pfizer

Deliverable Name Report on standard regulatory quality control parameters of mRNA formulations

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 50 Work Package No WP21

Description

List of regulatory quality control parameters of LNP/EV (standard operating procedures for particles characterization, in vitro/vivo toxicology studies, benchmark materials)

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Deliverable D21.7 – Regulatory compliant recommendations for rAAV analytics

Deliverable Number D21.7 Lead Beneficiary 77 - KU Leuven

Deliverable Name Regulatory compliant recommendations for rAAV analytics

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 58 Work Package No WP21

Description

Validation of the currently available protocols for assessing the critical quality attributes of rAAV and develop new ones to increase accuracy and throughput

Deliverable D22.1 – Accelerator Hub inclusion criteria for research projects

Deliverable Number D22.1 Lead Beneficiary 30 - AFM

Deliverable Name Accelerator Hub inclusion criteria for research projects

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 12 Work Package No WP22

Description

Description of criteria & process for identifying and selecting research projects for accelerator

Deliverable D22.2 – Impact analysis of Accelerator hub outputs 1

Deliverable Number D22.2 Lead Beneficiary 22 - EATRIS

Deliverable Name Impact analysis of Accelerator hub outputs 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP22

Description

Summary and assessment of support provided to projects

Deliverable D22.3 – Impact analysis of Accelerator hub outputs 2

Deliverable Number D22.3 Lead Beneficiary 22 - EATRIS

Deliverable Name Impact analysis of Accelerator hub outputs 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP22

Description

Summary and assessment of support provided to projects

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Deliverable D23.1 – Best practice guidelines for creation and promotion of NMGs

Deliverable Number D23.1 Lead Beneficiary 1 - INSERM

Deliverable Name Best practice guidelines for creation and promotion of NMGs

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 6 Work Package No WP23

Description

Guidance for countries to develop NMGs including information on successful implementation

Deliverable D23.2 – First report on the impact of NMGs

Deliverable Number D23.2 Lead Beneficiary 170 - UNEW

Deliverable Name First report on the impact of NMGs

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP23

Description

Analysis of the impact of the National Mirror Groups on ERDERA and on the national RD activities and strategies

Deliverable D23.3 – Second report on the impact of NMGs

Deliverable Number D23.3 Lead Beneficiary 170 - UNEW

Deliverable Name Second report on the impact of NMGs

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 80 Work Package No WP23

Description

Analysis of the impact of the National Mirror Groups on ERDERA and on the national RD activities and strategies

Deliverable D23.4 – Recommendations on the NMG sustainability

Deliverable Number D23.4 Lead Beneficiary 31 - CCUH

Deliverable Name Recommendations on the NMG sustainability

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 77 Work Package No WP23

Description

Recommendations how to maintain active and productive work of NMGs

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Deliverable D24.1 – Mapping of UCs specific needs and expectations 1

Deliverable Number D24.1 Lead Beneficiary 27 - VULSK

Deliverable Name Mapping of UCs specific needs and expectations 1

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 12 Work Package No WP24

Description

Description of needs and expectations of underrepresented countries in ERDERA, including information on current barriers to participation

Deliverable D24.2 – Mapping of UCs specific needs and expectations 2

Deliverable Number D24.2 Lead Beneficiary 27 - VULSK

Deliverable Name Mapping of UCs specific needs and expectations 2

Type R — Document, report Dissemination Level SEN - Sensitive

Due Date (month) 48 Work Package No WP24

Description

Description of needs and expectations of underrepresented countries in ERDERA, including information on current barriers to participation

Deliverable D24.3 – Focused guidelines for actions at the National level 1

Deliverable Number D24.3 Lead Beneficiary 93 - REUH

Deliverable Name Focused guidelines for actions at the National level 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 24 Work Package No WP24

Description

Development of focused guidelines for actions at the National level for capacity development of UC R&I

Deliverable D24.4 – Focused guidelines for actions at the National level 2

Deliverable Number D24.4 Lead Beneficiary 93 - REUH

Deliverable Name Focused guidelines for actions at the National level 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 48 Work Package No WP24

Description

Development of focused guidelines for actions at the National level for capacity development of UC R&I

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Deliverable D24.5 – Guidelines to increase UCs integration in ERDERA (including in funding activities) 1

Deliverable Number D24.5 Lead Beneficiary 32 - AICIB

Deliverable Name Guidelines to increase UCs integration in ERDERA (including in funding activities) 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 12 Work Package No WP24

Description

Recommendations for procedures and measures to be implemented to increase UCs participation, including guidelines for promoting the inclusion of UCs in ERDERA funded actions (JTCs, Networking Calls, and others)

Deliverable D24.6 – Guidelines to increase UCs integration in ERDERA (including in funding activities) 2

Deliverable Number D24.6 Lead Beneficiary 32 - AICIB

Deliverable Name Guidelines to increase UCs integration in ERDERA (including in funding activities) 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 48 Work Package No WP24

Description

Recommendations for procedures and measures to be implemented to increase UCs participation, including guidelines for promoting the inclusion of UCs in ERDERA funded actions (JTCs, Networking Calls, and others)

Deliverable D24.7 – Guidelines to increase UCs integration in ERDERA (including in funding activities) 3

Deliverable Number D24.7 Lead Beneficiary 32 - AICIB

Deliverable Name Guidelines to increase UCs integration in ERDERA (including in funding activities) 3

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 72 Work Package No WP24

Description

Recommendations for procedures and measures to be implemented to increase UCs participation, including guidelines for promoting the inclusion of UCs in ERDERA funded actions (JTCs, Networking Calls, and others)

Deliverable D25.1 – White paper on ERNs Living Lab 1

Deliverable Number D25.1 Lead Beneficiary 39 - ASU-FC

Deliverable Name White paper on ERNs Living Lab 1

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 36 Work Package No WP25

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Description

White paper on the advancement and outputs of the 24 ERNs Living Lab

Deliverable D25.2 – White paper on ERNs Living Lab 2

Deliverable Number D25.2 Lead Beneficiary 39 - ASU-FC

Deliverable Name White paper on ERNs Living Lab 2

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP25

Description

White paper on the advancement and outputs of the 24 ERNs Living Lab

Deliverable D25.3 – Sustainability Plan for established strategic alliances and partnerships

Deliverable Number D25.3 Lead Beneficiary 7 - ZonMw

Deliverable Name Sustainability Plan for established strategic alliances and partnerships

Type DEM — Demonstrator, pilot, prototype

Dissemination Level SEN - Sensitive

Due Date (month) 75 Work Package No WP25

Description

Strategies for long-term collaboration as part of the overarching ERDERA exit strategy

Deliverable D25.4 – Publication on the International CRNs

Deliverable Number D25.4 Lead Beneficiary 34 - RDI

Deliverable Name Publication on the International CRNs

Type R — Document, report Dissemination Level PU - Public

Due Date (month) 84 Work Package No WP25

Description

White Paper on the International Dimension of the Clinical Research Networks

Deliverable D26.1 – OEI - Requirement No. 1

Deliverable Number D26.1 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 1

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 1 Work Package No WP26

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Description

Ethics Advisor_The proposal already provides for the appointment of an independent Ethics Advisor. Periodic reports by the Ethics Advisor must be submitted to the granting authority every 12 months, starting at M12 of the project.

The CVs and declarations of absence of conflict of interest for the selected EA(s) must be kept on file and provided to the granting authority upon request. European Commission Guidance for Ethics Advisors/Boards can be found at: https://ec.europa.eu/info/funding-tenders/opportunities/docs/2021-2027/horizon/guidance/roles-andfunctions-of- ethics-advisory-ethics-advisory-boards-in-ec-funded-projects_he_en.pdf

Deliverable D26.2 – OEI - Requirement No. 2

Deliverable Number D26.2 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 2

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 12 Work Package No WP26

Description

First report by the Ethics Adviser.

Deliverable D26.3 – OEI - Requirement No. 3

Deliverable Number D26.3 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 3

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 24 Work Package No WP26

Description

The second report by the Ethics Adviser.

Deliverable D26.4 – OEI - Requirement No. 4

Deliverable Number D26.4 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 4

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 36 Work Package No WP26

Description

The third report by the Ethics Adviser.

Deliverable D26.5 – OEI - Requirement No. 5

Deliverable Number D26.5 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 5

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Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 48 Work Package No WP26

Description

The fourth report by the Ethics Adviser.

Deliverable D26.6 – OEI - Requirement No. 6

Deliverable Number D26.6 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 6

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 60 Work Package No WP26

Description

The fifth report by the Ethics Adviser.

Deliverable D26.7 – OEI - Requirement No. 7

Deliverable Number D26.7 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 7

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 72 Work Package No WP26

Description

Sixth report by the Ethics Advisor.

Deliverable D26.8 – OEI - Requirement No. 8

Deliverable Number D26.8 Lead Beneficiary 1 - INSERM

Deliverable Name OEI - Requirement No. 8

Type ETHICS Dissemination Level SEN - Sensitive

Due Date (month) 84 Work Package No WP26

Description

Seventh report by the Ethics Advisor.

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LIST OF MILESTONES

Milestones

Grant Preparation (Milestones screen) — Enter the info.

Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

1 ERDERA Kick-off meeting WP1 1 - INSERM Minutes of the Kick-off meeting 1

2 Governance and Advisory Bodies set up WP1 1 - INSERM List of members 6

3 Number of funded Clinical trials with at least 50% of patients sample size recruited

WP4 5 - FTELE Aggregated monitoring reports 80

4 Implementation of Networking Support Scheme

WP5 6 - LMT Opening of funding scheme announced on ERDERA website

9

5 Establishment of Data Availability Committee (DAC)

WP6 8 - UT Composition of DAC and report of first meeting 2

6 First federated data sharing and re-analysis approach focusing on genome data

WP6 8 - UT First satellite instance (node) of the CRN Diagnostic Research

36

7 Long-read sequencing data included in data readiness processes for re-analysis

WP6 8 - UT Updated process description and dataset identifier(s)

61

8 Establishment of Task Forces for Data Analysis (DATF) and interpretation (DITF)

WP6, WP7 10 - CNAG Composition of DITF-DATF structure and report of first meeting

3

9 Phase I (yr 4-7): Data freeze for combined approach of distributed and federated analysis and interpretation, including non-standard approaches

WP7, WP6, WP15, WP8

10 - CNAG Identifier of available Data sets for interpretation beyond standard exome and genome approaches

30

10 Phase II (yr 4-7): Data freeze for combined approach of distributed and federated analysis and interpretation, including non-standard approaches

WP7, WP6, WP15, WP8

10 - CNAG Report on Data freeze including distributed/ federated access datasets to at least 10,000 data sets

48

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Milestones

Grant Preparation (Milestones screen) — Enter the info.

Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

11 Phase I (yr 1-3): Data freeze for distributed analysis and interpretation

WP6, WP7, WP15 10 - CNAG Report on 50,000 datasets analysed and interpreted through a distributed approach

36

12 Phase III: transition to federated approach completed

WP6, WP7, WP15, WP8

10 - CNAG Demonstrator of Federated access to EU and non- EU partners of ERDERA

72

13 Analysis pipeline for long-read technologies established and tested on positive controls

WP8 11 - SRUMC LRS-analysis pipelines tested, validated and released

36

14 New diagnoses of RDs by long-read technologies, RNA-DNA or multi-omics analyses established 1

WP8 11 - SRUMC Report on Gene/loci identification by hidden variants utilizing innovative long-read methods and on Pathogenic variant identification by innovative data analysis

36

15 New diagnoses of RDs by long-read technologies, RNA-DNA or multi-omics analyses established 2

WP8 11 - SRUMC Report on Gene/loci identification by hidden variants utilizing innovative long-read methods and on Pathogenic variant identification by innovative data analysis

84

16 DNA-RNA analysis pipelines established WP8 11 - SRUMC Pipelines tested, validated and released on selected repository for DNA-RNA analysis

60

17 Multi-omics analysis pipelines established WP8 11 - SRUMC Pipelines tested, validated and released on selected repository for multi-omics analysis

72

18 All institutional approvals for data extraction and sharing are in place

WP9, WP18 13 - UKHD Confirmation of filing and provision on demand of the institutional approval documents

24

19 European and US databases interoperable for joint analysis

WP9, WP14 13 - UKHD RDCA-DAP database in place 30

20 All integrated cohorts FAIRified and onboarded to VP

WP9, WP13, WP14

13 - UKHD Cohorts queryable online 36

21 Proven interoperability of registries filled WP9, WP14 13 - UKHD Federated queries to participating registries operational

48

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

automatically from different EHR systems in multiple countries

22 Development and validation of disease- specific PROM scales 1

WP10 15 - VHIR Core impact set for five demonstrator diseases published

18

23 Development and validation of disease- specific PROM scales 2

WP10 15 - VHIR Core impact set for five demonstrator diseases published and corresponding PROM scale designed and corresponding PROM scale designed

24

24 Development and validation of disease- specific PROM scales 3

WP10 15 - VHIR Core impact set for longitudinal validation in ≥ 3 languages completed

60

25 Development and revision of study protocols for the 4 use cases 1

WP10 15 - VHIR Study protocols completed for the 4 use cases following standardised EC format

24

26 Development and revision of study protocols for the 4 use cases 2

WP10 15 - VHIR Study protocols completed for the 4 use cases following standardised EC format and revised based on pilot studies

48

27 Anchoring of disease-specific indirect COAs to patient-relevant core impacts 1

WP10 15 - VHIR Outcome matrix for indirect outcome assessments established

24

28 Anchoring of disease-specific indirect COAs to patient-relevant core impacts 2

WP10 15 - VHIR Outcome matrix for indirect outcome assessments established and protocol for validation studies available; longitudinal anchoring studies completed for ≥ 2 COA assessments per use case

72

29 Anchoring of disease-specific indirect COAs to patient-relevant core impacts 3

WP10 15 - VHIR Outcome matrix for indirect outcome assessments established and protocol for validation studies available; meaningful withinpatient change established

84

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

30 Standardized protocol for DALY and Cost of Illness

WP10 15 - VHIR Document that details the standardized protocols for DALY and Cost of Illness are finalized

60

31 Target diseases ranking with at least 3 ranked diseases that matched to technology platforms

WP11 1 - INSERM Report on a list of parameters indicating the selected diseases are qualified to be treated with technology platforms

24

32 List of ATMP dedicated Clinical Networks for the identified PoC platform

WP11 1 - INSERM Listing of the networks and their clinical sites 24

33 Selection of the platforms best matched to prioritized target diseases

WP11 1 - INSERM Identification of the best performing technological platform adapted to the treatment of the high-ranked diseases

36

34 Clinical Trial with ATMPs initiated WP11 1 - INSERM Clinical Trial Application filed 84

35 Preclinical efficacy studies use case 1 and 3 completed

WP12 16 - LUMC Publication on results of efficacy studies 36

36 First patient treated in underrepresented country in a named patient setting

WP12 16 - LUMC Pseudonymised Clinical Report 36

37 First patient treated in a named patient setting WP12 16 - LUMC Pseudonymised Clinical Report 48

38 Platform rolled out in 3 other centres WP12 16 - LUMC Clinical report and performance assessment report of platform roll-out

72

39 Landscape analysis of ERDERA data processing needs completed, including methodology for measuring acceleration rate of FAIRification

WP14 16 - LUMC Report on analysis results and roadmap for addressing identified needs with risk mitigation measures

8

40 Data models and ingestion results validated against EOSC FAIR data publishing recommendations

WP14, WP13 16 - LUMC Results of Automated compliance testing by the public FAIR Evaluator tool

36

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

41 First release of a portable data and analysis tracking system

WP6, WP7, WP16, WP14

19 - UMCG Software available in GitHub 6

42 Initial version of the ERDERA analysis platform deployed

WP9, WP6, WP7, WP16, WP13, WP14, WP10

19 - UMCG Platform available online 12

43 First version of the coordinated research diagnostics pipeline prioritised in 7 available for distributed implementation

WP9, WP7, WP16, WP10

19 - UMCG Pipeline available in a public repository 24

44 Two use cases on federated analysis completed

WP15 19 - UMCG Short report explaining the use cases outcomes, and the tools and services involved

36

45 First ontology versions for antenatal phenotypes for RD and functional impacts for RD

WP16 20 - AUMC Demonstrator (involved Task 16.1; 16.2 and 16.5) 18

46 Treatabolome with crowd-sourcing functionality available

WP16 20 - AUMC New version of the Treatabolome available online with the additional functionality (Task 16.3)

36

47 Graph-based infrastructure WP16 20 - AUMC A lightweight infrastructure to host, link to, and interlink semantic resources (Task 16.5)

80

48 Evaluation of the use of the Innovation Management Toolbox during within M1- M36 ERDERA lifespan

WP17 22 - EATRIS Report delivered to the Executive and Governing Bodies

36

49 Composition of the Regulatory Support Group and first Kick off meeting

WP18 23 - FGB Completion of composition and launch of Regulatory Support Group

3

50 First set of sources, guidance, recommendations and webinars delivered

WP18 23 - FGB Documents and webinars available on project internal channels

20

51 Second set of sources, guidance, recommendations and webinars delivered

WP18 23 - FGB Documents and webinars available on project internal channels

40

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

52 Regulatory recommendations for the technological deliverables of ERDERA

WP18 23 - FGB Recommendations available on project internal channels

24

53 Interaction workflows with the Data Services and Acceleration Hub 1

WP19, WP16, WP21, WP22

24 - APHP Report 36

54 Interaction workflows with the Data Services and Acceleration Hub 2

WP19, WP16, WP21, WP22

24 - APHP Report 84

55 Yearly rounds of RD research education and training activities successfully delivered (Y1)

WP20 26 - EURORDIS Datasets from satisfaction survey & participants acquired knowledge evaluation results

12

56 Yearly rounds of RD research education and training activities successfully delivered (Y2)

WP20 24 - APHP Datasets from satisfaction survey & participants acquired knowledge evaluation results

24

57 Yearly rounds of RD research education and training activities successfully delivered (Y3)

WP20 26 - EURORDIS Datasets from satisfaction survey & participants acquired knowledge evaluation results

36

58 Yearly rounds of RD research education and training activities successfully delivered (Y4)

WP20 26 - EURORDIS Datasets from satisfaction survey & participants acquired knowledge evaluation results

48

59 Yearly rounds of RD research education and training activities successfully delivered (Y5)

WP20 26 - EURORDIS Datasets from satisfaction survey & participants acquired knowledge evaluation results

60

60 Yearly rounds of RD research education and training activities successfully delivered (Y6)

WP20 26 - EURORDIS Datasets from satisfaction survey & participants acquired knowledge evaluation results

72

61 Yearly rounds of RD research education and training activities successfully delivered (Y7)

WP20 26 - EURORDIS Datasets from satisfaction survey & participants acquired knowledge evaluation results

84

62 Agreement on base manufacturing process of AAVs

WP21 28 - UC Report/Plan 6

63 Immune cell lines available for mRNA technology platform

WP21 28 - UC Delivery of specific primary T-cell lines and CAR-T cells as standard tools

12

64 Immune gene list content WP21 28 - UC Verification of the sequence of the genes 18

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

applicable to the transcriptomic design for DD- PCR – data set

65 Multiomics immune response analyses WP21 28 - UC Data set enabling selection of best suitable molecular, proteomics, metabolic, functional platforms, test for the further tasks of qualification and validation

24

66 Analytical toolbox for RNA formulations WP21 28 - UC Analytical toolbox for in vitro and in vivo characterization of efficiency, immunogenicity and safety of RNA formulations

36

67 Adoption of base manufacturing protocol and scale up

WP21 28 - UC Report/Plan 36

68 Treg development candidate WP21 28 - UC Laboratory Cas-specific Treg prototype generated under non-GMP conditions available for in depth PoC preclinical studies

36

69 AAV production process scale up WP21 28 - UC Larger scale bioprocessing using bioreactors and suspension producer cells

36

70 Analysis and test of RNA sequences WP21 28 - UC Design, production, analytical characterization of RNA molecules, and head-to-head comparison of RNA sequences in reporter cell lines

42

71 Validation of the assays for selected CQAs (minimum being genome titer) with accompanying inter-laboratory variability analysis

WP21 28 - UC Report 48

72 Identification of effective and safe gene delivery modalities for the selected organs

WP21 28 - UC Validation across different Tasks 48

73 Selection of in vivo gene delivery modalities WP21 28 - UC In vivo ranking of gene delivery modalities for the selected organs

48

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

74 PoC of gene correction in relevant in vivo models of the selected diseases

WP21 28 - UC Validation across different organs and diseases 58

75 Public-Private Partnership accelerator workflows completed

WP22 29 - FFRD Set of SOPs 12

76 Public-Private Partnership accelerator marketplace construction completed

WP22 29 - FFRD Webpages and inaugural meeting of investor launching accelerator

24

77 75% (23 out of 31) of NMGs established in ERDERA participating countries

WP23 1 - INSERM Aggregated report including lists of participants and three-year plan of NMGs

12

78 100% of NMGs established in ERDERA participating countries

WP23 1 - INSERM Aggregated report including lists of participants and three-year plan of NMGs

24

79 Successful gain and dissemination of specific knowledge among NMGs trough train the trainees and thematic workshops

WP23 1 - INSERM Results of Annual NMG surveys on knowledge dissemination among local NMG members

84

80 Workshops on actions to improve UCs participation (back-to-back with funders meeting) 1

WP24 32 - AICIB Reports including Statistical analysis of indicators implementation (number of actions implemented, number of actions accepted to implementation, etc.) to improve UCs participation in RDP funded activities

11

81 Workshops on actions to improve UCs participation (back-to-back with funders meeting) 2

WP24 32 - AICIB Reports including Statistical analysis of indicators implementation (number of actions implemented, number of actions accepted to implementation, etc.) to improve UCs participation in RDP funded activities

48

82 Impact of UCs on ERDERA activities demonstrated 1

WP6, WP7, WP16, WP11, WP4, WP5, WP15, WP18, WP14, WP26,

32 - AICIB Report Statistics analysis of implementation indicators (number of UCs cases describing impacts, number of UCs integrated in ERDERA activities)

48

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

WP10, WP1, WP2, WP25, WP8, WP9, WP19, WP17, WP21, WP12, WP23, WP22, WP13, WP20, WP3, WP24

83 Impact of UCs on ERDERA activities demonstrated 2

WP6, WP7, WP16, WP11, WP4, WP5, WP15, WP18, WP14, WP26, WP10, WP1, WP2, WP25, WP8, WP9, WP19, WP17, WP21, WP12, WP23, WP22, WP13, WP20, WP3, WP24

32 - AICIB Report Statistics analysis of implementation indicators (number of UCs cases describing impacts, number of UCs integrated in ERDERA activities)

72

84 Two Task Forces (TF) per year completed (Y2)

WP25 7 - ZonMw Article, white paper or planned outcomes of the Task Force

24

85 Two Task Forces (TF) per year completed (Y3)

WP25 7 - ZonMw Article, white paper or planned outcomes of the Task Force

36

86 Two Task Forces (TF) per year completed (Y4)

WP25 7 - ZonMw Article, white paper or planned outcomes of the Task Force

48

87 Two Task Forces (TF) per year completd (Y5) WP25 7 - ZonMw Article, white paper or planned outcomes of the Task Force

60

88 Two Task Forces (TF) per year completed (Y6)

WP25 7 - ZonMw Article, white paper or planned outcomes of the Task Force

72

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Milestones

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Milestone No

Milestone Name Work Package No Lead Beneficiary Means of Verification Due Date (month)

89 Two Task Forces (TF) per year completed (Y7)

WP25 7 - ZonMw Article, white paper or planned outcomes of the Task Force

84

90 Three International Conferences organised WP25 7 - ZonMw Dates, programmes and reports of Conference on website

84

LIST OF CRITICAL RISKS

Critical risks & risk management strategy

Grant Preparation (Critical Risks screen) — Enter the info.

Risk number

Description Work Package No(s)

Proposed Mitigation Measures

1 Incapacity or departure of coordinator [(i) likelihood: Low, (ii) severity: High]

WP1 High quality of the coordination team with possibility of interim by one of the Workstream leaders. Strong commitment of the Coordinator.

2 Misuse of resources [(i) likelihood: Low, (ii) severity: High]

WP1 Most partners’ institutions are experiences and equipped with internal financial accounting and auditing office. Close Data-Driven Monitoring will help in identifying abnormalities.

3 Financial bottleneck due to underestimation of contribution capacity of partner(s) and/or withdrawal of major partner [(i) likelihood: Medium, (ii) severity: High]

WP1 Detailed business plan prepared before launch of the project including 15% risk of budget loss. Financial management issues set up in Consortium Agreement. Emergency mitigation procedure set up – possibility to adjust the work plan and align budget-

4 Situations of force majeure [(i) likelihood: Medium, (ii) severity: Medium]

WP1 Agile management adapting to unforeseen events. Leveraging on past COVID-19 and Ukraine war experience (e.g., continuous information back-up on remote secured servers, hybrid/online shift of activities, strategic context intelligence).

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Critical risks & risk management strategy

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Risk number

Description Work Package No(s)

Proposed Mitigation Measures

5 Insufficient coordination of priorities / unresolved conflicts about strategy [(i) likelihood: Low, (ii) severity: Medium]

WP1 Criteria and process of prioritization agreed at the start of the project and validated by GB; Possibility to call for MAB recommendations; Flexibility of Annual Work Plans.

6 National financial bottlenecks for funding joint transnational research projects [(i) likelihood: Medium, (ii) severity: Medium]

WP3 Earmarking adequate national budgets in line with national research capacity and costs; Postevaluation negotiations on increasing national budget and/or budget negotiations with applicants; Use of EC co-funding to adjust national budget shortcomings.

7 Delay in the clinical trials progress (e.g., difficulties in recruiting subjects; difficulties in obtaining approvals by competent authorities) [(i) likelihood: Medium, (ii) severity: Medium]

WP4 The second step pf evaluation focuses on feasibility such that funded trials should be ready for approval. Active involvement of PAOs in the CTs. Payments against achievements of the milestones.

8 Delay in implementation and scalation of federated diagnostic research approach [(i) likelihood: High, (ii) severity: Medium]

WP6, WP7, WP15 Focus on National 1+MG/GDI infrastructures that are ready such as France and Sweden first, then progress according to readiness status.

9 Delay in data availability and sharing [(i) likelihood: High, (ii) severity: Medium]

WP6 According to data availability status, the sharing of data will be phased. While the risk for some centres for delay is high, the overall risk is medium or lower since delivering centres will compensate for delayed ones.

10 Delay in or Slower distributed re-analysis of REAL datasets [(i) likelihood: Medium, (ii) severity: Medium]

WP6, WP7, WP15 Apply to use EuroHPC resources. Include additional local clusters for the analysis. Prioritise reanalyses with higher solve rate. Phasing of re-analysis according to readiness levels.

11 Long-read technologies (and analyses) do not progress as fast as anticipated [(i) likelihood: Medium, (ii) severity: Medium]

WP7, WP8 Less recent (and/or contributed) data available than estimated, enlarge focus on re-analysis of already available data (6/7). Purposely, long-read data processing is based on three technologies to mitigate risk and adapt (PacBio, ONT, Bionano).

12 Too few (matched) DNA-RNA (or other -omics) datasets provided by partners [(i) likelihood: Medium, (ii) severity: Medium]

WP8 Integrative tools can largely be employed on already available data (e.g., Solve-RD); select best available use cases from new data. Data and tool federation (by 6-8) will allow including more data.

13 EHR access and linkage between registries and social security data restricted by local regulations

WP9 Good feasibility of initially selected use-cases; enrolment of alternative ERDERA partners to project; seek support from regulators & interaction with the EHDS.

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Critical risks & risk management strategy

Grant Preparation (Critical Risks screen) — Enter the info.

Risk number

Description Work Package No(s)

Proposed Mitigation Measures

and difficult to generalize across Europe [(i) likelihood: Medium, (ii) severity: High]

14 Data content and/or quality of patient cohorts insufficient [(i) likelihood: Low, (ii) severity: High]

WP9 Quality check of dataset extracts and metadata for cohort selection, review of existing pilot studies using selected cohorts, data improvement measures from DSH for still active cohort studies.

15 Insufficient predictive power of disease progression models [(i) likelihood: Low, (ii) severity: High]

WP9 Modify analytical approaches, obtain more / better data by international collaborations.

16 Delay in Outcome Research validation studies at some clinical site [(i) likelihood: Medium, (ii) severity: High]

WP10 Expand the network of study sites by additional ERN sites to ensure rapid recruitment.

17 PROMs not sensitive to change in the timeframe of validation studies (2 years) [(i) likelihood: Medium, (ii) severity: High]

WP10 Highly sensitive indirect COAs will be included and anchored to patient-relevant core impacts.

18 Delays on outcome research clinical studies implementation due to regulatory or ethics issues [(i) likelihood: Medium, (ii) severity: High]

WP10 Feasibility assessment of clinical sites. Early initiation of regulatory and ethics clearance tasks. Build on successful cross border cases of implementation of mHealth.

19 Patients not adhering to the clinical protocol [(i) likelihood: Low, (ii) severity: High]

WP10 Patient Engagement workshops in collaboration with EURORDIS, ePAGs and National Pos. Clinical protocol co-designed with patients.

20 No positive results from the PoC studies [(i) likelihood: Medium, (ii) severity: High]

WP11 Leverage on the experience from the high number of highly promising results obtained using various ATMPs platforms in preclinical models.

21 Difficult to implement ASO platform beyond initial partners, especially in centres from underrepresented countries [(i) likelihood: Medium, (ii) severity: Medium]

WP12 Not all components have to be implemented locally, treatment board, ASO design and testing can be done at LUMC and UT. Proactive support from ERNs for the individualised part of platform implementation.

22 New resources require significant modifications on the design and implementation of the RD- VP and its components, beyond the capacity of

WP13 RD-VP flexibility and design, with minimal requirements for onboarding resources.

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Critical risks & risk management strategy

Grant Preparation (Critical Risks screen) — Enter the info.

Risk number

Description Work Package No(s)

Proposed Mitigation Measures

the allocated resources [(i) likelihood: Low, (ii) severity: Medium]

23 Resources experiencing legal or IPR barriers to onboard [(i) likelihood: Medium, (ii) severity: Medium]

WP13 Legal, regulatory and IPR support from ERDERA experts to de-risk onboarding process.

24 CRN and funded projects deploy insufficient resources to contribute to the ERDERA-Data Hub [(i) likelihood: High, (ii) severity: Medium]

WP12 Earmarked CRN and JTC grant funding contributing to EREDERA Data Hub (ii) Added value demonstrated for the first projects contributing to the Data Hub.

25 Complexity of data and data access is greater than expected, or inability to address on-time the demand [(i) likelihood: High, (ii) severity: Medium]

WP14 Feasibility assessment of the request to DSH including flexible data processing scenarios. FAIRification targets will be reprioritized to ensure a critical mass is reached for analysis. Methodology can be developed based on historical data, as well as on simulated data addressingEthical and Regulatory related issues.

26 Data and analysis system not meeting the CRN needs [(i) likelihood: Low, (ii) severity: Medium]

WP15, WP14 Revise AGILE methodology with short-term sprints and promote higher involvement of end users in the process.

27 Insufficient compute and/or storage capacity available for the CRN and funded project needs [(i) likelihood: Medium, (ii) severity: Medium]

WP9, WP6, WP7, WP15, WP14, WP10, WP8

Collaboration with EU compute and storage initiatives (already initiated). Limit processing to downstream data (e.g. VCF files) and exclude raw data from analysis (e.g. full sequences). Prioritise focused analyses (e.g., on particular patient groups).

28 Insufficient resources to code portable pipelines and support local implementations for diagnostics. [(i) likelihood: Low, (ii) severity: Medium]

WP7, WP15, WP8 Leverage existing open-source pipelines. Rely on locally implemented pipelines (even if not portable versions).

29 GDI infrastructure and/or GA4GH standards not fit for purpose on time for federated learning [(i) likelihood: Medium, (ii) severity: Medium]

WP6, WP7, WP15, WP14

Bring in alternative solutions developed and/or implemented by participating partners in other projects (e.g. Genomed4All, DataSHIELD).

30 Fail to collaborate with HPO to extend its content to prenatal echographic phenotypes [(i) likelihood: Low, (ii) severity: Medium]

WP16 Collaborative generation of a stand-alone ontology of prenatal phenotypes of RD and an ontological module in ORDO.

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Critical risks & risk management strategy

Grant Preparation (Critical Risks screen) — Enter the info.

Risk number

Description Work Package No(s)

Proposed Mitigation Measures

31 Difficulty engaging with experts and patient organisations annotating RD with functional consequences [(i) likelihood: Medium, (ii) severity: Medium]

WP16 Revise prioritization of RD based on opportunities for collaboration after specific dissemination efforts to engage with stakeholders.

32 Low engagement of experts [(i) likelihood: Low, (ii) severity: High]

WP17, WP18 Leverage on existing database of already committed experts. Training and engagement activities. Recognition of the expert support to projects funded by ERDERA.

33 Low awareness of the services provided [(i) likelihood: Low, (ii) severity: Medium]

WP17, WP18 Maximizing the dissemination of the services by the communication & Dissemination task and by ERDERA Partner.

34 Non-availability of education and training providers for emerging training needs [(i) likelihood: Medium, (ii) severity: Medium]

WP20 Recruitment of training providers from ERDERA partners, extended to national systems (through NMGs) and to collaboration with other initiatives.

35 Insufficient number of education and training participants [(i) likelihood: Low, (ii) severity: Medium]

WP20 Increased awareness-raising and dissemination efforts in collaboration with 2, targeted at national systems (through NMGs) and RD research ecosystem (through ERDERA partners and collaborations).

36 In Vitro Transcription Amplification of DNA or RNA fails [(i) likelihood: Low, (ii) severity: Medium]

WP21 Development of recombinant production in yeast (ART-ARNm)

37 Therapy response and immunogenicity evaluation hampered by poor efficacy and/or delays in the technology development of T21.1 - T21.4 [(i) likelihood: Low, (ii) severity: Medium]

WP21 Application of relevant "use cases" (i.e., known gene therapy tools) to develop the test platforms in T21.5)

38 Low frequency of antigen-specific B cells in peripheral blood limiting the detection and/or selection and expansion of specific B cells [(i) likelihood: Medium, (ii) severity: Medium]

WP21 Samples from patients with preformed immunity or vaccinated as model systems to establish the B-cell methods.

39 There are not enough projects with high potential WP22 Initial ERDERA strategy fostering preclinical development as potential target for the calls

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Critical risks & risk management strategy

Grant Preparation (Critical Risks screen) — Enter the info.

Risk number

Description Work Package No(s)

Proposed Mitigation Measures

for acceleration, resources to bring projects to investment-readiness, and interest from investors. [(i) likelihood: Low, (ii) severity: High]

& matching the strategy of interested investors. Opening the accelerator services to external funding organisations/ initiatives with interest in RDs.

40 NMG not implemented in all EU participating countries after the second year of ERDERA [(i) likelihood: High, (ii) severity: Medium]

WP23 Potential NMGs already identified in 15 (out of 31) EU countries. Dissemination of the added value of existing NMGs (established in EJP RD). Close collaboration with national stakeholders.

41 Inactive or non-compliance of NMGs with the foreseen activities [(i) likelihood: Medium, (ii) severity: Medium]

WP23 Close monitoring of NMG activities and Financial support provided to active ones.

42 Potential lack of commitment by underrepresented countries and/or modifications in national priorities/ national contact points (due to governmental shifts) [(i) likelihood: Medium, (ii) severity: Medium]

WP24 Involvement, by design, of national representatives (policy makers, academics, clinicians, patient representatives, industry) and good communication with the national stakeholders.

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PROJECT REVIEWS

Project Reviews

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Review No

Timing (month)

Location Comments

RV1 12 online 5 experts

RV2 24 online 5 experts

RV3 36 online 5 experts

RV4 48 online 5 experts

RV5 60 online 5 experts

RV6 72 online 5 experts

RV7 84 online 5 experts

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[101156595] – [ERDERA] - Part B - Page 1 of 83

Part B: technical description

ERDERA - EUROPEAN RARE DISEASES RESEARCH ALLIANCE [This document is tagged. Do not delete the tags; they are needed for processing.] #@APP-FORM-HECOFUND@#

HISTORY OF CHANGES

Version

(date)

Changes Page

Annex 1 Part A

29.02.2024 Correction of the ISO code for Portugal (from PO to PT) and Poland (from PO to

PL)

NA

Change of Beneficiaries acronyms (from Utartu to UTARTU; from ICS to VHIR;

from AMC to AUMC; from Uhasselt to UHASSELT; from AOU MEYER IRCCS

to AOU Meyer IRCCS; from Centogene AG to Centogene; from Copenhagen

Economics to CE; from REGIONH (Copenhagen University Hospital) to

REGIONH; from FHG to Fraunhofer; from RS (GMS-GMCK) to RS; from MAPI

Research Trust to MRT; from SCIENSANO to Sciensano; from SONIO to Sonio;

from University of Campania L. to UNICAMPANIA; from University of Crete to

UoC; from Cadi Ayyad University to CAU; from Aalborg University Hospital to

Aalborg UH; from Aarhus Univeristy Hospital to Aarhus UH; from IABS to IABS-

EU; ; from CRG to CRG-CERCA; from GMS-Skane Region to GMS-RS.

NA

Removal of Beneficiaries: 59-Fraunhofer UK; 62-GA UK; 85-UNEW; 86-CUH; 90-

PMU; 128-ULEIC;143-QUB-UK; 149-UCL; 151-UCAM.

NA

Addition of Beneficiaries: 59-IFD; 62-MZd; 85-MoH SR; 86-RDG; 90-JSI; 128-

UANTWERPEN; 148-IPG; 149-UO.

NA

Addition of Affiliated Entities to Beneficiaries: 60-FSJD-CERCA – 60.1-HSJD; 62-

MZd – 62.1-AZV; 136.-LMU – 136.1-KUM.

NA

Addition of Associated Partners: 167*-Fraunhofer UK; 168*-GA UK; 169*-UNEW;

170*-CUH; 171*-ULEIC; 172*-QUB-UK; 173*-UCL; 174*-UCAM.

NA

Removal of Associated Partners: 170*-Utrecht UMC; 171*-LH (LabCorp). NA

Addition of Deliverables: D1.14 Report on cumulative expenditure incurred (M12);

D1.7 Annual Work Plan 6 (M69); D2.1 Project website (M3).

NA

Removal of Deliverables: D1.13 Annual Progress Report 6 (M75). NA

Modification of Deliverables due date: D1.8 from M15 to M18; D1.9 from M27 to

M36; D1.10 from M39 to M54; D1.11 from M51 to M72; D1.12 from M63 to M84.

NA

Change of Deliverables Lead Beneficiary: D13.1, D13.2, D13.3 from

Utwente/INSERM to INSERM; D14.1, D14.2, D14.3 from AMUC/SRUMC/LUMC

to LUMC; D22.2, D22.3 from EATRIS/FFRD to EATRIS; D24.1, D24.2 from

VULSK, REUH to VULSK; D24.3, D24.4 from REUH, VULSK to REUH; D24.5,

D24.6, D24.7 from AICIB, IMGGE to AICIB; D25.1, D25.2 from ASU-FC, HSJD

to ASU-FC.

NA

Removal of Milestones: MS2.1 Initial set of tools and activities (M6). NA

Precision in WP 1 description for the task 1.3 “Monitoring of the Partnership

activities” that this task will also consider criteria on ERDERA Results

exploitability by non-rare conditions; application of these criteria to funded research

activities will be considered in the funding opportunities. This addresses the

recommendation provided through the proposal reviewers’ feedback.

NA

Associated with document Ref. Ares(2024)5602406 - 02/08/2024

[101156595] – [ERDERA] - Part B - Page 2 of 83

Change of PIC from 998732274 to 919322739 for 20-AUMC; from 882251279 to

939076692 for 59-IFD; from 880953613 to 999602073 for 87-REGIONSYD; from

884513125 to 997381064 for 130-AalborgUH; and from 904861591to 999977172

for 174-UCAM

NA

20.03.2024 Addition of Deliverables: D1.14 Report on cumulative expenditure incurred (M12) NA

The deliverable “Annual Progress Report 1” has the delivery date been shifted from

Month 18 to Month 24

NA

Removal of Beneficiary 137. LUni NA

Addition of Affiliated Entities to Beneficiaries: 1. INSERM – 1.1 CAD. NA

Change of PIC from 944342531 to 878333449 for 43-CENTOGENE NA

30.04.2024 Harmonisation of Beneficiaries acronyms from ISCIII-IIIER/ISCIII-SGIPRI to

ISCIII; from CUH (NIHR-RDBR) to CUH; from UTartu to UTARTU; from ICS/

and ICS/VHIR to VHIR; from Astra Zeneca to AZ; from AMC or AMUC to

AUMC; from Uhasselt to UHASSELT; from Centogene to CENTOGENE; from

Copenhagen Economics to CE; from RS (GMS-GMCK) to RS; from MAPI

Research Trust to MRT; from SCI to Sciensano; from GMS-Skane Region to GMS-

RS; from Otago University to UO; from NIHDRJ to INSA; from Rare Care Centre

to RCC; from Sorbonne University to SU; from Motol University to MUH; from