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EP 4 551 245 A1

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(43) Date of publication: 14.05.2025 Bulletin 2025/20 (21) Application number: 23738510.9 (22) Date of filing: 04.07.2023

(51) International Patent Classification (IPC):  A61K 38/42(2006.01) A61P 3/00(2006.01) A61P 25/14(2006.01) C07K 14/47(2006.01) A61K 48/00(2006.01) A61P 25/00(2006.01) C12N 15/86(2006.01) (52) Cooperative Patent Classification (CPC): A61K 48/0075; A61K 38/42; A61P 25/14; A61P 25/00; A61P 3/00; C12N 15/86; C12N 2750/14143; C12N 2840/203; A61K 48/005; C07K 14/805 (86) International application number: PCT/EP2023/068369 (87) International publication number: WO 2024/008709 (11.01.2024 Gazette 2024/02)

(84) Designated Contracting States: AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC ME MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA Designated Validation States: KH MA MD TN (30) Priority: 05.07.2022 EP 22306002 (71) Applicants: Institut National de la Santé et de la Recherche Médicale (INSERM) 75013 Paris (FR) Association Française contre les Myopathies (A.F.M.) 75013 Paris (FR) Université Paris Cité 75006 Paris (FR)

(72) Inventors: CORRAL-DEBRINSKI, Marisol 75019 PARIS (FR) GRESSENS, Pierre 75019 PARIS (FR) (74) Representative: Plasseraud IP  104 Rue de Richelieu CS92104 75080 Paris Cedex 02 75080 Paris Cedex 02 (FR)