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EP 3 952 923 A1

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(43) Date of publication: 16.02.2022 Bulletin 2022/07 (21) Application number: 20787456.1 (22) Date of filing: 10.04.2020

(51) International Patent Classification (IPC):  A61K 48/00(2006.01) A61K 38/00(2006.01) C07H 21/04(2006.01) C12N 15/11(2006.01) C12N 15/00(2006.01) (52) Cooperative Patent Classification (CPC): A61K 38/00; A61K 9/0085; A01K 2227/105; A01K 2207/20; A01K 2267/0318; C12N 2750/14143; C12N 15/86; A61K 48/005; A61K 9/0019; C12N 2310/14; C12N 2320/31; C12N 2330/51; C12N 15/113; C12Y 302/01045; C12N 9/2402; C07K 14/475; C07K 14/705 (86) International application number: PCT/US2020/027658 (87) International publication number: WO 2020/210615 (15.10.2020 Gazette 2020/42)

(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 MK MT NL NO PL PT RO RS SE SI SK SM TR Designated Extension States: BA ME Designated Validation States: KH MA MD TN (30) Priority: 10.04.2019 US 201962831856 P 10.04.2019 US 201962831840 P 10.04.2019 US 201962831846 P 10.04.2019 US 201962832223 P 12.11.2019 US 201962934450 P 27.12.2019 US 201962954089 P 13.01.2020 US 202062960471 P 12.03.2020 US 202062998665 P 16.03.2020 US 202062990246 P (71) Applicant: Prevail Therapeutics, Inc. New York, NY 10016 (US)

(72) Inventors: ABELIOVICH, Asa New York, NY 10016 (US) HECKMAN, Laura New York, NY 10016 (US) RHINN, Herve New York, NY 10016 (US) (74) Representative: Mewburn Ellis LLP  Aurora Building Counterslip Bristol BS1 6BX Bristol BS1 6BX (GB)