(19)

(11)

EP 4 798 170 A1

(12)

(43) Date of publication: 02.09.2026 Bulletin 2026/36 (21) Application number: 24883275.0 (22) Date of filing: 23.10.2024

(51) International Patent Classification (IPC):  A61K 9/127(2025.01) C12N 15/88(2006.01) A61K 47/55(2017.01) A61K 48/00(2006.01) C12N 15/113(2010.01) A61K 31/7088(2006.01) A61K 47/60(2017.01) (52) Cooperative Patent Classification (CPC): C12N 15/88; A61K 48/0025; A61K 48/0041; A61K 48/0091; A61K 31/711; A61K 47/549; A61K 47/6929; A61K 47/62; A61K 45/06; A61K 9/5123 (86) International application number: PCT/US2024/052650 (87) International publication number: WO 2025/090663 (01.05.2025 Gazette 2025/18)

(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: GE KH MA MD TN (30) Priority: 24.10.2023 US 202363592852 P 01.12.2023 WO PCT/US2023/082037 18.09.2024 US 202463696269 P (71) Applicant: Generation Bio Co. Cambridge, Massachusetts 02142 (US)

(72) Inventors: STANTON, Mattthew, G. Cambridge, MA 02142 (US) ROSE, Douglas, A. Cambridge, MA 02142 (US) SHAH, Sachit Cambridge, MA 02142 (US) SYROVATKINA, Viktoriya Cambridge, MA 02142 (US) (74) Representative: Maiwald GmbH  Elisenhof Elisenstraße 3 80335 München 80335 München (DE)