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(11)

EP 4 580 644 A1

(12)

(43) Date of publication: 09.07.2025 Bulletin 2025/28 (21) Application number: 23861156.0 (22) Date of filing: 28.08.2023

(51) International Patent Classification (IPC):  A61K 35/42(2015.01) C12N 5/071(2010.01) C12N 5/074(2010.01) C12N 5/02(2006.01) A61P 11/00(2006.01) (52) Cooperative Patent Classification (CPC): A61K 35/42; A61P 11/00; C12N 5/0688; C12N 2502/27; C12N 2502/1323; C12N 2501/727; C12N 2513/00 (86) International application number: PCT/US2023/031312 (87) International publication number: WO 2024/049773 (07.03.2024 Gazette 2024/10)

(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: 29.08.2022 US 202263402056 P (71) Applicant: Children's Hospital Medical Center Cincinnati, OH 45229-3039 (US)	(72) Inventors: ZACHARIAS, William Cincinnati, Ohio 45229-3039 (US) TOTH, Andrea Cincinnati, Ohio 45229-3039 (US) (74) Representative: Potter Clarkson  Chapel Quarter Mount Street Nottingham NG1 6HQ Nottingham NG1 6HQ (GB)