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(11)

EP 4 680 730 A2

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(88) Date of publication A3: 05.06.2025 (43) Date of publication: 21.01.2026 Bulletin 2026/04 (21) Application number: 24775466.6 (22) Date of filing: 15.03.2024

(51) International Patent Classification (IPC):  C12N 5/0793(2010.01) A61P 25/00(2006.01) C07K 14/05(2006.01) A61K 35/12(2015.01) A61P 35/00(2006.01) (52) Cooperative Patent Classification (CPC): A61K 35/30; A61K 35/17; A61P 35/00; C07K 2317/31; C07K 16/2863; C07K 16/2803; C07K 16/2809; C07K 2317/622; C07K 16/3084; C07K 2317/73; A61K 40/10; A61K 40/4204; A61K 2239/13; A61K 2239/27; A61K 2039/5156 (86) International application number: PCT/US2024/020245 (87) International publication number: WO 2024/196794 (26.09.2024 Gazette 2024/39)

(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: 17.03.2023 US 202363490856 P (71) Applicant: The Children's Hospital Of Philadelphia Philadelphia, PA 19104 (US)	(72) Inventors: DE RAEDT, Thomas Philadelphia, PA 19146 (US) ANDERSON, Stewart Philadelphia, PA 19747 (US) BROSIUS, Stephanie Cheltenham, PA 19012 (US) MANLEY, William Philadelphia, PA 19139 (US) (74) Representative: Potter Clarkson  Chapel Quarter Mount Street Nottingham NG1 6HQ Nottingham NG1 6HQ (GB)