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EP 4 798 616 A1

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(43) Date of publication: 02.09.2026 Bulletin 2026/36 (21) Application number: 24883252.9 (22) Date of filing: 23.10.2024

(51) International Patent Classification (IPC):  C07K 14/775(2006.01) A61K 38/16(2006.01) C07K 14/005(2006.01) C07K 7/06(2006.01) C12N 7/00(2006.01) A61K 48/00(2006.01) C12N 15/86(2006.01) (52) Cooperative Patent Classification (CPC): A61K 38/16; C07K 7/06; C07K 14/005; C07K 14/775; C12N 15/86; C12N 2750/14143; C12N 2750/14145; C12N 2750/14122; C12N 2810/40; C12N 2830/008; A61K 48/005; A61K 48/0058; A01K 2217/075; A01K 2227/105; A61P 25/28 (86) International application number: PCT/US2024/052596 (87) International publication number: WO 2025/090631 (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: 23.10.2023 US 202363592359 P (71) Applicants: The Children's Hospital of Philadelphia Philadelphia, PA 19104 (US) The General Hospital Corporation Boston, Massachusetts 02114 (US)

(72) Inventors: RANUM, Paul T. Philadelphia, Pennsylvania 19104 (US) DAVIDSON, Beverly L. Philadelphia, Pennsylvania 19104 (US) KEISER, Megan S. Philadelphia, Pennsylvania 19104 (US) CHEN, Yonghong Philadelphia, Pennsylvania 19104 (US) CARRELL, Ellie Philadelphia, Pennsylvania 19104 (US) TECEDOR, Luis Philadelphia, Pennsylvania 19104 (US) JACKSON, Rosemary Boston, Massachusetts 02114 (US) HYMAN, Bradley Boston, Massachusetts 02114 (US) (74) Representative: Potter Clarkson  Chapel Quarter Mount Street Nottingham NG1 6HQ Nottingham NG1 6HQ (GB)