(19)
(11) EP 4 798 253 A1

(12)

(43) Date of publication:
02.09.2026 Bulletin 2026/36

(21) Application number: 24883241.2

(22) Date of filing: 23.10.2024
(51) International Patent Classification (IPC): 
A61K 48/00(2006.01)
C07K 14/005(2006.01)
C12N 7/00(2006.01)
A61P 25/28(2006.01)
C12N 15/86(2006.01)
C12N 9/64(2006.01)
(52) Cooperative Patent Classification (CPC):
A61P 25/28; C07K 14/005; C12N 15/86; C12N 2750/14145; C12N 2750/14143; C12N 2810/40; C12N 2750/14122; A01K 2227/105; A01K 2217/075; C12N 9/485
(86) International application number:
PCT/US2024/052555
(87) International publication number:
WO 2025/090598 (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 202363592318 P

(71) Applicant: The Children's Hospital of Philadelphia
Philadelphia, PA 19104 (US)

(72) Inventors:
  • RANUM, Paul T.
    Philadelphia, Pennsylvania 19104 (US)
  • DAVIDSON, Beverly L.
    Philadelphia, Pennsylvania 19104 (US)
  • KEISER, Megan S.
    Philadelphia, Pennsylvania 19106 (US)
  • CHEN, Yonghong
    Philadelphia, Pennsylvania 19104 (US)
  • TECEDOR, Luis
    Philadelphia, Pennsylvania 19104 (US)

(74) Representative: Potter Clarkson 
Chapel Quarter Mount Street
Nottingham NG1 6HQ
Nottingham NG1 6HQ (GB)

   


(54) AAV VECTORS FOR TREATMENT OF CLN2 DISEASE