(19)
(11) EP 4 798 254 A1

(12)

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

(21) Application number: 24883447.5

(22) Date of filing: 25.10.2024
(51) International Patent Classification (IPC): 
A61K 48/00(2006.01)
C07H 21/04(2006.01)
C12N 15/00(2006.01)
C07K 14/00(2006.01)
(52) Cooperative Patent Classification (CPC):
C12N 15/86; C12N 2750/14143; A61K 48/005; A61P 1/00; C12N 9/0014; C12Y 104/04002
(86) International application number:
PCT/US2024/053073
(87) International publication number:
WO 2025/090942 (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: 26.10.2023 US 202363593346 P

(71) Applicant: University of Notre Dame du Lac
South Bend, IN 46617 (US)

(72) Inventors:
  • HALDAR, Kasturi
    South Bend, Indiana 46617 (US)
  • PADMANABHAN, Prasad
    South Bend, Indiana 46617 (US)
  • ALAM, Md. Suhail
    South Bend, Indiana 46617 (US)
  • BALI, Adviti
    South Bend, Indiana 46617 (US)
  • LOPEZ RAMIREZ, Alejandro
    South Bend, Indiana 46617 (US)
  • CALHOUN, Shaun
    South Bend, Indiana 46617 (US)
  • BICKERTON, Caroline
    South Bend, Indiana 46617 (US)

(74) Representative: FRKelly 
Waterways House Grand Canal Quay
Dublin D02 PD39
Dublin D02 PD39 (IE)

   


(54) GENE THERAPY FOR TREATMENT OF NEUROMETABOLIC DISEASE