(19)
(11) EP 4 558 514 A2

(12)

(88) Date of publication A3:
07.03.2024

(43) Date of publication:
28.05.2025 Bulletin 2025/22

(21) Application number: 23843942.6

(22) Date of filing: 21.07.2023
(51) International Patent Classification (IPC): 
C07K 14/47(2006.01)
C12N 15/86(2006.01)
A61K 48/00(2006.01)
C12Q 1/6883(2018.01)
(52) Cooperative Patent Classification (CPC):
C12N 15/86; C12N 2830/008; C12N 2750/14143; C12N 2830/48; C12N 2830/50; C12N 2830/38; A61K 38/00; A61K 48/0058; C12N 2800/22; A61K 48/005; A61K 45/06; A61P 19/00
(86) International application number:
PCT/US2023/070765
(87) International publication number:
WO 2024/020574 (25.01.2024 Gazette 2024/04)
(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: 21.07.2022 US 202263391252 P

(71) Applicant: New York University
New York, NY 10012 (US)

(72) Inventors:
  • ABBADI, Dounia
    New York, NY 10016 (US)
  • SCHNEIDER, Robert, J.
    New York, NY 10003 (US)
  • KARUMUTHIL-MELETHIL, Subha
    Rockville, MD 20850 (US)
  • QIAO, Chunping
    Rockville, MD 20850 (US)
  • ELLIOTT, Kirk
    Rockville, MD 20850 (US)
  • LIU, Ye
    Rockville, MD 20850 (US)
  • DANOS, Olivier
    Rockville, MD 20850 (US)
  • FOLTZ, Steven
    Rockville, MD 20850 (US)

(74) Representative: Keltie LLP 
No. 1 London Bridge
London SE1 9BA
London SE1 9BA (GB)

   


(54) AUF1 GENE THERAPY FOR LIMB GIRDLE MUSCULAR DYSTROPHY