(19)
(11) EP 4 797 953 A1

(12)

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

(21) Application number: 24805633.5

(22) Date of filing: 23.10.2024
(51) International Patent Classification (IPC): 
A01K 67/0271(2024.01)
C12N 9/22(2006.01)
C12N 5/0789(2010.01)
C12N 15/113(2010.01)
(52) Cooperative Patent Classification (CPC):
A01K 67/0271; A01K 2207/12; A01K 2207/15; C12N 5/0647; C12N 2510/00; A01K 2227/105; A01K 2267/0306; A01K 2267/0387; C12N 15/113; C12N 9/93; C12N 15/1137; C12N 2310/11; A61K 35/28; C12N 2310/20; A61P 7/00
(86) International application number:
PCT/IB2024/060412
(87) International publication number:
WO 2025/088508 (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: 24.10.2023 IT 202300022191

(71) Applicants:
  • Fondazione Telethon ETS
    00185 Roma (IT)
  • Ospedale San Raffaele S.r.l.
    20132 Milano (MI) (IT)

(72) Inventors:
  • FERRARI, Samuele
    20100 Milano (IT)
  • NALDINI, Luigi
    20100 Milano (IT)
  • FIUMARA, Martina
    20100 Milano (IT)
  • MOLTENI, Raffaella
    20100 Milano (IT)
  • CAVALLI, Giulio
    20100 Milano (IT)

(74) Representative: Manfredi, Irene 
ADV IP S.r.l. Corso di Porta Vittoria, 29
20122 Milano
20122 Milano (IT)

   


(54) MEANS AND METHODS FOR GENETIC MODIFICATION OF UBA1