(19)
(11) EP 4 798 727 A1

(12)

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

(21) Application number: 24798444.6

(22) Date of filing: 24.10.2024
(51) International Patent Classification (IPC): 
C12N 15/86(2006.01)
(52) Cooperative Patent Classification (CPC):
C12N 15/86; C12N 2740/16043; C12N 2830/40; C12N 2830/00; C12N 15/90; A61K 48/0066
(86) International application number:
PCT/EP2024/080150
(87) International publication number:
WO 2025/088076 (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 EP 23205639

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

(72) Inventors:
  • MONTINI, Eugenio
    20132 Milan (IT)
  • CESANA, Daniela
    20132 Milan (IT)
  • VOLPIN, Monica
    20132 Milan (IT)

(74) Representative: D Young & Co LLP 
3 Noble Street
London EC2V 7BQ
London EC2V 7BQ (GB)

   


(54) POLYNUCLEOTIDES