(19)
(11) EP 4 680 250 A1

(12)

(43) Date of publication:
21.01.2026 Bulletin 2026/04

(21) Application number: 24775476.5

(22) Date of filing: 15.03.2024
(51) International Patent Classification (IPC): 
A61K 31/7105(2006.01)
A61P 7/00(2006.01)
C12N 15/113(2010.01)
A61K 9/00(2006.01)
C07K 14/755(2006.01)
C12Q 1/6883(2018.01)
(52) Cooperative Patent Classification (CPC):
A61K 31/7105; A61P 7/00; C07K 14/755; C12N 15/113; A61K 9/0019; A61K 9/08; C12N 2310/315; C12N 2310/11; C12N 2320/33; C12N 2310/321
 
C-Sets:
C12N 2310/321, C12N 2310/3525;
(86) International application number:
PCT/US2024/020275
(87) International publication number:
WO 2024/196804 (26.09.2024 Gazette 2024/39)
(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: 17.03.2023 US 202363453036 P

(71) Applicant: The Regents of the University of California
Oakland, CA 94607-5200 (US)

(72) Inventors:
  • SANFORD, Jeremy
    Santa Cruz, California 95064 (US)
  • STONE, Michael
    Santa Cruz, California 95064 (US)
  • TSE, Victor
    Santa Cruz, California 95064 (US)
  • CHACALTANA, Guillermo
    Santa Cruz, California 95064 (US)

(74) Representative: Boult Wade Tennant LLP 
Salisbury Square House 8 Salisbury Square
London EC4Y 8AP
London EC4Y 8AP (GB)

   


(54) FACTOR VIII SPLICE-MODULATING ANTISENSE OLIGONUCLEOTIDES AND METHODS OF USE