(19)

(11)

EP 4 798 718 A1

(12)

(43) Date of publication: 02.09.2026 Bulletin 2026/36 (21) Application number: 24883511.8 (22) Date of filing: 28.10.2024

(51) International Patent Classification (IPC):  C12N 15/113(2010.01) A61K 31/7125(2006.01) A61P 25/28(2006.01) A61K 31/712(2006.01) A61K 31/713(2006.01) (52) Cooperative Patent Classification (CPC): C12N 15/113; A61K 31/712; A61K 31/713; A61K 31/7125; A61P 25/28; C12N 2320/33; C12N 2310/11; C12N 2310/315; C12N 2310/322   C-Sets: C12N 2310/322, C12N 2310/3533; (86) International application number: PCT/US2024/053253 (87) International publication number: WO 2025/091028 (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: 27.10.2023 US 202363593746 P (71) Applicants: The Regents of The University of Michigan Ann Arbor, Michigan 48109 (US) Rosalind Franklin University of Medicine and Science North Chicago, IL 60064 (US) Ionis Pharmaceuticals, Inc. Carlsbad, CA 92010 (US)

(72) Inventors: HASTINGS, Michelle L. Ann Arbor, Michigan 48109 (US) CENTA, Jessica Chicago, Illinois 60064 (US) STRATTON, Matthew Chicago, Illinois 60064 (US) JAFAR-NEJAD, Paymaan Carlsbad, California 92010 (US) HUNYARA, John Carlsbad, California 92010 (US) (74) Representative: HGF  HGF Limited 4th Floor, 1 City Square Leeds LS1 2ES Leeds LS1 2ES (GB)