(19)
(11) EP 4 739 307 A2

(12)

(88) Date of publication A3:
01.05.2025

(43) Date of publication:
13.05.2026 Bulletin 2026/20

(21) Application number: 24836549.6

(22) Date of filing: 02.07.2024
(51) International Patent Classification (IPC): 
A61K 31/395(2006.01)
A61K 31/33(2006.01)
A61K 31/4709(2006.01)
(52) Cooperative Patent Classification (CPC):
C07D 471/04; C07D 519/00; A61K 31/437
(86) International application number:
PCT/US2024/036596
(87) International publication number:
WO 2025/010291 (09.01.2025 Gazette 2025/02)
(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: 03.07.2023 US 202363524837 P
14.12.2023 US 202363610275 P

(71) Applicant: The General Hospital Corporation
Boston, Massachusetts 02114 (US)

(72) Inventors:
  • HAGGARTY, Stephen J.
    Gloucester, Massachusetts 01930 (US)
  • ROSENTHAL, Zachary
    Cambridge, Massachusetts 02138 (US)
  • MAZITSCHEK, Ralph
    Weston, Massachusetts 02493 (US)
  • PATNAIK, Debasis
    Weymouth, Massachusetts 02191 (US)
  • PAYNE, Neil
    Boston, Massachusetts 02114 (US)
  • HOOKER, Jacob M.
    Belmont, Massachusetts 02478 (US)
  • PACZEK, Michael
    Boston, Massachusetts 02114 (US)
  • FASS, Daniel M.
    Boston, Massachusetts 02114 (US)

(74) Representative: Maiwald GmbH 
Engineering Elisenhof Elisenstrasse 3
80335 München
80335 München (DE)

   


(54) TARGETED EPIGENETIC THERAPIES FOR DISORDERS OF PROGRANULIN DEFICIENCY