(19)
(11) EP 4 680 745 A2

(12)

(88) Date of publication A3:
07.11.2024

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

(21) Application number: 24775437.7

(22) Date of filing: 15.03.2024
(51) International Patent Classification (IPC): 
C12N 15/113(2010.01)
A61K 31/7088(2006.01)
A61K 9/127(2025.01)
A61K 31/713(2006.01)
A61P 25/28(2006.01)
A61K 48/00(2006.01)
(52) Cooperative Patent Classification (CPC):
A61K 31/711; C12N 15/113; C12N 2310/315; C12N 2310/3515; C12N 2310/14; C12N 2320/11; C12N 2310/343; C12N 2320/32
 
C-Sets:
  1. C12N 2310/321, C12N 2310/3521;
  2. C12N 2310/322, C12N 2310/3533;

(86) International application number:
PCT/US2024/020125
(87) International publication number:
WO 2024/196753 (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 202363490866 P

(71) Applicant: Arrowhead Pharmaceuticals, Inc.
Pasadena, CA 91105 (US)

(72) Inventors:
  • SUK, Ji Young
    San Diego, California 92130 (US)
  • ESAU, Christine
    San Diego, California 92131 (US)
  • LI, Xiaokai
    San Diego, California 92131 (US)
  • LAZZARA, Phillip
    MADISON, Wisconsin 53719 (US)
  • PEI, Tao
    MADISON, Wisconsin 53719 (US)
  • NICHOLAS, Anthony
    Oregon, Wisconsin 53575 (US)
  • CARLSON, Jeffrey
    MADISON, Wisconsin 53719 (US)
  • STEIN, Ivar
    San Diego, California 92131 (US)

(74) Representative: Arnold & Siedsma 
Bezuidenhoutseweg 57
2594 AC The Hague
2594 AC The Hague (NL)

   


(54) RNAI AGENTS FOR INHIBITING EXPRESSION OF ATAXIN-2 (ATXN2), COMPOSITIONS THEREOF, AND METHODS OF USE