US 11,814,622 B2
OPA1 antisense oligomers for treatment of conditions and diseases
Isabel Aznarez, Boston, MA (US); Aditya Venkatesh, Natick, MA (US); and Gene Liau, Wayland, MA (US)
Assigned to STOKE THERAPEUTICS, INC., Bedford, MA (US)
Filed by Stoke Therapeutics, Inc., Bedford, MA (US)
Filed on Apr. 21, 2023, as Appl. No. 18/304,878.
Application 18/304,878 is a continuation of application No. 17/924,966, previously published as PCT/US2021/030254, filed on Apr. 30, 2021.
Claims priority of provisional application 63/112,458, filed on Nov. 11, 2020.
Claims priority of provisional application 63/023,013, filed on May 11, 2020.
Prior Publication US 2023/0250429 A1, Aug. 10, 2023
Int. Cl. C12N 15/113 (2010.01); A61P 27/02 (2006.01); A61K 9/00 (2006.01); C12N 15/86 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 9/0048 (2013.01); A61P 27/02 (2018.01); C12N 15/86 (2013.01); C12N 2310/11 (2013.01); C12N 2310/20 (2017.05); C12N 2310/314 (2013.01); C12N 2310/315 (2013.01); C12N 2310/321 (2013.01); C12N 2750/14143 (2013.01)] 21 Claims
 
1. A pharmaceutical composition comprising:
(a) a pharmaceutically acceptable excipient; and
(b) a therapeutic agent, wherein the therapeutic agent comprises an antisense oligomer that comprises the sequence set forth in any one of SEQ ID NO: 36, 92-96, or 166-168, and wherein the antisense oligomer comprises a 2′-O-methoxyethyl moiety.