US 12,377,150 B2
Exon skipping oligomer conjugates for muscular dystrophy
Marco A. Passini, Cambridge, MA (US); and Gunnar J. Hanson, Cambridge, MA (US)
Assigned to Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed by Sarepta Therapeutics, Inc., Cambridge, MA (US)
Filed on May 26, 2022, as Appl. No. 17/804,280.
Application 17/804,280 is a continuation of application No. 16/469,104, granted, now 11,382,981, previously published as PCT/US2017/066351, filed on Dec. 14, 2017.
Claims priority of provisional application 62/562,119, filed on Sep. 22, 2017.
Claims priority of provisional application 62/479,177, filed on Mar. 30, 2017.
Claims priority of provisional application 62/443,481, filed on Jan. 6, 2017.
Claims priority of provisional application 62/436,199, filed on Dec. 19, 2016.
Prior Publication US 2022/0387601 A1, Dec. 8, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. A61P 21/00 (2006.01); A61K 47/54 (2017.01); A61K 47/64 (2017.01); C12N 15/113 (2010.01)
CPC A61K 47/549 (2017.08) [A61K 47/645 (2017.08); A61P 21/00 (2018.01); C12N 15/113 (2013.01); C12N 2310/11 (2013.01); C12N 2310/3233 (2013.01); C12N 2310/3513 (2013.01); C12N 2320/35 (2013.01)] 18 Claims
 
1. A method for treating Duchenne muscular dystrophy (DMD) in a human subject in need thereof, wherein the human subject has a mutation of the dystrophin gene that is amenable to exon 45 skipping, the method comprising administering to the human subject an antisense oligomer conjugate of Formula (IV):

OG Complex Work Unit Chemistry
or a pharmaceutically acceptable salt thereof.