	US 11,655,472 B2
	Antisense nucleic acid that induces skipping of exon 50
Yukiko Enya, Tsukuba (JP); Yuta Sunadoi, Tsukuba (JP); Reiko Waki, Tsukuba (JP); Kaname Muchima, Tsukuba (JP); Shin'ichi Takeda, Tokyo (JP); and Yoshitsugu Aoki, Tokyo (JP)
Assigned to NIPPON SHINYAKU CO., LTD., Kyoto (JP); and NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY, Kodaira (JP)
Filed by NIPPON SHINYAKU CO., LTD., Kyoto (JP); and NATIONAL CENTER OF NEUROLOGY AND PSYCHIATRY, Kodaira (JP)
Filed on Jun. 23, 2022, as Appl. No. 17/847,333.
Application 17/847,333 is a continuation of application No. PCT/JP2020/048803, filed on Dec. 25, 2020.
Claims priority of application No. JP2019-236704 (JP), filed on Dec. 26, 2019.
Prior Publication US 2022/0333112 A1, Oct. 20, 2022
Int. Cl. C07H 21/04 (2006.01); C12N 15/113 (2010.01); A61P 21/00 (2006.01); A61K 31/4965 (2006.01); A61K 31/7088 (2006.01)

CPC C12N 15/113 (2013.01) [A61K 31/4965 (2013.01); A61K 31/7088 (2013.01); A61P 21/00 (2018.01); C12N 2310/11 (2013.01); C12N 2310/314 (2013.01); C12N 2310/321 (2013.01); C12N 2310/3233 (2013.01); C12N 2320/33 (2013.01)]

17 Claims

1. An antisense oligomer consisting of the base sequence of SEQ ID NO: 4 or SEQ ID NO: 5, or a pharmaceutically acceptable salt or hydrate thereof, wherein the antisense oligomer is (i) an oligonucleotide comprising at least one nucleotide having a modified sugar moiety or a modified phosphate bond moiety, or (ii) a morpholino oligomer.