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US 2025/0145997 A1Published application
Antisense Nucleic Acids
AssigneesNippon Shinyaku Co., Ltd., National Center of Neurology and Psychiatry
InventorsTatsushi Wakayama, Haruna Seo, Youhei Satou, Shin'ichi Takeda, Tetsuya Nagata
Life of this patent
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Abstract
Provided is a drug that allows highly efficient skipping of exon 51 in the human dystrophin gene. The present invention provides an antisense oligomer which enables exon 51 in the human dystrophin gene to be skipped.
Claims13
1-21. (canceled)
A pharmaceutical composition for the treatment of muscular dystrophy, comprising (i) an antisense oligomer consisting of the nucleobase sequence of SEQ ID NO:
- 1 or SEQ ID NO:
- 2, or a pharmaceutically acceptable salt or hydrate thereof;
- and (ii) a pharmaceutically acceptable additive.
Dependencies are inferred from the claim text.