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US 2025/0084412 A1Published application
Antisense Nucleic Acids
AssigneesNippon Shinyaku Co., Ltd., National Center of Neurology and Psychiatry
InventorsNaoki Watanabe, Yuuichirou Tone, 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. The present invention provides an antisense oligomer wherein two or more unit oligomers targeting sequences that are neither consecutive nor overlap with each other in the same exon are connected.
Claims23
An antisense oligomer having a length of 15 to 30 bases wherein
- (a) a first unit oligomer comprising a nucleotide sequence complementary to a first nucleotide sequence of 7 to 15 consecutive bases in a target exon in human dystrophin gene;
- and
- (b) a second unit oligomer comprising a nucleotide sequence complementary to a second nucleotide sequence of 7 to 15 consecutive bases in the target exon
- are connected, wherein
- the first nucleotide sequence and the second nucleotide sequence are neither consecutive nor overlap with each other, and
- the antisense oligomer induces skipping of the target exon, or a pharmaceutically acceptable salt or hydrate thereof.
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A method for screening of an antisense oligomer, which comprises:
- (a) selecting
- (i) a first unit oligomer comprising a nucleotide sequence complementary to a first nucleotide sequence of 7 to 15 consecutive bases in a target exon in human dystrophin gene;
- and
- (ii) a second unit oligomer comprising a nucleotide sequence complementary to a second nucleotide sequence of 7 to 15 consecutive bases in the target exon, wherein
- the first nucleotide sequence and the second nucleotide sequence are neither consecutive nor overlap with each other;
- (b) connecting the first and second unit oligomers to produce an antisense oligomer having a length of 15 to 30 bases;
- (c) measuring the efficiency of skipping by the antisense oligomer obtained in the step (b);
- and
- (d) selecting an antisense oligomer having the efficiency of skipping that exceeds a reference value.
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Dependencies are inferred from the claim text.