Title:
MODIFIED siRNA FOR SELECTIVELY INHIBITING EXPRESSION OF MUTANT FUS
Document Type and Number:
WIPO Patent Application WO/2023/171587
Kind Code:
A1
Abstract:
[Problem] To provide a chemically-modified siRNA that has improved stability against RNase enzymes and selectively inhibits the expression of P525L mutant FUS which is a causative gene of ALS. [Solution] Provided is a chemically-modified siRNA comprising a sense strand and an antisense strand, or a salt thereof, wherein: the antisense strand includes a complementary or substantially complementary region in a portion of a mRNA that codes P525L mutant FUS protein; the complementary region has a nucleotide length of 19 to 21; and the siRNA contains at least one substitution selected from the group consisting of a 2'-F- nucleotide, a 2'-OMe-nucleotide, a nucleotide in which a 2'-O atom and a 4'-O atom are crosslinked by methylene, a 2'-deoxy-nucleotide, and a phosphorothioate bond that forms an internucleotide bond.
Inventors:
MORITA NAOKI (JP)
HASHISEKO KAEDE (JP)
OKADA SUMIRE (JP)
YAMAMOTO YOSHINOBU (JP)
HASHISEKO KAEDE (JP)
OKADA SUMIRE (JP)
YAMAMOTO YOSHINOBU (JP)
Application Number:
PCT/JP2023/008197
Publication Date:
September 14, 2023
Filing Date:
March 06, 2023
Export Citation:
Assignee:
OHARA PHARMACEUTICAL CO LTD (JP)
International Classes:
A61K31/712; C12N15/113; A61K31/7125; A61K31/713; A61P21/02
Domestic Patent References:
| WO2018185253A1 | 2018-10-11 | |||
| WO2019193189A1 | 2019-10-10 | |||
| WO2022054801A1 | 2022-03-17 |
Foreign References:
| KR20150094311A | 2015-08-19 | |||
| JP2015520742A | 2015-07-23 |
Attorney, Agent or Firm:
Sha Patent and Trademark Firm (JP)
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