REGULATOR OF EXPRESSION AND/OR FUNCTION OF SCN1A GENE

WIPO Patent Application WO/2021/187540

A1

A single-stranded antisense oligonucleotide or pharmaceutically acceptable salt thereof for promoting expression and/or regulating functioning of the voltage-dependent sodium channel α1 subunit gene, wherein the single-stranded antisense oligonucleotide includes a gap region, a 3' wing region bonded to the 3' end of the gap region, and a 5' wing region bonded to the 5' end of the gap region, the gap region being a 5-20 mer nucleic acid in which the sugar part is deoxyribose, the 3' wing region and the 5' wing region each being a 1-5 mer modified nucleic acid, and the modified nucleic acid in each of the 3' wing region and the 5' wing region including at least one selected from the group consisting of AmNA, GuNA, and scpBNA.

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KURITA MITSUMASA (JP)
ASANO SHIGEHIRO (JP)
SEKIGUCHI KAZUO (JP)
KOBORI TAKURO (JP)
SUZUKI TAKAO (JP)
YAMAGAMI MASAKI (JP)
SHRESTHA AJAYA RAM (JP)
SATO HIDEAKI (JP)
UMEMOTO TADASHI (JP)

PCT/JP2021/010901

September 23, 2021

March 17, 2021

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SUMITOMO DAINIPPON PHARMA CO LTD (JP)
LUXNA BIOTECH CO LTD (JP)

C12N15/113; A61K31/711; A61P25/00; A61P43/00

WO2020027227A1	2020-02-06
WO2019009298A1	2019-01-10
WO2018139679A1	2018-08-02

JP2013535958A	2013-09-19
JP2014525446A	2014-09-29

FUKAMI PATENT OFFICE, P.C. (JP)

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