Search International and National Patent Collections

1. (WO2018123925) THERAPEUTIC DRUG FOR ALPORT'S SYNDROME

Pub. No.:    WO/2018/123925    International Application No.:    PCT/JP2017/046304
Publication Date: Fri Jul 06 01:59:59 CEST 2018 International Filing Date: Tue Dec 26 00:59:59 CET 2017
IPC: C12N 15/113
A61K 31/713
A61K 48/00
A61P 13/12
Applicants: DAIICHI SANKYO COMPANY, LIMITED
第一三共株式会社
NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY
国立大学法人神戸大学
Inventors: IIJIMA, Kazumoto
飯島 一誠
NOZU, Kandai
野津 寛大
SHONO, Akemi
庄野 朱美
KOIZUMI, Makoto
小泉 誠
ONISHI, Yoshiyuki
大西 朗之
TAKAISHI, Kiyosumi
高石 巨澄
ADACHI, Tomomi
足立 朝美
Title: THERAPEUTIC DRUG FOR ALPORT'S SYNDROME
Abstract:
The purpose of the present invention is to establish a molecular therapy for Alport's syndrome. Provided are the following: a 15 to 30 base-long oligonucleotide formed of a nucleotide sequence complimentary to cDNA of COL4A5 gene, the oligonucleotide having a truncating mutation in COL4A5 gene of an Alport's syndrome patient, and being able to induce the skipping of an exon where the number of bases is a multiple of 3; a pharmaceutically acceptable salt thereof, or a solvate thereof; and a pharmaceutical drug (therapeutic drug for Alport's syndrome) containing the oligonucleotide, a pharmaceutically acceptable salt thereof, or a solvate thereof.