- 专利标题: Therapeutic agent for glycogen storage disease type IA
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申请号: US16966132申请日: 2019-03-05
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公开(公告)号: US11958878B2公开(公告)日: 2024-04-16
- 发明人: Makoto Koizumi , Yoshiyuki Onishi , Takeshi Masuda , Mitsuhiro Iwamoto , Yukiko Sekiguchi , Kentaro Ito , Shinnosuke Tsuji , Masafumi Matsuo
- 申请人: DAIICHI SANKYO COMPANY, LIMITED , KOBE GAKUIN EDUCATIONAL FOUNDATION
- 申请人地址: JP Tokyo
- 专利权人: Daiichi Sankyo Company, Limited,Kobe Gakuin Educational Foundation
- 当前专利权人: Daiichi Sankyo Company, Limited,Kobe Gakuin Educational Foundation
- 当前专利权人地址: JP Tokyo; JP Hyogo
- 代理机构: Wenderoth, Lind & Ponack, L.L.P.
- 优先权: JP 18043524 2018.03.09 JP 18128015 2018.07.05
- 国际申请: PCT/JP2019/008713 2019.03.05
- 国际公布: WO2019/172286A 2019.09.12
- 进入国家日期: 2020-07-30
- 主分类号: C07H21/02
- IPC分类号: C07H21/02
摘要:
The present invention establishes a molecular therapy for glycogen storage disease type Ia. The present invention provides an oligonucleotide of 15-30 bases comprising a nucleotide sequence complementary to die cDNA of G6PC gene with c.648G>T mutation, wherein the oligonucleotide comprises a sequence complementary to a region comprising any site between the 82nd to the 92nd nucleotide from the 5′ end of exon 5 of the G6PC gene with c.648C>T mutation, a pharmacologically acceptable salt or solvate thereof. Also provided is a pharmaceutical drug comprising the oligonucleotide, a pharmacologically acceptable salt or solvate thereof (e.g., therapeutic drug for glycogen storage disease type Ia).
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