- 专利标题: Therapeutic drug for Alport's syndrome
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申请号: US16467224申请日: 2017-12-25
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公开(公告)号: US11230709B2公开(公告)日: 2022-01-25
- 发明人: Kazumoto Iijima , Kandai Nozu , Akemi Shono , Makoto Koizumi , Yoshiyuki Onishi , Kiyosumi Takaishi , Tomomi Adachi
- 申请人: DAIICHI SANKYO COMPANY, LIMITED , NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY
- 申请人地址: JP Tokyo; JP Hyogo
- 专利权人: DAIICHI SANKYO COMPANY, LIMITED,NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY
- 当前专利权人: DAIICHI SANKYO COMPANY, LIMITED,NATIONAL UNIVERSITY CORPORATION KOBE UNIVERSITY
- 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: JPJP2016-254906 20161228,JPJP2017-077374 20170410
- 国际申请: PCT/JP2017/046304 WO 20171225
- 国际公布: WO2018/123925 WO 20180705
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K31/713 ; A61P13/12
摘要:
The present invention aims at establishing a molecular therapy for Alport syndrome. The present invention provides an oligonucleotide of 15-30 bp comprising a nucleotide sequence complementary to the cDNA of COL4A5 gene, wherein the oligonucleotide is capable of inducing skipping of an exon which has a truncating mutation found in COL4A5 gene in Alport syndrome patients and whose nucleotide number is a multiple of 3, a pharmaceutically acceptable salt thereof, or a solvate thereof. Also provided is a pharmaceutical drug comprising the above oligonucleotide, a pharmaceutically acceptable salt thereof, or a solvate thereof (therapeutic drug for Alport syndrome).
公开/授权文献
- US20200080080A1 THERAPEUTIC DRUG FOR ALPORT'S SYNDROME 公开/授权日:2020-03-12
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