发明公开
EP3312281A2 COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
审中-公开
补体成分C5 IRNA组合物及其使用方法
- 专利标题: COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
- 专利标题(中): 补体成分C5 IRNA组合物及其使用方法
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申请号: EP17187047.0申请日: 2014-03-13
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公开(公告)号: EP3312281A2公开(公告)日: 2018-04-25
- 发明人: FITZGERALD, Kevin , BUTLER, James , BETTENCOURT, Brian , BORODOVSKY, Anna , KUCHIMANCHI, Satyanarayana , CHARISSE, Klaus , MANOHARAN, Muthiah , MAIER, Martin , RAJEEV, Kallanthottahil G.
- 申请人: Alnylam Pharmaceuticals, Inc.
- 申请人地址: 300 Third Street, 3rd Floor Cambridge, MA 02142 US
- 专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人地址: 300 Third Street, 3rd Floor Cambridge, MA 02142 US
- 代理机构: Hoffmann Eitle
- 优先权: US201361782531P 20130314; US201361837399P 20130620; US201361904579P 20131115; US201361912777P 20131206; US201461942367P 20140220
- 主分类号: C12N15/113
- IPC分类号: C12N15/113 ; A61K31/7088
摘要:
The invention relates to iRNA, e.g. , double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g. , paroxysmal nocturnal hemoglobinuria.
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