- 专利标题： COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
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申请号： EP17187047.0申请日： 2014-03-13
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公开(公告)号： EP3312281A3公开(公告)日： 2018-06-27
- 发明人: FITZGERALD, Kevin , BUTLER, James , BETTENCOURT, Brian , BORODOVSKY, Anna , KUCHIMANCHI, Satyanarayana , CHARISSE, Klaus , MANOHARAN, Muthiah , MAIER, Martin , RAJEEV, Kallanthottahil G. , FOSTER, Donald
- 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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