- 专利标题: Complement component C5 iRNA compositions and methods of use thereof
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申请号: US15151568申请日: 2016-05-11
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公开(公告)号: US09701963B2公开(公告)日: 2017-07-11
- 发明人: Kevin Fitzgerald , James Butler , Brian Bettencourt , Anna Borodovsky , Satyanarayana Kuchimanchi , Klaus Charisse , Muthiah Manoharan , Martin Maier , Kallanthottathil G. Rajeev , Donald Foster
- 申请人: Alnylam Pharmaceuticals, Inc.
- 申请人地址: US MA Cambridge
- 专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人: Alnylam Pharmaceuticals, Inc.
- 当前专利权人地址: US MA Cambridge
- 代理机构: McCarter & English, LLP
- 代理商 Maria Laccotripe Zacharakis; Deborah L. Nagle
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; C07H21/02 ; C07H21/04 ; C12N15/11 ; C12N15/113 ; A61K31/713 ; A61K39/395 ; C07K16/18
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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