发明名称 COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
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.
申请公布号 WO2014160129(A2) 申请公布日期 2014.10.02
申请号 WO2014US25882 申请日期 2014.03.13
申请人 ALNYLAM PHARMACEUTICALS, INC. 发明人 FITZGERALD, KEVIN;BUTLER, JAMES;BETTENCOURT, BRIAN;BORODOVSKY, ANNA;KUCHIMANCHI, SATYANARAYANA;CHARISSE, KLAUS;MANOHARAN, MUTHIAH;MAIER, MARTIN;RAJEEV, KALLANTHOTTATHIL, G.;FOSTER, DONALD
分类号 C12N15/113 主分类号 C12N15/113
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