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

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.
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