Kevin Fitzgerald,James Butler,Brian Bettencourt,Anna Borodovsky,Satyanarayana Kuchimanchi,Klaus Charisse,Muthiah Manoharan,Martin A. Maier,Kallanthottathil G. Rajeev,Donald Foster
申请号：
US16404862
公开号：
US20200032259A1
申请日:
2019.05.07
申请国别(地区):
US
年份:
2020
代理人:
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.
