Alnylam Pharmaceuticals, Inc.
COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
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Abstract:
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement factor B (CFB) gene, the complement component C3 gene, and the complement component C9 gene and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of CFB, C9 and/or C3 and to treat subjects having a complement component-associated disease, e.g., paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.
Status:
Application
Type:
Utility
Filling date:
18 Aug 2021
Issue date:
7 Jul 2022