- 专利标题： COMPLEMENT COMPONENT C3 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
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申请号： US17721530申请日： 2022-04-15
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公开(公告)号： US20220364088A1公开(公告)日： 2022-11-17
- 发明人: Mark Keating , James D. McIninch , Elane Fishilevich , Kristina Yucius , Sarah Solomon , Mark K. Schlegel , Adam Castoreno , Charalambos Kaittanis
- 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
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; C12N15/66
摘要：
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., cold agglutinin disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupis nephritis (LN), bullous pemphigoid, pemphigus, e.g., pemphigus vulgaris (PV) and pemphigus foliaceus (PF), and C3 glomerulopathy.
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