Complement component C3 iRNA compositions and methods of use thereof

US12258565B2 · kind B2 · utility

Assignee

• Alnylam Pharmaceuticals, Inc. · US

Inventors

• Mark Keating · Salt Lake City, US
• James D. McIninch · Burlington, US
• Elane Fishilevich · Indianapolis, US
• Kristina Yucius · Burlington, US
• Sarah Solomon · Cambridge, US
• Mark K. Schlegel · Cambridge, US
• Adam Castoreno · Framingham, US
• Charalambos Kaittanis · Cambridge, US

Key dates

• Filing date: Mar 4, 2024
• Grant date: Mar 25, 2025
• Expiry date: Mar 4, 2044

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/351
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

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.