Complement component C3 iRNA compositions and methods of use thereof

• Main Authors: SCHLEGEL MARK K, SOLOMON, STEVEN, CASTORENO, ANDREA, KEATING MARK, MCININCH JAMES D, YUSSIUS, KARIN, FISHLEVICH, ERIC, KATANIS CHARALAMPOS
• Format: Patent
• Language: Chinese; English
• Published: 11.10.2022
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

The present invention relates to RNAi agents, e.g., double-stranded RNA (dsRNA) agents, that target the complement component C3 gene (C3). The invention also relates to methods of using such RNAi agents to inhibit C3 gene expression and methods of preventing and treating C3-related conditions, such as condensation set disease (CAD), warm autoimmune hemolytic anemia, and paroxysmal nocturnal hemoglobinuria (PNH), lupus nephritis (LN), bullous pemphigus, pemphigus, such as pemphigus vulgaris (PV) and pemphigus leaf (PF), and C3 glomerulopathy. 本发明涉及靶向补体成分C3基因(C3)的RNAi剂，例如，双链RNA(dsRNA)剂。本发明还涉及使用此类RNAi剂抑制C3基因表达的方法以及预防和治疗C3相关病症的方法，例如，冷凝集素病(CAD)，温性自身免疫性溶血性贫血，和阵发性夜间血红蛋白尿(PNH)，狼疮肾炎(LN)，大疱性类天疱疮，天疱疮，例如寻常型天疱疮(PV)和叶型天疱疮(PF))，以及C3肾小球病。