Transthyretin (TTR) iRNA compositions and methods of use thereof

• Main Authors: SCHLEGEL MARK K, CASTORENO, ANDREA, MCININCH JAMES D
• Format: Patent
• Language: Chinese; English
• Published: 29.03.2024
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; DERIVATIVES THEREOF; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NUCLEIC ACIDS; NUCLEOSIDES; NUCLEOTIDES; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; SUGARS; VINEGAR; WINE

The present invention relates to RNAi agents, such as double-stranded RNA (dsRNA) agents, that target the transthyretin (TTR) gene. The invention also relates to methods of inhibiting expression of TTR genes using such RNAi agents, as well as methods of preventing and treating TTR-related conditions, the present invention relates to compositions and methods for treating diseases such as senile systemic amyloidosis (SSA), systemic familial amyloidosis, familial amyloid polyneuropathy (FAP), familial amyloid cardiomyopathy (FAC), cormeria/central nervous system (CNS) amyloidosis, and hyperthyroxinemia. 本发明涉及靶向转甲状腺素蛋白(TTR)基因的RNAi药剂，例如双链RNA(dsRNA)药剂。本发明还涉及使用此类RNAi药剂抑制TTR基因的表达的方法，以及预防和治疗TTR相关病症的方法，所述病症例如老年性系统性淀粉样变性(SSA)、系统性家族性淀粉样变性、家族性淀粉样多发性神经病(FAP)、家族性淀粉样心肌病(FAC)、软脑膜/中枢神经系统(CNS)淀粉样变性和高甲状腺素血症。