SNCA iRNA compositions for treating or preventing SNCA-related neurodegenerative diseases and methods of use thereof

• Main Authors: SCHLEGEL MARK K, TANG LOK THI HUA, SUNDARAPANDIAN MANOJ MOHAN, CASTORENO, ANDREA, MCKINNING JAMES D, KETTANIS CHRISTOS
• Format: Patent
• Language: Chinese; English
• Published: 05.09.2023
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES

The present disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions thereof that target SNCA genes, and methods of using such dsRNAi agents and compositions to inhibit expression of SNCA genes, and methods of treating individuals having SNCA-associated neurodegenerative diseases or disorders, the neurodegenerative disease or condition is, for example, Parkinson's disease (PD), multi-system atrophy, Lewis body dementia (LBD), and other synucleinopathies. 本公开内容是关于靶向SNCA基因的双股核糖核酸(dsRNAi)剂及其组成物，以及使用此类dsRNAi剂及组成物抑制SNCA基因的表达的方法、以及治疗患有SNCA相关神经退化性疾病或病症个体的方法，所述神经退化性疾病或病症是例如：帕金森病(PD)、多系统萎缩、路易氏体失智(LBD)以及其他突触核蛋白病。