Human chromosome 9 open reading frame 72 (C9ORF72) iRNA pharmaceutical compositions and methods of use thereof

• Main Authors: DANG LY THI THANH, SHAMAH-CANNING, ABDULLAH, SAVAGE BERNARD, FRENDEWEY DAVID, MCININCH JAMES D, NGUYEN, TRUC MINH
• Format: Patent
• Language: Chinese; English
• Published: 13.02.2024
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; ENZYMOLOGY; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; VINEGAR; WINE

The present disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions that target the human chromosome 9 open reading frame 72 (C9orf72) gene as well as methods of using such dsRNAi agents and compositions to inhibit expression of the C9orf72 gene and methods of treating diseases or disorders associated with C9orf72, e.g., diseases or disorders associated with C9orf72. The present invention relates to a method for treating a subject suffering from C9orf72 amyotrophic lateral sclerosis, frontotemporal dementia, or Huntington-Like Syndrome due to C9orf72 amplification, or a subject suffering from C9orf72 amyotrophic lateral sclerosis, frontotemporal dementia, or a Huntington-Like Syndrome due to C9orf72 amplification. 本公开涉及靶向人染色体9开放阅读框72(C9orf72)基因的双链核糖核酸(dsRNAi)药剂和组合物以及使用此类dsRNAi药剂和组合物来抑制C9orf72基因的表达的方法和治疗患有C9orf72相关疾病或病症，例如，C9orf72肌萎缩性侧索硬化症、额颞叶痴呆或由于C9orf72扩增引起的亨廷顿样综合征(Huntington-Like Syndrome)的受试者的方法。