CN117561334A — 人染色体9开放阅读框72(C9ORF72)iRNA药剂组合物和其使用方法
Assigned to Regeneron Pharmaceuticals Inc · Expires 2024-02-13 · 2y expired
What this patent protects
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。
USPTO Abstract
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。
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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