发明名称 Organic Compositions to Treat Beta-ENaC-Related Diseases
摘要 The present disclosure relates to RNAi agents useful in methods of treating BetaENaC-related diseases such as cystic fibrosis, pseudohypoaldosteronism type 1 (PHA1), Liddle's syndrome, hypertension, alkalosis, hypokalemia, and obesity-associated hypertension, using a therapeutically effective amount of a RNAi agent to Beta-ENaC.
申请公布号 US2016272979(A1) 申请公布日期 2016.09.22
申请号 US201615166576 申请日期 2016.05.27
申请人 Arrowhead Pharmaceuticals, Inc. 发明人 De Fougerolles Antonin;Diener John;Hickman Emma;Hinkle Gregory;Milstein Stuart;Pulichino Anne-Marie;Sprague Andrew Griffin
分类号 C12N15/113 主分类号 C12N15/113
代理机构 代理人
主权项 1. A composition comprising a RNAi agent comprising a first strand and a second strand, wherein: the sequence of the first strand comprises the sequence of nt 1-19 of any sequence disclosed in Table 1; wherein the length of the first and second strand are each no more than about 30 nucleotides; and wherein the first and/or second strand is modified or unmodified.
地址 Pasadena CA US