LI, Zhen,ZHU, Rui,PEI, Tao,NICHOLAS, Anthony,BUSH, Erik W.
申请号：
PH12019502660
公开号：
PH12019502660A1
申请日:
2019.11.25
申请国别(地区):
PH
年份:
2020
代理人:
摘要：
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of an alpha-ENaC (SCNN1A) gene. The alpha-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an alpha-ENaC gene. Pharmaceutical compositions that include one or more alpha-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described alpha-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of alpha-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects.
