RNAi Agents for Inhibiting Expression of Beta-ENaC, Compositions Thereof, and Methods of Use

    公开(公告)号:US20230013022A1

    公开(公告)日:2023-01-19

    申请号:US17731766

    申请日:2022-04-28

    IPC分类号: C12N15/113 A61K47/68

    摘要: Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a beta-ENaC (SCNN1B) gene. The beta-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a beta-ENaC gene. Pharmaceutical compositions that include one or more beta-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described beta-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of beta-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including chronic obstructive pulmonary disease (COPD).

    RNAi Agents for Inhibiting Expression of Mucin 5AC (MUC5AC), Compositions Thereof, and Methods of Use

    公开(公告)号:US20230002767A1

    公开(公告)日:2023-01-05

    申请号:US17824841

    申请日:2022-05-25

    IPC分类号: C12N15/113 A61P11/12

    摘要: Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Mucin 5AC (MUC5AC) gene. The MUC5AC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an MUC5AC gene. Pharmaceutical compositions that include one or more MUC5AC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MUC5AC RNAi agents to pulmonary epithelial cells, in vivo, provides for inhibition of MUC5AC gene expression and a reduction in MUC5AC production, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including mucoobstructive lung disease such as severe asthma and various cancers.