CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE

    公开(公告)号:EP4257691A2

    公开(公告)日:2023-10-11

    申请号:EP23181919.4

    申请日:2018-12-14

    IPC分类号: C12N15/63

    摘要: The present disclosure provides half duplex compounds comprising a first oligomeric compound and a second, shorter, oligomeric compound, wherein the first oligomeric compound is complementary to a target nucleic acid and the second oligomeric compound is complementary to the first oligomeric compound. In certain emobodiments, the compounds disclosed herein are useful for modulating the expression of extra-hepatic target nucleic acids.

    LINKAGE MODIFIED OLIGOMERIC COMPOUNDS
    8.
    发明公开

    公开(公告)号:EP3647318A1

    公开(公告)日:2020-05-06

    申请号:EP19191250.0

    申请日:2015-04-28

    摘要: The present invention provides oligomeric compounds comprising at least one neutral methoxypropyl phosphonate modified internucleoside linkage. Such oligomeric compounds have one or more improved properties such as selectivity, potency, improved toxicity profile and or improved proinflammatory profile. Such oligomeric compounds have enhanced stability to exposure to base during synthesis. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.