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68 条记录 (耗时 0.061 秒)

    COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION
    2.
    发明申请
    COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION 有权

    公开(公告)号：US20220177893A1

    公开(公告)日：2022-06-09

    申请号：US17437507

    申请日：2020-03-13

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： Huynh-Hoa Bui ,  Susan M. Freier

    IPC分类号： C12N15/113

    摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of KCNT1 RNA in a cell or subject, and in certain instances reducing the amount of KCNT1 protein in a cell or subject. These compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurological condition. Such symptoms and hallmarks include seizures, encephalopathy, and behavioral abnormalities. Non-limiting examples of neurological conditions that benefit from these compounds, methods, and pharmaceutical compositions are epilepsy of infancy with migrating focal seizures (EIMFS), autosomal dominant nocturnal frontal lobe epilepsy (ADNFLE), West syndrome, and Ohtahara syndrome.

    COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION
    4.
    发明申请
    COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION 有权

    公开(公告)号：US20210252039A1

    公开(公告)日：2021-08-19

    申请号：US16973652

    申请日：2019-06-14

    申请人： Ionis Pharmaceuticals, Inc. ,  Ludwig Institute For Cancer Research

    发明人： Huynh-Hoa Bui ,  Don W. Cleveland ,  Ze'ev Melamed

    IPC分类号： A61K31/7125 ,  C12N15/113

    摘要： Provided are compounds, methods, and pharmaceutical compositions for increasing the amount or activity of STMN2 RNA in a cell or animal, and in certain embodiments increasing the amount of STMN2 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include ataxia, neuropathy, synaptic dysfunction, deficits in cognition, and decreased longevity. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), Alzheimer's disease (AD), and dementia with Lewy bodies (DLB).

    Compounds and methods for reducing FXI expression
    5.
    发明授权
    Compounds and methods for reducing FXI expression 有权

    公开(公告)号：US11021710B2

    公开(公告)日：2021-06-01

    申请号：US17091891

    申请日：2020-11-06

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： Huynh-Hoa Bui

    IPC分类号： C12N15/113

    摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of FXI RNA in a cell or subject, and in certain instances reducing the amount of FXI protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to prevent, treat, or ameliorate at least one symptom of a thromboembolic condition without a significant increase in a bleeding risk. Such thromboembolic conditions include deep vein thrombosis, venous or arterial thrombosis, pulmonary embolism, myocardial infarction, stroke, thrombosis associated with chronic kidney disease or end-stage renal disease (ESRD), including thrombosis associated with dialysis, or other procoagulant condition. Such symptoms include decreased blood flow through an affected vessel, death of tissue, and death.