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575 条记录 (耗时 0.071 秒)

    COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
    5.
    发明申请
    COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION 有权

    公开(公告)号：US20230043648A1

    公开(公告)日：2023-02-09

    申请号：US17802324

    申请日：2021-02-26

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： Paymaan Jafar-Nejad ,  Susan M. Freier ,  Frank Rigo

    IPC分类号： C12N15/113 ,  C07H21/00 ,  A61K31/712 ,  A61K31/7115 ,  A61K31/7125

    摘要： Provided are compounds, methods, and pharmaceutical compositions for modulating SCN1A RNA and/or protein in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom of a developmental or epileptic encephalopathic disease, such as Dravet Syndrome. Such symptoms include seizures, sudden unexpected death in epilepsy, status epilepticus, behavioral and developmental delays, movement and balance dysfunctions, orthopedic conditions, motor and cognitive dysfunctions, delayed language and speech issues, visual motor integration dysfunctions, visual perception dysfunctions, executive dysfunctions, growth and nutrition issues, sleeping difficulties, chronic infections, sensory integration disorders, and dysautonomia.

    SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
    6.
    发明申请
    SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF 有权

    公开(公告)号：US20230002763A1

    公开(公告)日：2023-01-05

    申请号：US17586175

    申请日：2022-01-27

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： Michael Oestergaard ,  Punit P. Seth ,  Eric E. Swayze

    IPC分类号： C12N15/113 ,  C07H21/02

    摘要： The present invention provides oligomeric compounds. 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. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

    Compounds and methods for modulating ATXN1
    7.
    发明授权
    Compounds and methods for modulating ATXN1 有权

    公开(公告)号：US11542504B2

    公开(公告)日：2023-01-03

    申请号：US17751450

    申请日：2022-05-23

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： Keming Zhou

    IPC分类号： C07H21/04 ,  C12N15/113 ,  A61P25/00 ,  A61K31/712 ,  A61K35/30 ,  A61K47/02

    摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of ATXN1 RNA in a cell or subject, and in certain instances reducing the amount of ATXN1 in a cell or subject. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include gait and limb ataxia, cognitive impairments, difficulty with speaking and swallowing, atrophy of the cerebellum and brainstem in magnetic resonance imaging (MRI), neurochemical abnormalities in the cerebellum and brainstem detected via magnetic resonance spectroscopy (MRS), and death within 10-15 years of symptom onset. Such neurodegenerative diseases include Spinocerebellar ataxia type 1.

    COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION
    9.
    发明申请
    COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION 有权

    公开(公告)号：US20220380773A1

    公开(公告)日：2022-12-01

    申请号：US17424672

    申请日：2020-01-29

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： Susan M. Freier ,  Huynh-Hoa Bui ,  Holly Kordasiewicz ,  Eric E. Swayze

    IPC分类号： C12N15/113

    摘要： Provided are compounds, methods, and pharmaceutical compositions for reducing the amount or activity of APP RNA in a cell or animal, and in certain instances reducing the amount of APP protein in a cell or animal Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include cognitive impairment, including a decline in memory and language skills, behavioral and psychological symptoms such as apathy and lack of motivation, gait disturbances and seizures, progressive dementia, and abnormal amyloid deposits.