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48 条记录 (耗时 0.018 秒)

    COMPOUNDS AND METHODS FOR MODULATION OF SMN2
    2.
    发明公开
    COMPOUNDS AND METHODS FOR MODULATION OF SMN2 审中-公开

    公开(公告)号：EP4206213A1

    公开(公告)日：2023-07-05

    申请号：EP22206076.6

    申请日：2017-07-17

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： RIGO, Frank ,  PRAKASH, Thazha, P. ,  SETH, Punit, P.

    IPC分类号： C07H21/02 ,  A61K48/00 ,  A61P21/00 ,  A61P21/02 ,  A61P25/28 ,  A61P43/00

    摘要： Disclosed herein are compounds, compositions and compounds for use in modulating splicing of SMN2, said compounds consisting in oligomers comprising a modified oligonucleotide consisting of 14-25 linked nucleosides, wherein the modified oligonucleotide is complementary to an SMN2 pre-mRNA; and wherein at least one nucleoside of the modified oligonucleotide comprises a 2'-O(N-alkyl acetamide) modified sugar moiety. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of spinal muscular atrophy.

    COMPOSITIONS AND METHODS FOR MODULATING TAU EXPRESSION
    3.
    发明公开
    COMPOSITIONS AND METHODS FOR MODULATING TAU EXPRESSION 审中-公开

    公开(公告)号：EP3766974A1

    公开(公告)日：2021-01-20

    申请号：EP20181501.6

    申请日：2014-03-14

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： RIGO, Frank ,  BENNETT, C., Frank

    IPC分类号： C12N15/113 ,  C12Q1/68

    摘要： Disclosed are methods for modulating splicing of Tau mRNA in an animal with Tau antisense compounds. Also disclosed herein are methods for reducing expression of Tau mRNA and protein in an animal with Tau antisense compounds. Such compounds and methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration Tau antisense oligonucleotides include Alzheimer's Disease, Fronto-temporal Dementia (FTD), FTDP-17, Progressive Supranuclear Palsy, Chronic Traumatic Encephalopathy, Epilepsy, and Dravet's Syndrome.

    COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION
    9.
    发明公开
    COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION 审中-公开

    公开(公告)号：EP4166667A3

    公开(公告)日：2023-08-02

    申请号：EP22197003.1

    申请日：2014-10-11

    申请人： Ionis Pharmaceuticals, Inc.

    发明人： FREIER, Susan, M. ,  RIGO, Frank ,  SINGH, Priyam

    IPC分类号： C12N15/11 ,  A61K48/00 ,  C07H21/02 ,  C07H21/04

    摘要： Disclosed herein are compositions and methods for reducing expression of C9ORF72 mRNA and protein in an animal with C9ORF72 specific inhibitors. Also disclosed herein are compositions and methods of selectively inhibiting a C9ORF72 pathogenic associated mRNA variant by administering an antisense compound targeting the region beginning at the start site of exon 1A to the start site of exon IB of a C9ORF72 pre-mRNA. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 specific inhibitors include antisense compounds.