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    MODIFIED OLIGONUCLEOTIDES
    5.
    发明申请
    MODIFIED OLIGONUCLEOTIDES 有权

    公开(公告)号：US20220389419A1

    公开(公告)日：2022-12-08

    申请号：US17767190

    申请日：2020-10-13

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Muthiah MANOHARAN ,  Pawan KUMAR ,  Dhrubajyoti DATTA

    IPC分类号： C12N15/11 ,  A61K31/713 ,  C12N15/113

    摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. Other aspects of the invention relate to pharmaceutical compositions comprising these dsRNA molecules suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA molecules, e.g., for the treatment of various disease conditions.

    MODIFIED DOUBLE-STRANDED RNA AGENTS
    8.
    发明申请
    MODIFIED DOUBLE-STRANDED RNA AGENTS 有权

    公开(公告)号：US20210017519A1

    公开(公告)日：2021-01-21

    申请号：US16693683

    申请日：2019-11-25

    申请人： ALNYLAM PHARMACEUTICALS, INC.

    发明人： Martin MAIER ,  Don FOSTER ,  Stuart MILSTEIN ,  Satya KUCHIMANCHI ,  Vasant JADHAV ,  Kallanthottathil RAJEEV ,  Muthiah MANOHARAN ,  Rubina PARMAR

    IPC分类号： C12N15/113 ,  C12N15/11

    摘要： One aspect of the present invention relates to double-stranded RNA (dsRNA) agent capable of inhibiting the expression of a target gene. The sense strand of the dsRNA agent comprises at least one thermally destabilizing nucleotide, and at least one said thermally destabilizing nucleotide occurring at a site opposite to the seed region (positions 2-8) of the antisense strand; and the antisense strand of the dsRNA agent comprises at least two modified nucleotides that provide the nucleotide a steric bulk that is less than or equal to the steric bulk of a 2′-OMe modification, wherein said modified nucleotides are separated by 11 nucleotides in length. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.

    iRNA AGENTS WITH BIOCLEAVABLE TETHERS
    9.
    发明申请
    iRNA AGENTS WITH BIOCLEAVABLE TETHERS 审中-公开

    公开(公告)号：US20180327744A1

    公开(公告)日：2018-11-15

    申请号：US16042633

    申请日：2018-07-23

    申请人： Alnylam Pharmaceuticals, Inc.

    发明人： Muthiah MANOHARAN ,  Kallanthottathil G. RAJEEV

    IPC分类号： C12N15/113 ,  C12N15/11

    CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/323 ,  C12N2310/3511 ,  C12N2320/51

    摘要： The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.