发明名称 iRNA AGENTS WITH BIOCLEAVABLE TETHERS
摘要 The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking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.
申请公布号 US2013281685(A1) 申请公布日期 2013.10.24
申请号 US201313849017 申请日期 2013.03.22
申请人 ALNYLAM PHARMACEUTICALS, INC. 发明人 MANOHARAN MUTHIAH;RAJEEV KALLANTHOTTATHIL G.
分类号 C12N15/113;A61K31/70;A61K48/00;C07D473/14;C07H21/02;C12Q1/68 主分类号 C12N15/113
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