发明名称 Modified iRNA agents
摘要 Abstract The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose. The inclusion of such 5 a monomer can allow for modulation of a property of the iRNA agent into which it is incorporated e.g., by using the non-ribose moiety as a point to which a ligand or other entity, e.g., a lipophilic moiety e.g., cholesterol, is directly, or indirectly, tethered. The invention also relates to methods of making and using such modified iRNA agents. 69105941 (GHMatters) P58751.AU.5 PETERB 17/09/15 w O==P-O 0- tethering attachment point (R-2) backbone attachment points carrier LIGAND RRMS o tether O=P-O' w FG' 0 .R=H; A: carrier R R= R = ('w -- )+ (LIGAND) B: carrier R C: - E R'O N(R")2
申请公布号 AU2017201200(A1) 申请公布日期 2017.03.16
申请号 AU20170201200 申请日期 2017.02.22
申请人 Alnylam Pharmaceuticals, Inc. 发明人 MANOHARAN, Muthiah
分类号 C07D473/14;A61K31/70;A61K48/00;C07H21/02;C07H21/04;C12Q1/68 主分类号 C07D473/14
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