发明名称 Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
摘要 The present disclosure describes oligomeric compounds having at least one bicyclic nucleoside attached to the 3′ or 5′ termini by a neutral internucleoside linkage and methods of using the oligomeric compounds. In some embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
申请公布号 US9290534(B2) 申请公布日期 2016.03.22
申请号 US200912934498 申请日期 2009.04.03
申请人 Ionis Pharmaceuticals, Inc. 发明人 Seth Punit P.;Swayze Eric E.
分类号 C07H21/00 主分类号 C07H21/00
代理机构 McNeil Baur PLLC 代理人 McNeil Baur PLLC
主权项 1. A gapped oligomeric compound comprising a contiguous sequence of linked monomeric subunits having two external regions and an internal region including one neutral internucleoside linkage covalently attaching a 3′ or 5′-terminal bicyclic nucleoside to the gapped oligomeric compound wherein the gapped oligomeric compound comprises from 12 to 16 linked monomeric subunits and is complementary to at least a portion of a target RNA wherein said neutral internucleoside linkage is a phosphotriester, methylphosphonate, MMI (3′-CH2—N(CH3)—O-5′), amide-3 (3′-CH2—C(═O)—N(H)-5′), formacetal (3′-O—CH2—O-5′) or a thioformacetal (3′-S—CH2—O-5′).
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