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′). |
地址 |
Carlsbad CA US |