| 发明名称 |
In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages |
| 摘要 |
The present invention is directed compositions for delivery of RNA interference (RNAi) polynucleotides to cells in vivo. The compositions comprise amphipathic membrane active polyamines reversibly modified with enzyme cleavable dipeptide-amidobenzyl-carbonate masking agents. Modification masks membrane activity of the polymer while reversibility provides physiological responsiveness. The reversibly modified polyamines (dynamic polyconjugate or DPC) are further covalently linked to an RNAi polynucleotide or co-administered with a targeted RNAi polynucleotide-targeting molecule conjugate.
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| 申请公布号 |
US2012172412(A1) |
申请公布日期 |
2012.07.05 |
| 申请号 |
US201113336028 |
申请日期 |
2011.12.23 |
| 申请人 |
ROZEMA DAVID B.;WAKEFIELD DARREN H.;LEWIS DAVID L.;WOLFF JON A.;BLOKHIN ANDREI V.;BENSON JONATHAN D.;CARLSON JEFFREY C.;HADWIGER PHILIPP;KITAS ERIC A.;HOFFMANN TORSTEN;JAHN-HOFMANN KERSTIN;MOHR PETER;MUELLER HANS MARTIN;OTT GUENTHER;ROEHL INGO;ARROWHEAD MADISON INC. |
发明人 |
ROZEMA DAVID B.;WAKEFIELD DARREN H.;LEWIS DAVID L.;WOLFF JON A.;BLOKHIN ANDREI V.;BENSON JONATHAN D.;CARLSON JEFFREY C.;HADWIGER PHILIPP;KITAS ERIC A.;HOFFMANN TORSTEN;JAHN-HOFMANN KERSTIN;MOHR PETER;MUELLER HANS MARTIN;OTT GUENTHER;ROEHL INGO |
| 分类号 |
A61K48/00;C07C69/96;C08G63/91 |
主分类号 |
A61K48/00 |
| 代理机构 |
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