发明申请
US20130190484A1 In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages
有权
具有酶敏感连接的体内多核苷酸递送缀合物
- 专利标题： In Vivo Polynucleotide Delivery Conjugates Having Enzyme Sensitive Linkages
- 专利标题（中）： 具有酶敏感连接的体内多核苷酸递送缀合物
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申请号： US13833814申请日： 2013-03-15
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公开(公告)号： US20130190484A1公开(公告)日： 2013-07-25
- 发明人: David Rozema , David Lewis , Darren Wakefield , Eric Kitas , Philipp Hadwiger , Jon Wolff , Ingo Roehl , Peter Mohr , Torsten Hoffmann , Kerstin Jahn-Hofmann , Hans Martin Mueller , Guenther Ott , Andrei Blokhin , Jeffrey Carlson , Jonathan Benson
- 申请人： David Rozema , David Lewis , Darren Wakefield , Eric Kitas , Philipp Hadwiger , Jon Wolff , Ingo Roehl , Peter Mohr , Torsten Hoffmann , Kerstin Jahn-Hofmann , Hans Martin Mueller , Guenther Ott , Andrei Blokhin , Jeffrey Carlson , Jonathan Benson
- 申请人地址： US WI Madison
- 专利权人： ARROWHEAD MADISON INC.
- 当前专利权人： ARROWHEAD MADISON INC.
- 当前专利权人地址： US WI Madison
- 主分类号： C12N15/113
- IPC分类号： C12N15/113 ; C07C271/20 ; C07C69/96
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.