公开(公告)号：EP2838877A4

公开(公告)日：2016-01-27

申请号：EP13778145

申请日：2013-04-16

申请人： SIRNA THERAPEUTICS INC

发明人： COLLETI STEVEN L ,  STANTON MATTHEW G

IPC分类号： C07C229/12 ,  A61K9/51 ,  C07C229/28 ,  C07C229/30 ,  C12N15/113

CPC分类号： A61K9/5123 ,  A61K9/51 ,  A61K9/5146 ,  A61K31/713 ,  A61K47/48215 ,  A61K47/60 ,  C07C229/12 ,  C07C229/28 ,  C07C229/30 ,  C07C2101/02 ,  C07C2601/02 ,  C07F7/1852 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2320/32 ,  C12N2320/51

摘要： The instant invention provides for novel cationic lipids of Formula A that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhances efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.

摘要翻译： 本发明提供用于式A确实可以与其他脂质组分组合使用的新型阳离子脂质：如胆固醇和PEG-脂质以形成用寡核苷酸的脂质纳米颗粒。 它是本发明的一个目的是提供阳离子脂质支架没有增强的效力地连同较低的肝毒性如在肝中低的脂质水平的结果恶魔trates。 本发明采用的低分子量阳离子脂质与在脂链加上列入水解功能上，以增强siRNA在体内递送的效率性和耐受一个短脂质链。