[EN] AMINO LIPID BASED IMPROVED LIPID FORMULATION<br/>[FR] FORMULATION LIPIDIQUE AMÉLIORÉE À BASE D'AMINO LIPIDE
申请人:ALNYLAM PHARMACEUTICALS INC
公开号:WO2009132131A1
公开(公告)日:2009-10-29
The present invention provides compositions and methods for the delivery of therapeutic agents to cells. In particular, these include novel lipids and nucleic acid-lipid particles that provide efficient encapsulation of nucleic acids and efficient delivery of the encapsulated nucleic aicd to cells in vivo. The compositions of the present invention are highly potent, thereby allowing effective knock-down of specific target protein at relatively low doses. In addition, the compositions and methods of the present invention are less toxic and provide a greater therapeutic index compared to compositions and methods previously known in the art. Formulae (I), (II), (III), (IV).
本发明提供了将治疗剂传递给细胞的组合物和方法。特别是,这些包括新型脂质和核酸-脂质颗粒,它们能够高效地封装核酸并将封装的核酸有效地传递到体内细胞中。本发明的组合物非常强效,因此可以在相对较低的剂量下有效地降低特定靶蛋白的表达。此外,与先前已知的技术中的组合物和方法相比,本发明的组合物和方法毒性更低,疗效指数更高。公式(I)、(II)、(III)、(IV)。