[EN] LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS<br/>[FR] LIPIDES POUR L'ADMINISTRATION D'AGENTS ACTIFS
申请人:ALNYLAM PHARMACEUTICALS INC
公开号:WO2013086373A1
公开(公告)日:2013-06-13
The present invention relates to novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides, to facilitate the cellular uptake and endosomal escape, and to knockdown target mRNA both in vitro and in vivo. The invention also relates to lipid particles comprising a neutral lipid, a lipid capable of reducing aggregation, a cationic lipid of the present invention, and optionally, a sterol. The lipid particle may further include a therapeutic agent such as a nucleic acid.