申请人:ALNYLAM PHARMACEUTICALS, INC.
公开号:US20160009637A1
公开(公告)日:2016-01-14
The present invention relates to a cationic lipid having one or more biodegradable groups located in the mid- or distal section of a lipidic moiety (e.g., a hydrophobic chain) of the cationic lipid. These cationic lipids may be incorporated into a lipid particle for delivering an active agent, such as a nucleic acid. 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.
本发明涉及一种阳离子脂质,其具有位于脂质基团(例如,疏水链)的中部或远端部位的一个或多个可生物降解的基团。这些阳离子脂质可以被纳入脂质颗粒中,用于传递活性剂,例如核酸。该发明还涉及包括中性脂质、能够减少聚集的脂质、本发明的阳离子脂质以及可选地固醇的脂质颗粒。脂质颗粒还可以进一步包含治疗剂,如核酸。