申请人:ALNYLAM PHARMACEUTICALS, INC.
公开号:US09061063B2
公开(公告)日:2015-06-23
The present invention relates to a cationic lipid having one or more biodegradable groups located in 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,其中具有一个或多个可生物降解的基团位于脂质部分(例如,疏水链)中。这些阳离子脂质可以被纳入脂质粒中,以传递活性剂,例如核酸。该发明还涉及脂质粒,包括中性脂质、能够减少聚集的脂质、本发明中的阳离子脂质和可选的固醇。脂质粒还可以包括治疗剂,例如核酸。