[EN] BRANCHED ALKYL AND CYCLOALKYL TERMINATED BIODEGRADABLE LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS<br/>[FR] LIPIDES BIODÉGRADABLES RAMIFIÉS À TERMINAISONS ALKYLE ET CYCLOALKYLE DESTINÉS À L'ADMINISTRATION D'AGENTS ACTIFS
申请人:ALNYLAM PHARMACEUTICALS INC
公开号:WO2013086322A1
公开(公告)日:2013-06-13
The present invention relates to a cationic lipid of formula (I) 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.
本发明涉及具有一个或多个生物可降解基团的阳离子脂质的化学式(I),所述生物可降解基团位于阳离子脂质的脂质部分(例如,疏水链)中。这些阳离子脂质可以被纳入脂质粒子中,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粒子。脂质粒子还可以进一步包括治疗剂,例如核酸。