Inventors:
Muthiah MANOHARAN - Cambridge MA, US
Martin Maier - Cambridge MA, US
Muthusamy Jayaraman - Cambridge MA, US
Shigeo Matsuda - Cambridge MA, US
Akin Akinc - Cambridge MA, US
Thomas A. Baillie - Seattle WA, US
Assignee:
ALNYLAM PHARMACEUTICALS, INC. - Cambridge MA
International Classification:
A61K 47/44, A61K 31/713, A61P 37/04, C12N 5/071, C12N 15/85, C07C 229/30, A61K 31/7088
US Classification:
4242831, 554110, 514785, 514 44 A, 514 44 R, 435375, 435455
Abstract:
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.