LIPID FORMULATIONS COMPRISING CATIONIC LIPID AND A TARGETING LIPIC COMPRISING N-ACETYL GALACTOSAMINE FOR DELIVERY OF NUCLEIC ACID

摘要

The invention features an improved lipid formulation comprising a cationiclipid of formula (A), a neutral lipid, asterol and a PEG or PEG-modified lipid, where R1 and R2 are independentlyalkyl, alkenyl or alkynyl, each can be optionallysubstituted, and R3 and R4 are independently lower alkyl or R3 and R4 can betaken together to form an optionally subsitutedheterocyclic ring. In one embodiment, R1 and R2 are independently selectedfrom oleoyl, pamitoyl, steroyl, linoleyl and R3 and R4 aremethyl. Also disclosed are targeting lipids, and specific lipid formulationscomprising such targeting lipids.