Akinc, Akin,Querbes, William,Wong, Frances,Dorkin, Joseph Robert,Qin, Xiaojun,Cantley, William,Borodovsky, Anna,De, Soma,Manoharan, Muthiah,Jayaraman, Muthusamy,Rajeev, Kallanthottathil G.
申请号：
AU2016228298
公开号：
AU2016228298A1
申请日:
2016.09.16
申请国别(地区):
AU
年份:
2016
代理人:
摘要：
#$%^&*AU2016228298A120161006.pdf#####IMPROVED LIPID FORMULATION FOR THE DELIVERY OF NUCLEIC ACIDS Abstract: The invention features an improved lipid formulation comprising a cationic lipid of formula (A), a neutral lipid, a sterol and a PEG or PEG-modified lipid, where R 1 and R 2 are independently alkyl, alkenyl or alkynyl, each can be optionally substituted, and R 3 and R 4 are independently lower alkyl or R 3 and R 4 can be taken together to form an optionally subsituted heterocyclic ring. In one embodiment, R 1 and R 2 are independently selected from oleoyl, pamitoyl, steroyl, linoleyl and R 3 and R 4 are methyl. Also disclosed are targeting lipids, and specific lipid formulations comprising such targeting lipids. R3 N-R4 (A) o 0 1j>； R2
