STANTON, Matthew G.,BUDZIK, Brian W.,BEUTNER, Gregory L.,LIAO, Hongbiao
申请号：
AU2018202688
公开号：
AU2018202688A1
申请日:
2018.04.18
申请国别(地区):
AU
年份:
2018
代理人:
摘要：
#$%^&*AU2018202688A120180510.pdf#####NOVEL LOW MOLECULAR WEIGHT CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY ABSTRACT The instant invention provides for novel cationic lipids that can be used in combination with other lipid components such as cholesterol and PEG-lipids to form lipid nanoparticles with oligonucleotides. It is an object of the instant invention to provide a cationic lipid scaffold that demonstrates enhanced efficacy along with lower liver toxicity as a result of lower lipid levels in the liver. The present invention employs low molecular weight cationic lipids with one short lipid chain to enhance the efficiency and tolerability of in vivo delivery of siRNA.
