STANTON, Matthew, G.,BUDZIK, Brian, W.,COLLETTI, Steven, L.
申请号：
DK13767382
公开号：
DK2830594T3
申请日:
2013.03.25
申请国别(地区):
DK
年份:
2018
代理人:
摘要：
Disclosed herein are 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. The cationic lipids can demonstrate 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 coupled with inclusion of hydrolysable functionality in the lipid chains to enhance the efficiency and tolerability of in vivo delivery of siRNA.
