发明公开
EP2838877A4 NOVEL DIESTER AND TRIESTER BASED LOW MOLECULAR WEIGHT, BIODEGRADABLE CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY 审中-公开
新颖,可生物降解的,阳离子脂质ON二酯和低分子以发行OLIGONUCLEOTIDES三BASE

NOVEL DIESTER AND TRIESTER BASED LOW MOLECULAR WEIGHT, BIODEGRADABLE CATIONIC LIPIDS FOR OLIGONUCLEOTIDE DELIVERY
摘要:
The instant invention provides for novel cationic lipids of Formula A 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 enhances 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.
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