Novel low molecular weight cationic lipids for oligonucleotide delivery
AssigneeSirna Therapeutics, Inc.
InventorsMatthew G. Stanton, Brian W. Budzik, Gregory L. Beutner, Hongbiao Liao
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Abstract
The instant invention provides for novel catiomc 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.
Claims13
-10. (canceled)
A compound of formula (II),
- wherein
- L1 is selected from C4-C24 alkyl and C4-C24 alkenyl, said alkyl and alkenyl are optionally substituted with one or more substituents selected from le, wherein
- R′ is independently selected from halogen, R″, OR″, SR″, CN, CO2R″ or CON(R″)2;
- and
- R″ is independently selected from H and (C1-C6)alkyl, wherein said alkyl is optionally substituted with halogen or OH.
Dependencies are inferred from the claim text.