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 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.
Claims8
A compound of formula (II),
- wherein
- L1 is selected from C4-C24 alkyl and C4-C24 alkenyl, with the proviso that each of the C4-C24 alkyl and the C4-C24 alkenyl contain at least one cyclopropyl group;
- said C4-C24 alkyl and said C4-C24 alkenyl are further optionally substituted with one or more substituents selected from R′, 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 (C1-C6) alkyl is optionally substituted with halogen or OH;
- or any pharmaceutically acceptable salt or stereoisomer thereof.
Dependencies are inferred from the claim text.