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US 8,273,869 B2Granted
Lipid formulated dsRNA targeting the PCSK9 gene
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsKevin Fitzgerald, Gregory Hinkle, Akin Akinc, Stuart Milstein
Life of this patent
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Abstract
This invention relates to composition and methods using lipid formulated siRNA targeted to a PCSK9 gene.
Claims27
A composition comprising a nucleic acid lipid particle comprising a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a human PCSK9 gene in a cell, wherein:
- the nucleic acid lipid particle comprises a lipid formulation comprising 45-65 mol % of a cationic lipid, 5 mol % to about 10 mol %, of a non-cationic lipid, 25-40 mol % of a sterol, and 0.5-5 mol % of a PEG or PEG-modified lipid and the cationic lipid comprises MC3 (((6Z,9Z,28Z,31Z)-heptatriaconta-6,9,28,31-tetraen-19-yl 4-(dimethylamino)butanoate),
- the dsRNA consists of a sense strand and an antisense strand,
- wherein the sense strand consists of the nucleotide sequence of SEQ ID NO:1227 and the antisense strand consists of the nucleotide sequence of SEQ ID NO:1228.
Dependencies are inferred from the claim text.