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US 2017/0000815 A1Published application
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.
Claims25
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,
- the dsRNA consists of a sense strand and an antisense strand, and the sense strand comprises a first sequence and the antisense strand comprises a second sequence complementary to at least 15 contiguous nucleotides of a nucleotide sequence of a target sequence of a dsRNA found in Table 1a, Table 2a, Table 5a, Table 6, Table 7, Table 8,
- wherein the first sequence is complementary to the second sequence and wherein the dsRNA is between 15 and 30 base pairs in length.
-9. (canceled)
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-33. (canceled)
-43. (canceled)
Dependencies are inferred from the claim text.