Irna compositions and methods for silencing complement component 3 (c3)
AssigneeAlnylam Pharmaceuticals, Inc.
InventorJoseph Barry
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Abstract
The present invention relates to RNAi agents, e.g., double stranded RNA (dsRNA) agents, targeting a complement component C3 (C3) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a C3 gene and to methods of preventing and treating a C3-associated disorder, e.g., paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), atypical hemolytic uremic syndrome (aHUS), neuromyelitis optica (NMO), multifocal motor neuropathy (MMN), myasthenia gravis (MG), C3 glomerulonephritis, or systemic lupus erythmatosis.
Claims30
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement component C3 (C3) in a cell, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region,
- (a) wherein the antisense strand comprises a region of complementarity to an mRNA encoding C3, and wherein the region of complementarity comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the antisense nucleotide sequences in any one of Tables 2-5;
- or
- (b) wherein the sense strand comprises at least 15 contiguous nucleotides differing by no more than three nucleotides from any one of the nucleotide sequence of nucleotides 809-831, 810-832, 811-831, 812-832, 2632-2654, 2633-2655, 2634-2654, 2634-2656, or 2635-2655 of SEQ ID NO:
- 901, and the antisense strand comprises at least 15 contiguous nucleotides from the corresponding nucleotide sequence of SEQ ID NO:902.
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Dependencies are inferred from the claim text.