Complement component C3 iRNA compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsGregory Hinkle, Anna Borodovsky
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Abstract
The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the complement factor C3 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of a C3 gene and to treat subjects having a complement component C3-associated disease, 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), and C3 glomerulonephritis.
Claims24
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement component C3 in a cell, wherein said dsRNA comprises a sense strand and an antisense strand forming a double stranded region,
- wherein each strand is independently 15-30 nucleotides in length,
- wherein the antisense strand comprises at least 15 contiguous nucleotides from the complement of nucleotides 4614-4632 of SEQ ID NO:1,
- wherein all of the nucleotides of the sense strand and all of the nucleotides of the antisense strand comprise a nucleotide modification, and
- wherein at least one strand is conjugated to a ligand.
A double stranded ribonucleic acid (dsRNA) agent for inhibiting expression of complement component C3 in a cell,
- wherein said dsRNA comprises a sense strand and an antisense strand forming a double stranded region,
- wherein each strand is independently 15-30 nucleotides in length,
- wherein the antisense strand comprises at least 15 contiguous nucleotides from the complement of nucleotides 4614-4632 of SEQ ID NO:1,
- wherein all of the nucleotide of the sense strand and all of the nucleotides of the antisense strand comprise a nucleotide modification,
- wherein the dsRNA agent comprises 6-8 phosphorothioate internucleotide linkages, and
- wherein at least one strand is conjugated to an N-acetylgalactosamine ligand.
Dependencies are inferred from the claim text.