COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS FOR USE IN THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsAnna Borodovsky, Bret Lee Bostwick
Life of this patent
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Abstract
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene for methods of using such iRNA to inhibit expression of C5 and to treat subjects having ALS.
Claims24
A method for treating amyotrophic lateral sclerosis (ALS) in a subject in need thereof, the method comprising administering to the subject a double-stranded ribonucleic acid (dsRNA) agent, or a salt thereof, which inhibits expression of complement component C5,
- a) wherein said dsRNA comprises a sense strand and an antisense strand, wherein said sense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO:1 and said antisense strand comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence of SEQ ID NO:5,
- wherein at least one nucleotide comprises a nucleotide modification, and
- wherein at least one strand is conjugated to a ligand;
- or
- b) wherein said dsRNA comprises a sense strand and an antisense strand, the antisense strand comprising a region of complementarity which comprises at least 15 contiguous nucleotides differing by no more than 3 nucleotides from any one of the antisense sequences listed in any one of Tables 3, 4, 5, 6, 18, 19, 20, 21, and 23,
- wherein at least one nucleotide comprises a nucleotide modification, and
- wherein at least one strand is conjugated to a ligand.
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Dependencies are inferred from the claim text.