Complement component C5 iRNA compositions and methods of use thereof
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsKevin Fitzgerald, James Butler, Brian Bettencourt, Anna Borodovsky, Satyanarayana Kuchimanchi, Klaus Charisse, Muthiah Manoharan, Martin A Maier, Kallanthottathil G. Rajeev, Donald Foster
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Abstract
The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.
Claims31
A method of inhibiting complement component C5 expression in a cell, the method comprising:
- (a) contacting the cell with a double-stranded ribonucleic acid (dsRNA) agent that inhibits expression of complement component C5, or a pharmaceutically acceptable salt thereof, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region, wherein the antisense strand comprises at least 17 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence 5′-UAUUAUAAAAAUAUCUUGCUUUU-3′ of SEQ ID NO:113, and wherein the dsRNA agent comprises at least one modified nucleotide;
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- (b) maintaining the cell produced in step (a) for a time sufficient to obtain degradation of the mRNA transcript of a complement component C5 gene, thereby inhibiting expression of the complement component C5 gene in the cell.
A method of treating a subject having a disease or disorder that would benefit from reduction in complement component C5 expression, the method comprising administering to the subject a therapeutically effective amount of a double-stranded ribonucleic acid (dsRNA) agent that inhibits expression of complement component C5, or a pharmaceutically acceptable salt thereof, wherein the dsRNA agent comprises a sense strand and an antisense strand forming a double stranded region, wherein the antisense strand comprises at least 17 contiguous nucleotides differing by no more than 3 nucleotides from the nucleotide sequence 5′-UAUUAUAAAAAUAUCUUGCUUUU-3′ of SEQ ID NO:113, and wherein the dsRNA agent comprises at least one modified nucleotide, thereby treating the subject.
Dependencies are inferred from the claim text.