Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsJohn M. Gansner, David Erbe
Life of this patent
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Abstract
The present invention provides methods for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder that would benefit from reduction in oxalate, and compositions comprising nucleic acid inhibitors, e.g., double stranded ribonucleic acid (dsRNA) agents or single stranded antisense polynucleotide agents targeting lactate dehydrogenase A (LDHA), hydroxyacid oxidase (HAO1) and/or proline dehydrogenase 2 (PRODH2), for treating such subjects.
Claims28
A method for inhibiting the expression of hydroxyacid oxidase (HAO1) in a human subject having a non-primary hyperoxaluria disease or disorder that would benefit from reduction in urinary oxalate, comprising
- administering to the subject a fixed dose of about 200 mg to about 600 mg of a double stranded ribonucleic acid (dsRNA) agent, or salt thereof, which inhibits the expression of of HAO1,
- thereby inhibiting the expression of HAO1 in the subject.
A method for reducing urinary oxalate levels in a human subject having a non-primary hyperoxaluria disease or disorder that would benefit from reduction in urinary oxalate, comprising
- administering to the subject a fixed dose of about 200 mg to about 600 mg of a double stranded ribonucleic acid (dsRNA) agent, or salt thereof, which inhibits the expression of of HAO1,
- thereby reducing urinary oxalate levels in the subject.
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A method for treating a subject having a non-primary hyperoxaluria disease or disorder that would benefit from reduction in oxalate, the method comprising
- administering to the subject a therapeutically effective amount of a nucleic acid inhibitor of hydroxyacid oxidase (HAO1) and/or a nucleic acid inhibitor of Proline Dehydrogenase 2 (PRODH2),
- thereby treating the subject having the non-primary hyperoxaluria disease or disorder that would benefit from reduction in oxalate.
A method for treating a subject at risk of developing a non-primary hyperoxaluria disease or disorder that would benefit from reduction in oxalate, the method comprising
- administering to the subject a therapeutically effective amount of a nucleic acid inhibitor of lactate dehydrogenase A (LDHA), a nucleic acid inhibitor of hydroxyacid oxidase (HAO1), and/or a nucleic acid inhibitor of Proline Dehydrogenase 2 (PRODH2),
- thereby treating the subject at risk of developing the non-primary hyperoxaluria disease or disorder that would benefit from reduction in oxalate.
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Dependencies are inferred from the claim text.