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US 12,723,247 B2Granted
Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
AssigneeAlnylam Pharmaceuticals, Inc.
InventorsDavid V. Erbe, Tracy L McGregor
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Abstract
The invention relates methods of using RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1.
Claims23
A method of treating a human subject having Primary Hyperoxaluria Type 1 (PH1), the method comprising
- subcutaneously administering to the subject 3.0 mg per kg bodyweight or 6.0 mg per kg bodyweight of a double stranded ribonucleic acid (dsRNA) agent which inhibits the expression of hydroxyacid oxidase 1 (HAO1), or a salt thereof,
- wherein the dsRNA agent, or a salt thereof, comprises a sense strand comprising the nucleotide sequence 5′-gsascuuuCfaUfCfCfuggaaauaua-3′ of SEQ ID NO:
- 14 and an antisense strand comprising the nucleotide sequence 5′-usAfsuauUfuCfCfaggaUfgAfaagucscsa-3′ of SEQ ID NO:15,
- wherein a, c, g, and u are 2′-O-methyl (2′-OMe) A, C, G, and U, respectively;
- Af, Cf, and Uf are 2′-fluoro A, C, and U, respectively;
- and s is a phosphorothioate linkage, and
- wherein the 3′-end of the sense strand is conjugated to an N-acetylgalactosamine (GalNAc) 3 ligand which is
- thereby treating the human subject having PH1.
5 more elements
A method of treating a human subject having Primary Hyperoxaluria Type 1 (PH1), the method comprising
- subcutaneously administering to the subject 3.0 mg per kg bodyweight or 6.0 mg per kg bodyweight of a double stranded ribonucleic acid (dsRNA) agent which inhibits the expression of hydroxyacid oxidase 1 (HAO1), or a salt thereof,
- wherein the dsRNA agent, or a salt thereof, comprises a sense strand consisting of the nucleotide sequence 5′-gsascuuuCfaUfCfCfuggaaauaua-3′ of SEQ ID NO:14 and an antisense strand consisting of the nucleotide sequence 5′-usAfsuauUfuCfCfaggaUfgAfaagucscsa-3′ of SEQ ID NO:15,
- wherein a, c, g, and u are 2′-O-methyl (2′-OMe) A, C, G, and U, respectively;
- Af, Cf, and Uf are 2′-fluoro A, C, and U, respectively;
- and s is a phosphorothioate linkage, and
- wherein the 3′-end of the sense strand is conjugated to an N-acetylgalactosamine (GalNAc) 3 ligand which is
- thereby treating the human subject having PH1.
4 more elements
Dependencies are inferred from the claim text.