Compositions and methods for inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) gene expression
US10478500B2 · kind B2 · utility
0Cited by
6References
19Claims
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Assignee
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Key dates
| Filing date | Oct 9, 2015 |
| Grant date | Nov 19, 2019 |
| Priority date | — |
| Expiry date | Oct 9, 2035 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y101/03015
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The invention relates to RNAi agents, e.g. double-stranded RNAi agents, targeting the HAO1 gene, and method of using such RNAi agents to inhibit expression of HAO1 and methods of treating subjects having, e.g., PH1. Described herein are double-stranded RNAi agents which inhibit the expression of a HA01 gene in a cell, such as a cell within a subject, e.g., a mammal, such as a human having a HAO1 associated disorder, and uses of such double-stranded RNAi agents. In certain aspects of the invention, substantially all of the nucleotides of an iRNA of the invention are modified.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.