RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
US11845937B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Mar 9, 2023 |
| Grant date | Dec 19, 2023 |
| Priority date | — |
| Expiry date | Mar 9, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/32
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a double homeobox 4 (DUX4) gene. The DUX4 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a DUX4 gene. Pharmaceutical compositions that include one or more DUX4 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described DUX4 RNAi agents to skeletal muscle cells in vivo, provides for inhibition of DUX4 gene expression and a reduction in DUX4 levels, which can provide a therapeutic benefit to subjects, including human subjects, suffering from certain skeletal muscle-related diseases or disorders including Facioscapulohumeral Muscular Dystrophy (FSHD).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.