RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use
US11912997B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Jun 14, 2023 |
| Grant date | Feb 27, 2024 |
| Priority date | — |
| Expiry date | Jun 14, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2310/3515
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Superoxide Dismutase 1 (SOD1) gene. The SOD1 RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an SOD1 gene. Pharmaceutical compositions that include one or more SOD1 RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described SOD1 RNAi agents to central nervous system (CNS) tissue, in vivo, provides for inhibition of SOD1 gene expression and a reduction in SOD1 activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including amyotrophic lateral sclerosis (ALS.).
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.