RNAi agents for inhibiting expression of complement component C3 (C3), pharmaceutical compositions thereof, and methods of use
US12110492B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Oct 26, 2023 |
| Grant date | Oct 8, 2024 |
| Priority date | — |
| Expiry date | Oct 26, 2043 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2320/32
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents or siRNAs, able to inhibit Complement Component C3 (C3) gene expression. Also disclosed are pharmaceutical compositions that include C3 RNAi agents and methods of use thereof. The C3 RNAi agents disclosed herein may be conjugated to targeting ligands, including ligands that comprise N-acetyl-galactosanine, to facilitate the delivery to hepatocyte cells. Delivery of the C3 RNAi agents in vivo provides for inhibition of C3 gene expression. The RNAi agents can be used in methods of treatment of diseases, disorders, or symptoms mediated in part by C3 gene expression, including IgA nephropathy, C3 glomerulopathy, paroxysmal nocturnal hemoglobinuria, and/or other complement-mediated renal diseases.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.