RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use
US12054718B2 · kind B2 · utility
Assignee
Inventors
Key dates
| Filing date | Mar 26, 2021 |
| Grant date | Aug 6, 2024 |
| Priority date | — |
| Expiry date | Jun 7, 2041 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y301/01003
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Described are RNAi agents, e.g., double stranded RNAi agents, for inhibiting patatin-like phospholipase domain-containing protein 3 (PNPLA3) gene expression. Also disclosed are pharmaceutical compositions that include PNPLA3 RNAi agents and methods of use thereof. The PNPLA3 RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Delivery of the PNPLA3 RNAi agents in vivo provides for inhibition of PNPLA3 gene expression. The RNAi agents can be used in methods of treatment of PNPLA3-related diseases and disorders, including non-alcoholic fatty liver disease (NAFLD), nonalcoholic steatohepatitis (NASH), hepatic fibrosis, and alcoholic or non-alcoholic liver diseases, including cirrhosis.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.