Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
US10450565B2 · kind B2 · utility
7Cited by
33References
39Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | Jan 10, 2018 |
| Grant date | Oct 22, 2019 |
| Priority date | — |
| Expiry date | Jan 10, 2038 |
Classification
- Technology area (CPC G)Physics
- CPC primaryG01N2800/085
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
RNAi agents for inhibiting the expression of the alpha-1 antitrypsin (AAT) gene, compositions including AAT RNAi agents, and methods of use are described. Also disclosed are pharmaceutical compositions including one or more AAT RNAi agents together with one or more excipients capable of delivering the RNAi agent(s) to a liver cell in vivo. Delivery of the AAT RNAi agent(s) to liver cells in vivo inhibits AAT gene expression and treats diseases associated with AAT deficiency such as chronic hepatitis, cirrhosis, hepatocellular carcinoma, transaminitis, cholestasis, fibrosis, and fulminant hepatic failure.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.