Engineered nucleases useful for treatment of hemophilia A
US11278632B2 · kind B2 · utility
0Cited by
7References
16Claims
0Family size
Assignee
Inventors
Key dates
| Filing date | May 3, 2017 |
| Grant date | Mar 22, 2022 |
| Priority date | — |
| Expiry date | May 3, 2037 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12Y301/00
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention encompasses engineered nucleases which recognize and cleave a recognition sequence within the int22h-1 sequence of a Factor VIII gene. The present invention also encompasses methods of using such engineered nucleases to make genetically-modified cells, and the use of such cells in a pharmaceutical composition and in methods for treating hemophilia A. Further, the invention encompasses pharmaceutical compositions comprising engineered nuclease proteins, nucleic acids encoding engineered nucleases, or genetically-modified cells of the invention, and the use of such compositions for treating of hemophilia A.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.