Gene editing for hemophilia A with improved factor VIII expression
US11529427B2 · kind B2 · utility
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20Claims
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Key dates
| Filing date | Apr 15, 2020 |
| Grant date | Dec 20, 2022 |
| Priority date | — |
| Expiry date | Sep 2, 2040 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/22
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
Provided herein, in some embodiments, are materials and methods for treating hemophilia A in a subject ex vivo or in vivo. Also provided herein, in some embodiments, are materials and methods for knocking in a coding sequence encoding a synthetic FVIII having a B domain substitute into a genome.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.