Compositions and methods for treating hemoglobinopathies
US11752202B2 · kind B2 · utility
1Cited by
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28Claims
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Key dates
| Filing date | Apr 13, 2022 |
| Grant date | Sep 12, 2023 |
| Priority date | — |
| Expiry date | Apr 13, 2042 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2800/80
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
Abstract
The present invention features compositions and methods for editing deleterious mutations associated with hemoglobinopathies, such as sickle cell disease (SCD). In particular embodiments, the invention provides methods for correcting mutations in a beta globin polynucleotide using modified adenosine base editors termed “ABE8” having unprecedented levels (e.g., >60-70%) of efficiency.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.