Compositions and methods for treating hemoglobinopathies
US11344609B2 · kind B2 · utility
1Cited by
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34Claims
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Key dates
| Filing date | Mar 26, 2021 |
| Grant date | May 31, 2022 |
| Priority date | — |
| Expiry date | Mar 26, 2041 |
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.