Patent · US Active

Compositions and methods for treating hemoglobinopathies

US11142760B2 · kind B2 · utility

1Cited by
0References
30Claims
0Family size

Assignee

Inventors

Key dates

Filing dateMar 26, 2021
Grant dateOct 12, 2021
Priority date
Expiry dateMar 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.