Vertex Pharmaceuticals Incorporated
MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
Last updated:
Abstract:
Materials and methods for treating a patient with a hemoglobinopathy, both ex vivo and in vivo, and materials and methods for deleting, modulating, or inactivating a transcriptional control sequence of a BCL11A gene in a cell by genome editing.
Status:
Application
Type:
Utility
Filling date:
2 Feb 2022
Issue date:
7 Jul 2022