Invention Grant
- Patent Title: Materials and methods for treatment of hemoglobinopathies
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Application No.: US16909283Application Date: 2020-06-23
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Publication No.: US12134767B2Publication Date: 2024-11-05
- Inventor: Matthew Hebden Porteus
- Applicant: Vertex Pharmaceuticals Incorporated
- Applicant Address: US MA Boston
- Assignee: Vertex Pharmaceuticals Incorporated
- Current Assignee: Vertex Pharmaceuticals Incorporated
- Current Assignee Address: US MA Boston
- Agency: McNeill PLLC
- Main IPC: C12N15/113
- IPC: C12N15/113 ; A61K35/28 ; A61K35/545 ; A61K38/42 ; A61K48/00 ; C12N5/074 ; C12N5/0789 ; C12N15/10 ; C12N15/63

Abstract:
The present application provides materials and methods for treating hemoglobinopathies. More specifically, the application provides methods for producing progenitor cells that are genetically modified via genome editing to increase the production of fetal hemoglobin (HbF), as well as modified progenitor cells (including, for example, CD34+ human hematopoietic stem cells) producing increased levels of HbF, and methods of using such cells for treating hemoglobinopathies such as sickle cell anemia and β-thalassemia.
Public/Granted literature
- US20210317450A9 MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES Public/Granted day:2021-10-14
Information query
IPC分类: