Materials and methods for treatment of hemoglobinopathies

US12043843B2 · kind B2 · utility

Assignee

• Vertex Pharmaceuticals Incorporated · US

Inventors

• Matthew H. Porteus · Stanford, US
• Melanie Ruth ALLEN · Cambridge, US
• Chad A. Cowan · Cambridge, US
• Ante Sven Lundberg · Cambridge, US
• Michelle I-Ching Lin · Cambridge, US
• Jeffrey Li · Santa Clara, US
• Thao Thi NGUYEN · Cambridge, US

Key dates

• Filing date: Nov 4, 2016
• Grant date: Jul 23, 2024
• Expiry date: Dec 9, 2037

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2800/80
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.