Transfusion-dependent β-thalassemia (TDT) research hub

A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in subjects with Transfusion-Dependent β-Thalassemia

This study looks at a new gene therapy, Casgevy, for people with severe beta-thalassemia who need regular blood transfusions. Researchers want to see if this treatment is safe and can help patients produce enough healthy red blood cells on their own, reducing or stopping the need for transfusions.