Vertex and CRISPR Therapeutics Present New Data on More Patients With Longer Follow-Up Treated With
– Data from 75 patients with transfusion-dependent beta thalassemia or severe sickle cell disease with follow-up of up to 37.2 months continue to demonstrate that exa-cel has the potential to be a one-time functional cure –– Safety profile generally consistent with myeloablative conditioning and autologous stem cell...