CRISPR Therapeutics and Vertex Pharmaceuticals are co-developing CTX001, an investigational ex vivo CRISPR gene-edited therapy for patients suffering from β-thalassemia and sickle cell disease in which a patient’s hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. A Phase 1/2 study of CTX001 is currently enrolling patients. In addition, CRISPR Therapeutics is independently advancing allogeneic CAR-T therapies for both heme and solid cancers.