CTX001 to Be Developed as Gene-Editing Therapy for Sickle Cell Disease
Vertex Pharmaceuticals and CRISPR Therapeutics recently announced a joint collaboration to co-develop and co-commercialize CTX001 as a gene-editing treatment of diseases of hemoglobin, including β-thalassemia and sickle cell disease (SCD). The announcement follows the presentation of preclinical data for CTX001 at the American Society of Hematology…