The FDA granted fast track designation to CTX001, a gene-edited hematopoietic stem cell therapy, for the treatment of sickle cell disease.
CTX001 (CRISPR Therapeutics, Vertex Pharmaceuticals Inc.) is an investigational, autologous gene therapy under evaluation for patients with severe hemoglobinopathies. The ex-vivo therapy involves engineering a patient’s hematopoietic stem cells to produce high levels of fetal hemoglobin in red blood cells, with the goal of alleviating transfusion requirements for patients with beta-thalassemia and sickle crises for patients with sickle cell disease.
Enrollment in a phase 1/phase 2 trial of CTX001 in patients with sickle cell disease is underway in the U.S. The gene therapy also is under investigation in a phase 1/phase 2 trial that is enrolling patients with beta-thalassemia in Europe.