Posted by Michael Wonder on 17 Apr 2019
CRISPR Therapeutics and Vertex announce FDA fast track designation for CTX001 for the treatment of beta thalassemia
16 April 2019 - CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. FDA has granted fast track designation for CTX001 for the treatment of transfusion-dependent beta thalassemia.
CTX001 is an investigational, autologous, gene-edited haematopoietic stem cell therapy for patients suffering from severe haemoglobinopathies.
In February 2019, CRISPR Therapeutics and Vertex announced that the first patient had been treated with CTX001 in a Phase 1/2 clinical study of patients with TDT, marking the first company-sponsored use of a CRISPR/Cas9 therapy in a clinical trial. The Phase 1/2 open-label trial is designed to assess the safety and efficacy of a single dose of CTX001 in patients ages 18 to 35 with TDT, non-beta zero/beta zero subtypes.
