Posted by Michael Wonder on 11 May 2020
CRISPR Therapeutics and Vertex Pharmaceuticals announce FDA regenerative medicine advanced therapy designation granted to CTX001 for the treatment of severe hemoglobinopathies
11 May 2020 - CTX001 has received orphan drug designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia.
CRISPR Therapeutics and Vertex Pharmaceuticals today announced that the U.S. FDA granted regenerative medicine advanced therapy designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease and transfusion-dependent beta thalassemia.
