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CRISPR Therapeutics and Vertex Pharmaceuticals Have Been Awarded an FDA RMAT Designation for CTX001™ in the Treatment of Severe Hemoglobinopathies
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CTX001 has received Orphan Drug Designation from the U.S. FDA for transfusion-dependent beta thalassemia and from the European Medicines Agency (EMA) for sickle cell disease and transfusion-dependent beta thalassemia
ZUG, Switzerland and Cambridge, MA, May 11, 2020 — CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. FDA RMAT designation to CTX001, an investigational, autologous, gene-edited hematopoietic stem cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). [Read more…]




