CRISPR Therapeutics and Vertex Pharmaceuticals Announce FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to CTX001â„¢ for the Treatment of Severe Hemoglobinopathies

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May 11, 2020
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CTX001 has received Orphan Drug Designation from the U.S. Food and Drug Administration for transfusion-dependent beta thalassemia and from the European Medicines Agency for sickle cell disease and transfusion-dependent beta thalassemia