Derailed by the pandemic, FDA offers CRISPR, Vertex drug program VIP status for its comeback play
CRISPR Therapeutics may have run into a hurdle for its clinical program to advance CTX001 in severe hemoglobinopathies, but the FDA is on record that it will do everything it can to get the drug back on track and headed to the finish line.
CRISPR and its partners at Vertex announced Monday morning that the FDA had awarded them the Regenerative Medicine Advanced Therapy — RMAT — designation for their program to treat severe sickle cell disease and transfusion-dependent beta thalassemia. And that should give them an open door as they hustle through the clinical program.
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