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FDA tags Editas’ thalassemia cell therapy an orphan drug as it rounds final bend in race to clinic

May 13, 2022

Editas Medicine has picked up more regulatory privileges for its ex vivo gene-edited medicine, securing FDA orphan-drug designation in beta thalassemia ahead of the start of a clinical trial in the indication.

The candidate, EDIT-301, is in development as a treatment for transfusion-dependent beta thalassemia and sickle cell disease. Editas is advancing the cell therapy in the indications in the belief that mimicking the naturally occurring mutations in patients with hereditary persistence of fetal hemoglobin will restore a healthy level of fetal hemoglobin, thereby freeing beta thalassemia patients from transfusions.

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