Home » Editas Gets Orphan Drug Designation for Beta Thalassemia Gene Therapy Drug
Editas Gets Orphan Drug Designation for Beta Thalassemia Gene Therapy Drug
The FDA granted Editas Medicine’s investigational gene editing therapy drug EDIT-301 an Orphan Drug designation for the inherited blood disorder beta thalassemia — a disease that prevents patients from producing enough hemoglobin.
The gene therapy previously received an Orphan Drug designation for sickle cell disease and a Rare Pediatric Disease designation.
Editas Medicine said it plans to begin a study of the gene editing drug’s effects on participants with transfusion-dependent beta thalassemia later this year. In the meantime, a separate ongoing study is evaluating EDIT-301 in patients with severe sickle cell disease.
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