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.