By Denny Jacob
The shares of Editas Medicine Inc. were down 1.5% to $10.65 in after-hours commerce Thursday after the Food and Drug Administration granted EDIT-301 orphan drug designation.
EDIT-301 is an investigational gene-editing drug for the treatment of beta-thalassemia, a blood disorder. The company, which develops and commercializes genome editing technology, said the FDA has previously granted rare pediatric disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease.
The company said the FDA’s orphan drug designation is for diseases that affect fewer than 200,000 people in the U.S.
EDIT-301 is currently being investigated in a clinical trial in patients with severe sickle cell disease, the company said. Editas said it expects to initiate a phase 1/2 trial of EDIT-301 in patients with transfusion-dependent beta-thalassemia in 2022.
Write to Denny Jacob at [email protected]