Editas Medicine receives FDA ODD for EDIT-301

Editas Medicine announced that the U.S. Food and Drug Administration, FDA, granted Orphan Drug Designation, ODD, to EDIT-301, an investigational, gene editing medicine, for the treatment of sickle cell disease. The FDA previously granted Orphan Drug Designation to EDIT-301 for the treatment of beta thalassemia and Rare Pediatric Disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease. "Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and early death. Receiving Orphan Drug Designation for EDIT-301 for sickle cell disease highlights the urgent need for new treatment options for patients and supports our belief that EDIT-301 can be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical benefits to patients," Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. "I would like to thank the participants, their families, clinicians, and colleagues at collaborating institutions that contribute to the RUBY trial. We look forward to sharing further clinical updates and clinical data for the trial in the near future."