Editas Medicine receives FDA Orphan Drug Designation for EDIT-301 for the treatment of sickle cell disease

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Editas Medicine’s 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,” said 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.”

The FDA’s Orphan Drug Designation program provides orphan status to drugs or biologics intended for the prevention, diagnosis, or treatment of diseases that affect fewer than 200,000 people in the United States. Sponsors of medicines that are granted Orphan Drug Designation are entitled to certain incentives, including tax credits for qualified clinical trials, prescription drug user-fee exemptions, and potential seven-year marketing exclusivity upon FDA approval.

EDIT-301 is currently being investigated in a clinical study in patients with severe sickle cell disease (RUBY trial, NCT04853576) and transfusion-dependent beta thalassemia (EDITHAL trial, NCT#05444894). Editas Medicine will present clinical data updates from the RUBY trial twice this year, mid-year and again by year-end. Additionally, the company is on-track to dose 20 patients in the RUBY trial by year-end.

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