Editas Medicine Granted FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for EDIT-301 for the Treatment of Severe Sickle Cell Disease

CAMBRIDGE, Mass., Oct. 16, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical-stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to EDIT-301, an investigational, gene editing medicine, for the treatment of severe sickle cell disease (SCD).

“Sickle cell disease is a devastating disease that leads to anemia, pain crises, organ failure, and early death. Receiving RMAT designation for EDIT-301 for severe sickle cell disease highlights the urgent need for new treatment options for patients and supports our belief that EDIT-301 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 including additional data for the trial prior to year-end.”

Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the development and review processes for promising regenerative medicine therapies. An investigational cell therapy medicine or gene editing medicine is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the experimental medicine has the potential to address unmet medical needs for the disease or condition. Advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including but not limited to intensive FDA guidance on efficient and expedited drug development, possible rolling review, and priority review of the biologics license application (BLA), and FDA’s organizational commitment involving senior managers.

The FDA previously granted Orphan Drug Designation and Rare Pediatric Disease designation to EDIT-301 for the treatment of sickle cell disease and beta thalassemia.

About Sickle Cell Disease
Sickle cell disease is an inherited blood disorder caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin (HbS). In sickle cell disease, the red blood cells are misshapen in a sickle shape instead of a typical disc shape. The abnormal shape causes the red blood cells to have shortened lifespan and to block blood flow causing anemia, pain crises, organ failure, and early death. There are an estimated 100,000 people in the United States currently living with sickle cell disease. Higher levels of fetal hemoglobin (HbF) inhibit HbS polymerization, thus reducing the manifestation of sickling.