Editas Medicine Receives Rare Pediatric Disease Designation for EDIT-301 for the Treatment of Sickle Cell Disease

CAMBRIDGE, Mass., Aug. 24, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for EDIT-301, an experimental, autologous cell medicine, being developed as a potentially best-in-class, durable medicine for sickle cell disease. The Company plans to file an investigational new drug application (IND) for EDIT-301 by the end of 2020.

“The Editas team has a bold vision to unlock the potential of CRISPR to design and develop game-changing medicines. We are making tremendous progress towards this vision with the continued development of EDIT-301, a potentially transformative medicine for the treatment of sickle cell disease, and we are pleased to receive Rare Pediatric Disease designation from the FDA for this program,” said Cynthia Collins, Chief Executive Officer, Editas Medicine.  “We know patients are counting on us, and this designation is a significant milestone for the program that highlights the serious, life-threatening manifestations of sickle cell disease.”

The FDA defines a rare pediatric disease as a serious or life-threating disease in which the serious or life-threatening disease manifestations primarily affect individuals aged from birth to 18 years. Pediatric diseases recognized as “rare” affect under 200,000 people in the United States. Under the FDA’s Rare Pediatric Disease Designation and Voucher Programs, a sponsor who receives an approval for a drug or biologic for a "rare pediatric disease" may be eligible for a voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product.

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 protein (HbS). In sickle cell disease, the red blood cells are misshapen, in a sickle shape instead of the disc shape. The abnormal shape causes the cells 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. Fetal hemoglobin (HbF) protects against sickle cell disease by inhibiting HbS polymerization.