FDA grants rare pediatric disease designation to sickle cell disease therapy

The U.S. Food and Drug Administration has granted rare pediatric disease designation to EDIT-301, an experimental, autologous cell medicine for sickle cell disease. Editas Medicine, Inc plans to file an investigational new drug application for EDIT-301 by the end of 2020, according to a company press release.

“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 in a press release.  “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.”

Read the full press release here.