The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to EDIT-301, an investigational, gene-edited medicine for the treatment of beta thalassemia from leading genome editing company, Editas Medicine, Inc. (NASDAQ:EDIT).
EDIT-301was previously awarded Rare Pediatric Disease designation for the treatment of sickle cell disease.
James C. Mullen, Chairman, President, and Chief Executive Officer, commented, “Beta thalassemia is a devastating disease that leads to severe anemia, organ failure, and premature death. Receiving Rare Pediatric Disease designation for EDIT-301 for beta thalassemia highlights the dire need for new treatment options”.
Additionally, EDIT-301 is being evaluated in the RUBY clinical study for the patients with severe sickle cell disease (SCD).
Editas is planning to initiate a Phase 1/2 clinical study of EDIT-301 in patients with transfusion-dependent beta thalassemia in 2022.
The FDA defines a rare pediatric disease as a serious or life-threatening disease, that may affect fewer than 200,000 people in the United States, in which the disease manifestations primarily affect individuals aged from birth to 18 years.
Editas Medicineis currently trading down 2.75 percent at $14.58
