Following a management shake-up, Editas Medicine received some welcome good news. The biotech has scored a second rare pediatric disease designation from the FDA for its gene edited medicine EDIT-301.
EDIT-301 already holds the designation for treating sickle cell disease and is currently under investigation in a clinical study. Following the FDA’s most recent designation, Editas expects to launch a phase 1/2 study of EDIT-301 in patients with transfusion-dependent beta thalassemia this year.
The gene editing biotech has been making headlines recently, but not necessarily for its clinical treatments. The company announced several leadership shake-ups, bringing on Sarepta’s Chief Medical Officer Gilmore O’Neill to replace Chairman, President and CEO James Mullen, who will become Editas’ executive chairman, effective June 1. Interestingly, O’Neill himself isn’t filling the biotech’s chief medical officer role, which currently sits empty after the company ousted Lisa Michaels, M.D., without explanation in February.
The company is also involved in an ongoing patent dispute in which it came out on top in February when the U.S. Patent and Trademark Office ruled in favor of Harvard and the Broad Institute of MIT in a dispute over CRISPR gene editing tech patents. Editas holds its patents with this group and saw a bump in its share price after the decision. However, the opposing group have filed an appeal with the U.S. Court of Appeals for the Federal Circuit, leaving the matter hanging in the balance.
The company revealed its first proof-of-concept data last fall, but the limited readout failed to impress investors. The newest FDA tag for beta thalassemia may help bolster the company’s clinical case for its gene editing therapies.
“Beta thalassemia is a devastating disease that leads to severe anemia, organ failure, and premature death,” Mullen said in an April 26 release. “EDIT-301 is a potentially transformative medicine for patients living with beta thalassemia, and we look forward to dosing the first patient in our clinical trial this year.”