Editas sees clinical promise with new FDA pediatric disease tag for another blood disorder

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.

Click here to read the full article at Fierce Biotech

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