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.
