Editas Medicine Gets Orphan Drug Designation for EDIT-301

By Chris Wack

Editas Medicine Inc. said Thursday that the U.S. Food and Drug Administration granted its Orphan Drug Designation to EDIT-301, an investigational gene-editing medicine, for the treatment of sickle cell disease.

The clinical stage genome-editing company said the FDA previously granted Orphan Drug Designation to EDIT-301 for the treatment of beta thalassemia and Rare Pediatric Disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease.

The FDA's Orphan Drug Designation program provides orphan status to drugs or biologics intended for the prevention, diagnosis, or treatment of diseases that affect fewer than 200,000 people in the U.S.

EDIT-301 is currently being investigated in a clinical study in patients with severe sickle cell disease and transfusion-dependent beta thalassemia. Editas Medicine said it is on track to dose 20 patients in the sickle-cell trial by year end.

Editas shares were up 6% at $8.40 in premarket trading.

Write to Chris Wack at chris.wack@wsj.com

(END) Dow Jones Newswires

April 27, 2023 09:33 ET (13:33 GMT)

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