US FDA grants orphan drug status to Editas Medicines EDIT-301 to treat beta thalassemia
Editas Medicine, Inc., a leading genome editing company, announced that the US Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of beta thalassemia. The FDA previously granted Rare Paediatric Disease designation to EDIT-301 for the treatment of beta thalassemia and sickle cell disease.
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