bluebird bio Receives U.S. and European Orphan Drug Designation for Novel Gene Therapy to Treat Adrenoleukodystrophy
Published: Jun 19, 2012
CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD). The product consists of the patient’s own CD34+ hematopoietic stem cells transduced with bluebird bio’s lentiviral vector, Lenti-D, encoding the human ABCD1 cDNA. Based on promising early clinical proof of concept results, bluebird bio plans to initiate a Phase 2/3 clinical study in childhood cerebral ALD in both the United States and Europe in 2013.