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Bluebird Bio (BLUE) will present data from the ongoing Phase 2/3 Starbeam Study (ALD-102) for the treatment of cerebral adrenoleukodystrophy (CALD) at the American Academy of Neurology (AAN) 2016 Annual Meeting

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Management Comment “The childhood form of cerebral adrenoleukodystrophy is a devastating neurodegenerative genetic disease that affects boys and is generally fatal if left untreated. Allogeneic hematopoietic stem cell transplant (allo-HSCT) is currently the only available effective therapy, but is potentially associated with serious safety risks, including graft rejection, graft-versus-host disease and transplant-related mortality. The Lenti-D gene therapy product candidate consists of a patient’s own modified hematopoietic stem cells and should avoid the problems of immune-incompatibility that can complicate allo-HSCT,” said David Davidson, M.D., chief medical officer, bluebird bio. “We are excited to report interim data from the Starbeam Study of Lenti-D at this year’s AAN meeting. While these data are still early, we are encouraged by the safety profile and the radiographic and neurologic results reported in the abstract. We look forward to presenting updated data and additional findings in Vancouver next month.”
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Related Keywords Phase 2/3 Study, Cald, Cerebral Adrenoleukodystrophy, Allo-hsct, Lenti-d