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Bluebird Bio (BLUE) to complete Phase 2/3 Study of Hematopoietic Stem Cells Transduced with Lenti-D Lentiviral Vector for the treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD) in second half of 2018

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Additional Relevant Details Our Lenti-D product candidate has the potential to stabilize or prevent progression of CALD with one treatment. Our approach involves inserting a functional copy of the ABCD1 gene in the patient’s hematopoietic stem cells outside the body (ex vivo), then re-introducing the genetically modified cells into the patient.  We are presenting interim data from the Starbeam Study at this year’s American Academy of Neurology (AAN) Annual Meeting.
http://www.bluebirdb...
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Related Keywords Phase 2/3 Study, Lenti-d, Ccald, Cerebral Adrenoleukodystrophy, Hsct, Hematopoietic Stem Cell Transplantation