bluebird bio Inc.'s investigational gene therapy Lenti-D for the treatment of patients with cerebral adrenoleukodystrophy (CALD). Also known as Lorenzo’s Oil disease, CALD is rare, genetic progressive neurodegenerative disease caused by mutations in the ABCD1 gene, which leads to the toxic accumulation of very long chain fatty acids. Breakthrough status for Lenti-D is based on the ongoing Phase II/III Starbeam Study, bluebird announced May 23, where 15 of 17 boys aged 17 and younger "remained alive and free of major functional disabilities at 2 years post-treatment, the primary efficacy endpoint of the trial." Additionally, the safety profile was consistent with that of myeloablative chemotherapy, according to the drugmaker. A one-time gene therapy, Lenti-D also has orphan drug status and a rare pediatric disease designation.

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