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  • Conversation: Bluebird bags breakthrough status for CALD gene therapy

    • May 24, 2018 12:16 PM BST
    • Bluebird bags breakthrough status for CALD gene therapy

      Bluebird Bio has claimed an FDA breakthrough designation for its Lenti-D gene therapy for cerebral adrenoleukodystrophy (CALD) – a rare neurological disease mainly affecting young males.
      The new status – which could result in a speedy review by the US regulator and shorten the time to market for the therapy – comes after impressive data from a phase II/III trial suggesting it could stabilise disease for more than two years in boys with the highly progressive and life-threatening disorder.
      Data from 17 patients in the STARBEAM study was published in the New England Journal of Medicine last October, and showed that Lenti-D (elivaldogene tavalentivec) stabilised disease in 15 of them, with one boy dying as a result of the disease and another progressing despite therapy.
      Read more: http://www.pmlive.com/pharma_news/bluebird_bags_breakthrough_status_for_cald_gene_therapy_1236797

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