Adrenoleucodystrophy gene therapy - bluebird bio

Drug Profile

Adrenoleucodystrophy gene therapy - bluebird bio

Latest Information Update: 16 Jul 2016

Price : $50

At a glance

  • Originator Genetix Pharmaceuticals; INSERM
  • Developer bluebird bio
  • Class Adrenoleucodystrophy gene therapies; Gene therapies
  • Mechanism of Action Gene transference
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Adrenoleucodystrophy
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity No

Highest Development Phases

  • Phase II/III Adrenoleucodystrophy

Most Recent Events

  • 16 Jul 2016 No recent reports of development identified for preclinical development in Adrenoleucodystrophy in USA (Parenteral, Infusion)
  • 20 Apr 2016 Additional interim efficacy and adverse events data from phase II/III trial for Adrenoleukodystrophy presented at the 68th Annual Meeting of the American Academy of Neurology (AAN-2016)
  • 03 Mar 2016 Interim efficacy and adverse events data from a phase II/III trial for Adrenoleukodystrophy (In children) released by bluebird bio
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