Beta-globin gene therapy - bluebird bio

Drug Profile

Beta-globin gene therapy - bluebird bio

Alternative Names: Beta-thalassaemia gene therapy; LentiGlobin™ BB305; Lentiviral beta AT87Q-Globin vector; Lentiviral β-A(T87Q)-Globin Vector; Sickle cell anaemia gene therapy

Latest Information Update: 12 Dec 2016

Price : $50

At a glance

  • Originator Genetix Pharmaceuticals
  • Developer bluebird bio
  • Class Antianaemics; 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 - Sickle cell anaemia
  • 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.

  • New Molecular Entity No

Highest Development Phases

  • Phase III Beta-thalassaemia
  • Phase I/II Sickle cell anaemia

Most Recent Events

  • 05 Dec 2016 Efficacy and adverse event data from the phase I/II Northstar trial in Beta-thalassaemia and the phase I/II HGB-206 trial in Sickle cell anaemia released by bluebird bio
  • 03 Dec 2016 Efficacy data from a phase I/II trial in Beta-thalassaemia presented at the 58th American Society of Hematology Annual Meeting (ASH-2016)
  • 02 Nov 2016 Phase-III clinical trials in Beta-thalassaemia in USA (IV) (NCT02906202)
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