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Beta-globin gene therapy - bluebird bio

Drug Profile

Beta-globin gene therapy - bluebird bio

Alternative Names: Autologous CD34+ cells encoding βA-T87Q-globin gene gene therapy - bluebird bio; BB305-beta-A(T87Q)-globin-gene-therapy-bluebird-bio; Beta-globin-gene-therapy-bluebird-bio; HPV569-beta-A(T87Q)-globin-gene-therapy-bluebird-bio; HPV569-beta-globin-gene-therapy-bluebird-bio; LentiGlobin gene therapy; LentiGlobin-HPV569; LentiGlobin™ BB305; Lentiviral beta AT87Q-Globin vector; Zynteglo

Latest Information Update: 04 Sep 2019

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At a glance

  • Originator Genetix Pharmaceuticals
  • Developer bluebird bio
  • Class Antianaemics; Gene therapies; Stem cell 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 - Beta-thalassaemia; Sickle cell anaemia
  • New Molecular Entity No

Highest Development Phases

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

Most Recent Events

  • 31 Oct 2019 bluebird bio plans to launch Beta-globin gene therapy for Beta-thalassaemia in early 2020
  • 01 Aug 2019 bluebird bio announces intention to launch beta-globin gene therapy for Beta-thalassaemia in Europe
  • 19 Jun 2019 Efficacy data from a phase III Northstar-3 trial in Beta-thalassaemia released by bluebird bio
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