Beta-globin gene therapy - bluebird bio

Drug Profile

Beta-globin gene therapy - bluebird bio

Alternative Names: 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; LentiGlobin gene therapy; LentiGlobin-HPV569; LentiGlobin™ BB305; Lentiviral beta AT87Q-Globin vector

Latest Information Update: 11 Oct 2018

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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
  • New Molecular Entity No

Highest Development Phases

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

Most Recent Events

  • 05 Oct 2018 Preregistration for Beta-thalassaemia (In adolescents, In adults) in European Union (IV)
  • 05 Oct 2018 The EMA accepts MAA for beta-globin gene therapy for transfusion dependant β-thalassaemia
  • 26 Jul 2018 The Committee for Medicinal Products for Human Use grants accelerated assessment to beta-globin gene therapy for Beta-thalassaemia
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