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-HPV569; LentiGlobin™ BB305; Lentiviral beta AT87Q-Globin vector

Latest Information Update: 03 Aug 2017

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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

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

Most Recent Events

  • 01 Jul 2017 Phase-III clinical trials in Beta-thalassaemia (In children, In adolescents, In adults) in USA (IV) (NCT03207009)
  • 30 Jun 2017 bluebird bio completes enrolment in the Northstar-2 trial for Beta-thalassaemia in USA (IV) (NCT02906202)
  • 23 Jun 2017 Follow-up safety and efficacy data from the phase I/II HGB-205 trial in ß-Thalassemia and Sickle cell disease released by bluebird bio
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