A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Beta-Hemoglobinopathies (Sickle Cell Disease and Beta-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral Beta-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)

Trial Profile

A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the Beta-Hemoglobinopathies (Sickle Cell Disease and Beta-Thalassemia Major) by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With a Lentiviral Beta-A-T87Q Globin Vector (LentiGlobin BB305 Drug Product)

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 02 Aug 2017

At a glance

  • Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
  • Indications Beta-thalassaemia; Sickle cell anaemia
  • Focus Adverse reactions; Proof of concept; Therapeutic Use
  • Sponsors bluebird bio
  • Most Recent Events

    • 02 Aug 2017 According to a bluebird bio media release, the company presented new data from this study at European Hematology Association (EHA) Annual Meeting.
    • 23 Jun 2017 According to a bluebird bio media release, as of June 2, 2017 four patients with Transfusion-Dependent Beta-Thalassemia and three patients with severe Severe Sickle Cell Disease undergone infusion in this trial.
    • 23 Jun 2017 Results published in the bluebird bio Media Release
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