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A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)

Trial Profile

A Phase 1/2 Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects With β-Thalassemia Major by Transplantation of Autologous CD34+ Cells Transduced Ex Vivo With a Lentiviral βA-T87Q-Globin Vector (LentiGlobin® BB305 Drug Product)

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 06 Nov 2019

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At a glance

  • Drugs BB305 beta-globin gene therapy bluebird bio (Primary)
  • Indications Beta-thalassaemia
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms NorthStar
  • Sponsors bluebird bio
  • Most Recent Events

    • 06 Nov 2019 According to a Bluebird bio media release, updated efficacy and safety data will be presented at the upcoming 61st American Society of Hematology (ASH) Annual Meeting and Exposition in Orlando, Florida, December 7 - 10.
    • 14 Jun 2019 Results presented in a bluebird bio Media Release.
    • 03 Jun 2019 The European Commission (EC) has granted conditional marketing authorization for ZYNTEGLO for patients 12 years and older with transfusion-dependent beta-thalassemia for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available. This approval is supported by data from HGB-205 ,HGB-204, HGB-207, HGB-212 and LTF-303 studies.
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