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Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Trial Profile

Longterm Follow-up of Subjects With Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Recruiting
Phase of Trial: Phase III

Latest Information Update: 08 Feb 2019

At a glance

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

    • 05 Oct 2018 Based on the study HGB-204, HGB-205, HGB-207 and long-term follow-up study LTF-303, the European Medicines Agency (EMA) accepted the company's marketing authorization application (MAA) for its investigational LentiGlobin gene therapy for the treatment of adolescents and adults with transfusion-dependent beta-thalassemia (TDT) and a non-beta/beta genotype
    • 06 Aug 2018 Planned number of patients changed from 86 to 94.
    • 26 Jul 2018 According to a bluebird bio media release, based on the study HGB-204, HGB-205, HGB-207 and long-term follow-up study LTF-303, the Committee for Medicinal Products for Human Use (CHMP) of EMA has granted the Accelerated Assessment for its marketing authorization application (MAA) for LentiGlobin gene therapy for the treatment of adolescent and adult patients with transfusion-dependent beta-thalassemia (TDT). The company is intend to file an MAA in 2018.
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