EMA Assessing Gene Therapy for Sickle Cell Disease

The European Medicines Agency (EMA) accepted the autologous gene therapy, known as LentiGlobin BB305 Drug Product, for sickle cell disease (SCD) for its accelerated assessment.

With LentiGlobin BB305 Drug Product, a functioning hemoglobin (Hb) gene (an engineered ßA-T87Q-globin gene) is transported into a patient’s hematopoietic stem cells ex vivo before being infused back into the patient’s blood.

This drug product previously received breakthrough therapy designation from the U.S. Food and Drug Administration for treating transfusion-dependent beta-thalassemia.

Source: Bluebird Bio press release, September 27, 2016.

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