Bluebird Bio Launches First Gene Therapy for Beta Thalassemia in Germany

The US company bluebird bio has launched the first gene therapy for the blood disorder transfusion-dependent beta thalassemia in the EU, with a hospital in Germany becoming its first qualified treatment center. Bluebird bio’s gene therapy, branded as Zynteglo, was given conditional market approval by the EU in June 2019. It was the first gene therapy approved to treat people over 12 years of age with all but the most severe form of transfusion-dependent beta thalassemia, a condition that normally requires the patient to have a lifetime of blood transfusions to stay alive. To be eligible for treatment with the therapy, the patients must have no matching donor available for a stem cell transplant, one of the current methods for treating the condition.