bluebird bio BLUE today announced that the first subject with
beta-thalassemia major has been enrolled in its phase 1/2 HGB-205 study
in France and has undergone infusion with bluebird bio's LentiGlobin
drug product in an autologous hematopoietic stem cell transplantation.
“We believe gene therapy represents a potentially new and exciting
treatment option for patients with severe forms of beta-thalassemia and
sickle cell disease,” stated Marina Cavazzana, MD, Professor of Medicine
at Paris Descartes University and Research Director at the Centre for
Clinical Research in Biotherapy, Necker Hospital, Paris France. “The
beta-hemoglobinopathies are the most prevalent inherited disorders
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