March 27, 2014
bluebird bio, Inc. a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced that the first subject with beta-thalassemia major has been enrolled in its phase 1/2 Northstar Study (HGB-204) in the United States and has undergone infusion with bluebird bio’s LentiGlobin drug product in an autologous hematopoietic stem cell transplantation.
“We are very excited to have reached this critical milestone today with the infusion of modified blood stem cells in our patient with thalassemia. We hope this innovation will be a major advancement in thalassemia treatment,” Alexis Thompson, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine and Director of the Comprehensive Thalassemia Program at Ann & Robert H. Lurie Children’s Hospital of Chicago. “Patients with beta thalassemia major suffer from complications of their disease as well as its treatment that can take a significant physical and emotional toll. The potential of gene therapy to reestablish more effective hemoglobin production and, therefore, free patients from dependence on blood transfusions and iron chelation therapies is compelling.”
“We welcome the start of the Northstar Study, the second beta thalassemia major trial initiated by bluebird bio with our LentiGlobin gene therapy product candidate,” stated David Davidson, MD, bluebird bio’s Chief Medical Officer. “We are grateful for the tremendous efforts of our clinical investigators and the patients who have volunteered to undertake this pioneering work. Restoring the ability of a patient’s own blood stem cells to produce red blood cells with functional beta-globin offers the potential for a transformative, one-time treatment for this devastating disease.”
About the Northstar (HGB-204) Study
The phase 1/2 study is designed to evaluate the feasibility, safety and efficacy of LentiGlobin (BB305) drug product in the treatment of subjects with beta-thalassemia major. The study is designed to enroll up to fifteen subjects. Subjects will be evaluated for safety and efficacy post-transplant.
About beta-thalassemia major
Beta-thalassemia major is a rare hereditary blood disorder caused by a genetic abnormality of the beta globin gene resulting in defective red blood cells. Symptoms of beta-thalassemia include severe anemia, splenomegaly and iron overload in major organs. It is estimated that about 288,000 patients with beta-thalassemia major are alive, of which an estimated 15,000 live in the United States and Europe.
Read more here.