CAMBRIDGE, Mass., March 16, 2011 – bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced that it has entered into an agreement with the French Muscular Dystrophy Association (AFM), a French non-profit entity, whereby the company will receive an initial amount of approximately $1.4 million in cash to support the development of LentiGlobin®, the company’s development-stage program for the treatment of beta-thalassemia and sickle cell anemia. As part of the research agreement, bluebird bio has the option to draw upon an additional amount of up to $2.8 million in credit toward the manufacturing of cGMP clinical trial material at Généthon.
In December 2010, bluebird bio entered into an agreement with Généthon designed to enable substantial advances in the existing manufacturing process of lentiviral vectors for the benefit of both partners.
bluebird bio’s LentiGlobin introduces a fully functional human beta-globin gene, under the control of the beta-globin promoter and locus control regions, into the patient’s own hematopoietic stem cells. bluebird bio is currently conducting a Phase 1/2 trial examining the feasibility, safety and efficacy of LentiGlobin in the treatment of beta-thalassemia and sickle cell anemia. Based on clinical data published in the September 2010 issue of Nature, LentiGlobin therapy has shown the potential to eliminate the need for monthly blood transfusions in patients with beta-thalassemia, without the risk of graft-versus-host disease.
“This funding from the AFM will not only support our ongoing thalassemia and sickle cell clinical trial, but also signifies the beginning of an important collaboration with the AFM,” said Nick Leschly, president and chief executive officer of bluebird bio. “We are grateful for the AFM’s commitment to the advancement of a treatment that has the potential to greatly improve the lives of patients.”
Beta-thalassemia is an inherited blood disorder that is named for defects in production of the beta-globin chain of hemoglobin, the protein in red blood cells that carries oxygen. Approximately 60,000 children are diagnosed with the disease each year throughout the world. Patients typically require monthly supportive red blood cell transfusions to treat their severe anemia for life. Sickle cell anemia is characterized by clotting of improperly shaped red blood cells, which leads to a wide variety of serious health problems including chronic pain and high
risk for stroke. Sickle cell anemia affects millions throughout the world, including approximately 95,000 people in the United States.