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news.bluebird bio has entered into an agreement with the French Muscular Dystrophy Association (AFM), under which the company will receive an initial amount of approximately $1.4m in cash to support the development of LentiGlobin, for the treatment of beta-thalassemia and sickle cell anemia.
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As part of the research agreement, bluebird bio has the option to draw upon an additional amount of up to $2.8m in credit toward the manufacturing of cGMP clinical trial material at Genethon.
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.