Bluebird bio will receive up to $4.2 million from the French Muscular Dystrophy Association (AFM) toward continued development of LentiGlobin®. The gene therapy is currently in Phase I/II trials as a treatment for beta thalassemia and sickle cell anemia.
Clinical data published in the September 2010 issue of Nature demonstrated that LentiGlobin has potential to eliminate the need for monthly blood transfusions in patients with beta-thalassemia without the risk of graft-versus-host disease, bluebird bio reports.
The funding consists of an initial $1.4 million in cash as well as up to $2.8 million in credit toward the manufacturing of cGMP clinical trial material at AFM’s Généthon research lab. In December, bluebird bio entered into an agreement with Généthon designed to enable substantial advances in the existing manufacturing process of lentiviral vectors for use by both.
Bluebird bio, formerly Genetix Pharmaceuticals, focuses on developing gene therapies for severe genetic disorders, inserting a healthy version of a disease-causing gene using stem cells harvested from a patient’s bone marrow. Last year, bluebird bio completed a $35 million series B financing round.
AFM currently funds 36 clinical trials for 30 different genetic diseases including diseases of the eye, blood, brain, immune system, and muscle. That research is largely funded through donations from France's annual Telethon, which raised €95 million (about $132.5 million) in 2009.