Northstar study: gene therapy for beta-thalassemia major using LentiGlobin BB305 drug product

Press brief by Mark C. Walters, MD, at the American Socitety of Hematology (ASH) 2015 annual meeting. Dr Walters provides an update on the Northstar study of subjects treated with the LentiGlobin BB305 Drug Product. Previously reported early results in subjects with ?-thalassemia major participating in the ongoing HGB-205 and HGB-204 (Northstar) studies suggest that transplantation with autologous CD34+ cells transduced with a replication-defective, self-inactivating LentiGlobin BB305 lentiviral vector containing an engineered ?A-T87Q?globin gene (LentiGlobin BB305 Drug Product) has a positive safety profile and leads to ?A-T87Qglobin production and can lead to transfusion independence.