bluebird bio starts Phase III for LentiGlobin BB305 gene therapy

bluebird bio Inc. (NASDAQ:BLUE) began the U.S. Phase III Northstar-3 (HGB-212) trial to evaluate LentiGlobin BB305 gene therapy to treat 15 patients with transfusion-dependent beta thalassemia of the beta-0/beta-0 genotype.

The single-arm, open-label study's primary endpoint is the proportion of patients with transfusion reduction, defined as at least 60% in volume of red blood cell transfusion requirements during months 12 to 24 post-transplant compared to the average annual transfusion requirement in the 24 months prior to enrollment. Secondary endpoints include transfusion independence, defined as a 12-month transfusion-free period after transplant...