21 June 2018 - - Cambridge, Massachusetts-based gene therapies specialist bluebird bio, Inc. (NASDAQ: BLUE) has presented new data from the completed phase 1/2 Northstar study in adolescents and adults with transfusion-dependent β-thalassemia (TDT) and any genotype, and the ongoing phase 3 Northstar-2 multicenter clinical study of LentiGlobin investigational gene therapy in patients with TDT and non-β0/β0 genotypes, at the Annual Congress of the European Hematology Association, the company said.
The primary endpoint is the proportion of treated subjects who meet the definition of transfusion independence, defined as total hemoglobin levels of at least 9 g/dL without any red blood cell transfusions for a continuous period of at least 12 months at any time during the study.
The studies showed 7/8 non-β0/β0 patients with ≥ 6 months follow-up were producing normal or near-normal amounts of total hemoglobin and are transfusion free. Also 8/10 of non-β0/β0 patients were achieving and maintaining transfusion independence for up to 3 years.
The completed Phase 1/2 Northstar study was an open-label, single-dose, non-randomized, multi-center study designed to evaluate LentiGlobin in increasing hemoglobin production and eliminating or reducing transfusion dependence in subjects with TDT. The study treated 18 adults and adolescents who are being followed to evaluate safety and efficacy following LentiGlobin infusion.
Northstar-2 is a Phase 3 global, multi-center study designed to evaluate LentiGlobin drug product in patients with TDT and non-β0/β0 genotypes. For this study, the manufacturing process by which the patient's cells are transduced with the LentiGlobin viral vector has been improved, with the intent of increasing vector copy number and the percentage of cells successfully transduced.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. The company has operations in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina; and Zug, Switzerland.