bluebird bio initiates phase 3 trial for beta-thalassemia

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bluebird bio, a clinical-stage company specialized in developing lentiviral-based gene therapies for severe genetic diseases, has announced the initiation of its phase 3 gene therapy trial in patients with transfusion-dependent beta-thalassemia.

The study, HGB-207, is a phase 3, global, multi-center study designed to evaluate the safety and efficacy of gene therapy in subjects with transfusion-dependent beta-thalassemia by transplantation of autologous CD34+ cells transduced ex vivo with a lentiviral β-A(T87Q)-globin vector (LentiGlobin BB305).

The trial will include the addition of two transduction enhancers to the hematopoietic stem cell (HSC) manufacturing process to increase transduction efficiency. The rationale for this modified protocol arises from accumulating clinical data that shows a correlation between vector copy number, percentage of vector-containing cells and the amount of hemoglobin produced by patients treated with LentiGlobin. The study will be conducted under the same investigational new drug application as other studies of LentiGlobin in beta-thalassemia. The primary endpoint of the study is transfusion independence, defined as a 12-month transfusion free period after HSC transplantation. This study is intended to be pivotal in the US and confirmatory in the EU.

LentiGlobin BB305 is currently in three clinical studies for the treatment of transfusion-dependent beta-thalassemia and severe sickle cell disease.

Dr David Davidson, bluebird’s chief medical officer commented: “We believe that the addition of our transduction enhancers to our manufacturing process has the potential to substantially increase the hemoglobin levels in patients with transfusion dependent beta-thalassemia and increase their likelihood of achieving clinically meaningful reductions in transfusion requirements or transfusion independence, the ultimate goal of our therapy.”

Source: bluebird bio Opens Phase 3 Study of LentiGlobin™ Drug Product in Patients with Transfusion-Dependent Beta-Thalassemia; Press Release