With our lentiviral-based gene therapy and gene editing capabilities, we have built an integrated product platform with broad potential application to severe genetic diseases and T cell-based immunotherapies for cancer. Our clinical programs include Lenti-D™, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and LentiGlobin®, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of transfusion-dependent ß-thalassemia (also known as ß-thalassemia major); a single-center Phase 1/2 study in France (HGB-205) for the treatment of transfusion-dependent ß-thalassemia or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of severe sickle cell disease (HGB-206). We also have ongoing preclinical CAR T immuno-oncology programs, as well as discovery research programs utilizing megaTAL/homing endonuclease gene editing technologies.
Our gene therapy process involves extracting certain cell types from a patient, inserting genetic material into the patient’s own cells and then re-infusing the gene-modified cells back into the patient. For beta-thalassemia, sickle cell disease and adrenoleukodystrophy, we insert a functional copy of the target gene into the patient’s hematopoietic stem cells (HSCs), enabling patients to produce proteins that are otherwise missing or dysfunctional as a result of their hereditary disease. In oncology, the gene therapy process targets a different cell type, called T-cells. In this case, genetic sequences are inserted into a patient’s own T-cells to program the T cells to specifically recognize and attack cancer cells. bluebird bio’s approach represents a highly personalized therapy and a potential paradigm shift in the treatment of severe genetic and orphan diseases.