Gene Therapy - True Personalized Medicine
Our objective is to develop and commercialize a next generation of products based on the transformative potential of gene therapy as a one-time treatment option for patients with severe genetic and rare diseases. Central to this effort is a collective determination within our company to provide these patients with hope for a better life in the face of limited or no long-term safe and effective treatment options.
Our approach represents highly personalized, important new therapeutic modalities for patients with severe genetic and rare diseases with significant unmet medical need. We have clinical-stage programs in development for childhood cerebral adrenoleukodystrophy, beta-thalassemia and sickle cell disease, as well as a preclinical oncology program in the chimeric antigen receptor (CAR) T cells field under a collaboration with Celgene Corporation and early stage programs using gene editing capabilities through the recent acquisition of Pregenen.
Gene therapy has the potential to change the way patients are treated by correcting the underlying genetic defect that is the cause of their disease or by providing additional functionality that can eliminate the disease, rather than offering solutions that only address their symptoms. At bluebird bio, we believe gene therapy has the potential to provide transformative disease modifying effects with life-long clinical benefits and we are advancing several gene therapy technologies.
Lentiviral Gene Transfer
Our lentiviral gene therapy process involves inserting genetic material into the patient's own cells ex vivo, then re-introducing the cells to the patient. For beta-thalassemia, sickle cell disease and adrenoleukodystrophy, we insert a functional copy of the malfunctioning gene into the patient's hematopoietic stem cells (HSCs), with the goal of genetically modifying a patient's own cells to fundamentally correct or address the genetic basis underlying a disease.
In oncology, the gene therapy process targets a different cell type, called T cells. Chimeric antigen receptor (CAR) T cell therapy represents a promising, emerging approach to treating cancer. Blood is withdrawn from a patient and the T cells are extracted and genetically modified to recognize and attack cancer cells. The modified cells are re-introduced into the patient's blood to selectively bind and kill the cancer cells.
As a result of the recent acquisition of gene editing pioneer, Pregenen, we have now have access to technologies known as Homing Endonucleases (HEs) and MegaTALs, which have broad potential application in the gene therapy and cancer immunotherapy fields including application to both Hematopoietic Stem Cells (HSCs) and CAR T cells. HEs and MegaTALs are part of the broad class of emerging gene editing technologies that offer the potential to create customized constructs that specifically target DNA sequences within a cell to correct specific gene mutations, to functionally regulate gene expression, and to insert genes at specific sites. These technologies provide us with access to new potential targets and opportunities to transform medicine.