bluebird bio

Our current platform is the beginning of a continuum of investment in next generation gene therapy technologies and capabilities. We have licensed or developed proprietary technologies, processes and products to design, produce and deliver high quality gene therapy products at scale.
Recent advances in our platform have improved fundamental aspects of gene therapy such as vector purity, potency and scalability, as well as overall transduction efficiency, something we call the "industrialization of gene therapy." These advances will allow us to develop first and best in class products that deliver high expression levels of missing or dysfunctional proteins and that are scalable for commercial production and application. These improvements have the potential to provide increased therapeutic benefit in upcoming beta-thalassemia and CCALD trials and to enable the expansion of clinical programs into a broader set of severe genetic and orphan diseases, including sickle cell disease and oncology. The strength of our platform provides for broad therapeutic potential beyond its core indications.
As a result of the broad potential application of our platform, we established a global strategic collaboration with Celgene Corporation in March 2013 to discover, develop and commercialize novel disease-altering gene therapies in oncology by utilizing a patient’s own genetically modified T cells, known as chimeric antigen receptor (CAR) T cells, to selectively target and destroy cancer cells.