let's recode the science
Integrated product platforms
Each person inherits features in the form of genes, which are made up of a molecule called DNA. Genetic diseases involve problems with the functioning of certain genes. Our investigational gene therapy technology intends to make up for genetic problems by introducing new genes that express functional proteins and thereby addressing the disease at the genetic level. We believe our approach to gene therapy has the potential to provide transformative, disease-modifying effects.
our gene therapy
We are leveraging our core expertise in gene transfer technology and our experience in implementing gene therapy clinical trials to build a broad, fully integrated immuno-oncology franchise starting with T cell therapies that seek to help the body’s own immune system attack and destroy cancer cells.
Our cancer immunotherapy research group is expert in the latest innovations in T cell engineering and is creating a pipeline of T cell product candidates to treat a wide variety of blood cancers and solid tumors.
Our gene therapy technology seeks to genetically modify specific cells from a patient’s own immune system (T cells) to target and destroy cancer cells. Like our programs for hematopoietic stem cells (blood-producing stem cells) in severe genetic disorders, the goal of our CAR T technology is to customize a lentiviral vector to alter T cells so the T cells can recognize specific proteins on the surface of cancer cells and destroy them.
Gene editing has generated a lot of excitement in academia and drug development. Emerging science suggests that permanently fixing or “editing” mutated genes or creating potent cell-based products using this technology may provide treatment at the genetic level for patients suffering from a broad range of diseases.
bluebird’s expert gene editing team is expanding our discovery research efforts in this emerging field. We are focused on homing endonuclease and megaTAL gene editing technologies in a variety of potential applications and disease areas. Reprogrammed homing endonucleases and megaTALs are novel enzymes that provide a highly specific and efficient way to potentially treat a variety of diseases by silencing, editing or inserting genetic components into a cell.