Research & Development

our science

We are leading the gene therapy revolution. Our integrated product platforms encompass gene therapy, cancer immunotherapy and gene editing – providing us with the potential to treat, and hopefully cure, a broad range of serious diseases.

integrated product platforms

Our objective is to develop and bring to market the most advanced products based on the transformative potential of gene therapy as a one-time treatment option for patients with severe genetic and rare diseases and cancer. At the core of this effort is our collective determination to provide these patients hope for a better life in the face of limited or no long-term safe and effective treatment options.

gene therapy

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 gene therapy technology intends to make up for genetic problems by creating new copies of genes that express functional proteins and thereby correct or address the underlying cause of the disease. We believe our approach to gene therapy has the potential to provide transformative, disease-modifying effects – potentially with life-long clinical benefits after a single stem cell transplant.

how our gene therapy platform works

Our investigational gene therapy process works by genetically modifying a patient’s own cells. We have extensive expertise in the design and manufacturing of vector delivery systems, which we have developed into a potent gene therapy platform with potentially broad applications in a wide variety of indications where significant medical need remains.

cancer immunotherapy

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 attach 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 liquid and solid tumor cancers.

CAR T program: leveraging our gene therapy expertise to treat cancer

Our gene therapy technology seeks to genetically modify 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 kill them.

gene editing

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 safer and more potent cell-based products using this technology could help provide curative, one-time treatments 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.

why megaTALs?

All the gene editing technologies currently being explored by the pharmaceutical industry, including zinc finger nucleases, CRISPR (clustered, regularly interspaced short palindromic repeats)/Cas9 (CRISPR associated protein 9) and TALENs (transcription activator-like effector nucleases), perform DNA recognition and DNA cutting functions. They differ in specificity, size, ease of delivery, and the detailed biochemistry that underlies their DNA recognition, cleavage, and repair mechanisms. Homing endonucleases are the only monomeric, naturally occurring proteins to bind and cleave DNA in a highly sequenced specific fashion. megaTALs are fusion proteins that combine homing endonucleases with the modular DNA binding domains of TALENs, resulting in precise DNA sequence targeting. Since these hybrid nucleases still cut DNA using homing endonuclease cleavage biochemistry, they engage DNA repair pathways in a manner distinct from all other gene editing nucleases. The compact format and ultra-efficient nature of our nucleases make them powerful tools in our ongoing effort to build advanced gene editing processes and products for a broad range of therapeutic applications.