let's recode the science
our science
We are leading the gene therapy revolution. Our integrated product platforms encompass gene therapy and gene editing — providing us with the potential to treat a broad range of serious diseases with the potential to provide lifelong benefits.
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 treatment option for patients with severe genetic and rare diseases. At the core of this effort is our collective determination to provide these individuals 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 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
Our investigational gene therapy process is intended to genetically modify a patient’s own cells. We focus on the development of vector delivery systems for a potent gene therapy platform with potentially broad applications in a wide variety of indications where significant medical need remains.
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 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.
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, delivery method, 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, the goal being 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 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 potential therapeutic applications.