Novo Nordisk, Bluebird Bio Partner to Develop Gene TherapiesBy
Novo Nordisk and Bluebird Bio, a developer of gene therapies for severe genetic diseases and cancer, have entered into a three-year research collaboration to jointly develop in vivo genome-editing treatments for genetic diseases, including hemophilia.
During the three-year research collaboration, Bluebird and Novo Nordisk will focus on identifying a gene-therapy candidate for treating people with hemophilia. Hemophilia is the result of decreased or defective production of the blood-clotting Factor VIII (FVIII). The collaboration will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.
The collaboration will use Bluebird’s proprietary mRNA-based megaTAL technology that has the potential to provide a highly specific way to silence, edit or insert genetic components. MegaTALs are a single-chain fusion enzyme that combine the natural DNA cleaving processes of homing endonucleases with the DNA binding region of transcription activator-like effectors. These are easily engineered proteins that recognize specific DNA sequences. This protein-fusion architecture allows the generation of active and highly specific and compact nucleases that are compatible with current viral and non-viral cell delivery methods, according to information from the companies.