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The companies will collaborate to identify and develop in-vivo genome editing therapeutic candidates for genetic diseases, including hemophilia.
Novo Nordisk and bluebird bio announced on Oct. 9, 2019 that they have entered into a three-year research collaboration to jointly develop next-generation in-vivo genome editing treatments for genetic diseases, including hemophilia. During the three-year research collaboration, the companies will focus on identifying and developing a gene therapy candidate for treating hemophilia A.
The collaboration will use bluebird bio’s proprietary mRNA-based megaTAL technology, which has the potential to provide a highly specific and efficient way to silence, edit, or insert genetic components. Aligned with Novo Nordisk’s hemophilia portfolio, the collaboration will initially focus on correcting factor VIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.
“We are pleased to announce our collaboration with bluebird whose demonstrated capabilities in gene therapy will enable the next-generation of innovative products to make a significant impact on patients’ lives,” said Marcus Schindler, senior vice-president for Global Drug Discovery at Novo Nordisk, in a company press release. “This important research collaboration aimed at addressing genetic diseases at the DNA level reflects Novo Nordisk’s enduring commitment and dedication to inventing disease-modifying medicines that can truly change the lives of people living with hemophilia and other genetic diseases.”
“bluebird has made tremendous progress on enabling an in-vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification,” said Philip Gregory, chief scientific officer, bluebird bio, in the press release. “We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases. Moreover, we are thrilled to be able to combine this new platform technology with Novo Nordisk’s deep expertise in hemophilia research and therapeutics. We believe this collaboration will move us toward our shared goal of recoding the treatment paradigm and substantially reduce the burden of disease for patients with factor VIII deficiency.”
MegaTALs are a single-chain fusion enzyme that combines the natural DNA cleaving processes of homing endonucleases with the DNA-binding region of transcription activator-like (TAL) effectors. TALs are easily engineered proteins that recognize specific DNA sequences. This protein fusion architecture allows the generation of extremely active and highly specific and compact nucleases that are compatible with all current viral and non-viral cell delivery methods.
Source: Novo Nordisk