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Cobra strikes with CPI to advance regenerative medicines
KEELE, U.K.—Aimed at advancing regenerative medicine and gene therapy and riding the wave of the future, Cobra Biologics Ltd., an international contract development manufacturing organization of biologics and pharmaceuticals, is collaborating with the Centre for Process Innovation (CPI), a Cleveland, U.K.-based technology innovation center, on a game-changing project focusing on developing an industrial manufacturing platform for adeno-associated virus (AAV) production.
Led by Cobra, the £1.8-million project (approximately $2.56 million) is being funded with £1.4 ($1.99 million) from Innovate UK, sponsored by the Department for Business, Innovation and Skills, to Cobra for placing in the top five companies competing in Innovate’s 2015 competition. Innovate UK agreed to invest a total of £8 million ($11.3 million) into all five winning R&D projects addressing challenges in developing regenerative medicines and cell therapies as clinical treatments and commercial products.
Focusing on AAV vectors, currently the delivery vehicle of choice for gene therapy treatments, the proposed collaboration between Cobra and CPI will develop a manufacturing process to produce, purify and characterize a range of AAV vectors, according to Cobra Biologics.
Daniel C. Smith, chief scientific officer of Cobra Biologics, tells DDNews, “This collaborative project was designed around 12 months ago, and discussions and reiterations have been ongoing since then. Over the next three years, we will start the project fully and work through the experimental, dissemination and exploitation plans.”
Each project had to show a strong business case with defined return on investment and market understanding, along with a strong and innovative scientific challenge.
Smith believes the U.K. collaborative model for research and development is the best way to meet their lofty goals. A key difference between the United Kingdom and United States in the development and manufacture of biological material “is the underpinning science and technology required to support such development,” Smith says. “In the U.K., there is a strong and connected network of leading academic and clinical centers working closely with each other and with industry to further the understanding of bioprocessing. This is further supported via U.K. government funding supplied from scientific research councils for academic science, and Innovate UK for company innovation.”
The partnership between Cobra and CPI “will focus on AAV vectors which are safe and effective and the delivery vehicle of choice for gene therapy treatments,” Smith reiterates. “However, the advancement of these therapies into clinical trials is currently hampered by the lack of robust scalability needed to manufacture AAV vectors.”
“An industrial-scale platform, meeting regulatory expectations, can be applied to multiple AAV products—thus, de-risking and reducing process development costs and time,” Smith says. “But currently, AAV production is based on traditional laboratory processes where scale-up is complex as it relies on partially defined process criticalities and current production scales unable to meet demands for late-stage clinical trials, let alone in-market needs, for all but ultra-orphan conditions.”
For AAV, “a number of production strategies exist, including engineered producer cell lines and/or baculovirus, but these approaches require helper viruses, thus increasing the purification challenge for the final product,” he notes.
Transient triple-transfection is the chosen strategy by many providing a faster route to clinic, Smith noted, in part because it means no helper virus contamination, low initial investment and no intellectual property issues, “but scalability remains a barrier for industrialization.”
Cobra sees competitive advantages to moving from lab-based semi-defined approaches (involving high labor and material costs) to an industrial manufacturing platform (which is scalable and controlled) to produce therapeutically relevant AAV serotypes for use at all stages of clinical development and in-market supply, he explains.
The Cobra and CPI venture “aims to develop a platform approach to AAV manufacturing to support a wide range of target diseases that may require the use of AAV as a gene therapy vector,” Smith said. “At present, AAV is being evaluated as a vector to deliver genes to treat disease areas such as ocular diseases and some thalassemias.”
The hope of the partnership is to develop in-depth scientific and technical strategies to accelerate more potential products into clinical testing—and ultimately result in new approved medicines. This, in turn, will increase the chances of treatments being developed for a whole range of currently intractable diseases.
“Cobra has been a leading organization in the development of the gene therapy sector for over 15 years, now, through its plasmid DNA and viral service offerings,” Peter Coleman, CEO of Cobra Biologics, stated in a news release. “We have seen through our customers an extraordinary growth in new potential medicines being put forward that utilize viral vectors as part of their production, such as chimeric antigen receptor T cell therapies.”
Dr. Fergal O’Brien, director of biologics at CPI, stated, “CPI is delighted to be collaborating with Cobra Biologics and applying our expertise in developing scalable and industrial manufacturing platforms to AAV production. We see this project as a key enabler in meeting the current and future needs of the biologics industry, and are delighted to be supporting a leading U.K. company in this sector.”
“Since CPI began operations in 2004, we have established a track record of delivering major impact and value for the U.K. and for our partners,” adds Nigel J. Perry, CEO of CPI. “We have worked with more than 1,000 companies, delivering over £350 million of projects with an overall benefit to the U.K. of £2.4 billion. We have won more than €150 million of European projects allowing U.K. companies to work with European collaborators to develop and commercialize new products and processes. For every pound of U.K. taxpayers’ money invested in CPI, it is estimated that we return £10 to the U.K. economy.