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Robust Adeno-associated Virus Purification System for Gene Therapy Development

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CPI has announced the success of its CRD IUK project to develop a scalable, cost-effective purification process for adeno-associated viruses (AAVs), in partnership with Cobra Biologics and GE Healthcare Life Sciences. The aim of the new process is to remove a significant bottleneck in the development of gene therapies and enable novel products to be brought to market more quickly.

AAVs have become the most important technology for in vivo delivery of gene therapy products, transporting genetic material into patient cells to provide a cure for otherwise untreatable diseases. However, there are significant challenges associated with AAV production and purification. The low efficiency of AAV production slows down the overall development timescale of gene therapies and increases the cost for payers in healthcare systems, reducing the availability of these innovative treatments for unmet medical needs.

The CRD IUK project was funded by a £570K grant from Innovate UK and focused on optimizing an AAV purification process using GE Healthcare Life Sciences’ innovative FibroTM chromatography material. The FibroTM material is based on electrospun cellulose nanofibers that are derivatized with different chromatography functionalities, and it overcomes both the capacity and recovery limitations of existing chromatographic supports.

The technology was known to be highly effective for purification of biomolecules such as monoclonal antibodies, and the CRD IUK project extended knowledge of the effectiveness to species the size and complexity of AAVs. Initially, CPI carried out an assessment of the FibroTM technology and generated data supporting its application to AAV purification. Results demonstrated that in spite of the size of AAV particles, high flowrates and high capacities were achievable, in turn providing high productivities and yields with small, single-use chromatography devices.

Subsequently, an industrially-relevant, robust, multistep purification process was developed for AAV purification incorporating FibroTM chromatography. This process was shown to be simple, rapid and suitable for biomanufacturing.

Daniel Smith, Chief Scientific Officer, Cobra Biologics, said: “We are delighted to have been part of this collaboration working to develop robust processes for use in the development of gene therapies. This project has provided a scalable, cost-effective fiber-based chromatography method for production of AAVs that will greatly enhance development of innovative new treatments.”

John Liddell, Chief Technologist, CPI, said: “Gene therapies have the potential to be transformative for disease areas with unmet clinical need, and effective manufacturing processes are crucial for reaching the time and cost points necessary for achieving commercialization. This was the second Innovate UK-funded project related to viral vectors for CPI and therefore further enhances the Catapult center’s ability to support growth of this emerging sector, which has been confirmed in subsequent gene therapy projects.”

Oliver Hardick, Business Leader, Puridify, GE Healthcare Life Sciences, said: “This has been an excellent collaboration with Cobra Biologics and CPI. Together, we have made a big step forward in the production of viral vectors to be used in gene therapies. The success of the project will significantly reduce the cost and time associated with development and manufacturing of AAVs, helping to accelerate delivery of gene therapy products to market.”