A partnership between the Centre for Process Innovation (CPI), Cobra Biologics and GE Healthcare Life Sciences has resulted in the development of a scalable, cost-effective purification process for adeno-associated viruses (AAVs). The CRD IUK project has created a new process that will remove a significant bottleneck in the development of gene therapies, and enable these innovative products to be brought to market more quickly.
Daniel Smith, Chief Scientific Officer of Cobra Biologics, said: "This project has provided a scalable, cost-effective fibre-based chromatography method for production of AAVs that will greatly enhance development of innovative new treatments."
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 (US$732K) grant from Innovate UK and focused on optimising an AAV purification process using GE Healthcare Life Sciences' Fibro chromatography material.
The project focused on optimising an AAV purification process using GE Healthcare Life Sciences' Fibro chromatography material
The Fibro material is based on electrospun cellulose nanofibers that are derivatised 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 Fibro 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 Fibro chromatography. This process was shown to be simple, rapid and suitable for biomanufacturing.
John Liddell, Chief Technologist at 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 commercialisation."
Liddel explained that this was the second Innovate UK-funded project related to viral vectors for CPI and further enhances the Catapult centre's ability to support growth of this emerging sector, which has been confirmed in subsequent gene therapy projects. CPI forms part of the High Value Manufacturing Catapult but its network and partnerships flow across the entire Catapult network.