Scalable AAV production in high density suspension HEK293SF-3F6 cell culture using Nanocin transfection

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Viral vectors are central to the gene therapy revolution of medicine and have demonstrated transformative patient benefits. The manufacturing of viruses is inherently complex, where a key challenge is the introduction of virus encoding DNA into producer cells grown in culture. This area of high-value manufacturing is exciting, strategically important, and currently transforming as manufacturers develop innovative processes. This project joins a rapidly growing UK SME with a world-leading bioprocess development centre. This partnership complements strengths and a joint motivation to develop an improved solution for adeno- associated virus (AAV) production, which can be considered to be a "work horse" within the gene therapy area. The aim is to develop flexible processes that are more efficient, less expensive, resilient to supply challenges, and compatible with customers' existing facilities and long-term manufacturing plans. We will reach our goals by combining and building on previous work in the area-field.The project will use the industry-leading PEI product (Polyplus;EU/US), as a benchmark, where we aim to exploit the advantageous properties of Nanocin and develop production processes in a scalable format that is compatible with clinical applications. The low toxicity properties of Nanocin enable repeat transfection (DNA delivery), and such advances, integrated into commercial bioreactors can provide step changes in manufacturing efficiencies and cost reductions.Benefits will flow to Tecrea and the UK high-value advanced medicines manufacturing industry, both regionally (in terms of potential clustering) and nationally. NRC will benefit by advancing its bioprocessing capacities and most importantly improve the quality and cost of gene therapies to the extent that they can have a substantial public health benefit within Canada.