The growing interest in viral vectors for gene therapy requires efficient and cost-effective manufacturing platforms for industrialization. Significant improvements have already been recorded over the last decade. However, robust and large-scale production technologies that meet current Good Manufacturing Practices (cGMP) are still critically needed.<br/>Viral vector delivery systems, of which mainly adeno-associated viruses (AAV) and lentiviruses-based are mainly produced by transient transfection of mammalian producer HEK-293 cell lines. Virus vector production using the right transient transfection method is crucial to provide the flexibility and reproducibility needed to scale-up from initial process development up to large scale manufacturing of high-quality grade viral vectors.<br/>Here we describe PEIpro®, an optimized PEI-based transient transfection system that utilizes our long standing cationic polymer expertise combined with advances in manufacturing platform technologies for large scale and high-yielding viral vector production. We demonstrate the robustness, scalability and reliability of our PEI-based transient gene expression approach to manufacture GMP-grade viral vectors at a sufficiently large scale for more advanced clinical trials, and <i>in fine</i> for commercialization. To address the requirement for the release of a Final Drug Product: (control and detection of residual levels of any raw material/impurities), we have developed a proprietary analytical method tailored for detection and quantification of residual PEIpro® throughout downstream purification processes up to the Final Drug Product.

Efficiency, quality and traceability of PEIpro transfection reagent for clinical grade viral vector manufacturing

2019 Spring National Meeting

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