Next-generation automation and PAT implementation for QbD and enhanced approaches for cell and gene therapy

The therapeutic potential for cell and gene therapies is immense, with treatments approved for previously intractable diseases. (e.g. Kymriah for blood cancers, Zolgensma for spinal muscular atrophy, and Luxturna for inherited retinal dystrophy) However, the price remains prohibitively high. Limiting patient access to relatively rare indications or narrow patient populations. If cell and gene therapies are going to be used to treat highly prevalent diseases, then costs must come done. Inefficiencies in manufacturing are a significant component of the current product cost. For example, currently, analysis of the process is a laborious manual process conducted by highly trained operators at single time points, leading to high costs, patchy information and increased contamination risks. This project aims to address this problem by together UK and Swiss biotechnology consortium with world leaders in cell and gene therapy manufacturing, automation and modelling to implement improvement in the process analytical technology (PAT) used by the industry. This project will develop new automation to improve the characterisation and control of cell and gene therapy manufacturing processes, ultimately with the goal of making the therapies more affordable and effective.