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

细胞和基因疗法的治疗潜力是巨大的，治疗方法已被批准用于以前的顽固性疾病。（例如治疗血癌的Kymriah，治疗脊髓性肌萎缩症的Zolgensma和治疗遗传性视网膜营养不良的Luxturna）然而，价格仍然高得令人望而却步。限制患者获得相对罕见的适应症或狭窄的患者群体。如果细胞和基因疗法要用于治疗高度流行的疾病，那么就必须确定成本。生产效率低下是当前产品成本的一个重要组成部分。例如，目前的过程分析是由训练有素的操作人员在单个时间点进行的费力的人工过程，导致成本高，信息不完整，污染风险增加。该项目旨在通过英国和瑞士生物技术联盟与世界领先的细胞和基因治疗制造、自动化和建模来解决这个问题，以实现行业使用的过程分析技术（PAT）的改进。该项目将开发新的自动化技术，以改善细胞和基因治疗制造过程的表征和控制，最终目标是使治疗更实惠和有效。

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.