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

QbD 的下一代自动化和 PAT 实施以及细胞和基因治疗的增强方法

• 批准号: 10087446
• 金额: $ 50.8万
• 依托单位: CELL THERAPY CATAPULT SERVICES LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2024
• 资助国家: 英国
• 起止时间: 2024 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10087446
• 关键词: Next; generation; automation; PAT; implementation

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.

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难治性疾病。(e.g. Kymriah治疗血癌，Zolgensma治疗脊髓性肌萎缩症，Luxturna治疗遗传性视网膜营养不良）然而，价格仍然高得令人望而却步。限制患者进入相对罕见的适应症或狭窄的患者人群。如果要用细胞和基因疗法来治疗高度流行的疾病，那么成本就必须降低，制造业的低效是当前产品成本的重要组成部分。例如，目前，该过程的分析是由训练有素的操作员在单个时间点进行的费力的手动过程，导致成本高、信息不完整和污染风险增加。该项目旨在通过英国和瑞士生物技术联盟与细胞和基因治疗制造，自动化和建模领域的世界领导者一起解决这一问题，以改进该行业使用的过程分析技术（PAT）。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加实惠和有效。