Advanced process control of AAV manufacture: digitalisation, PAT and automation to improve productivity, quality and consistency

AAV 制造的先进流程控制：数字化、PAT 和自动化，以提高生产率、质量和一致性

• 批准号: 10064155
• 金额: $ 127.26万
• 依托单位: RENTSCHLER ATMP LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2023
• 资助国家: 英国
• 起止时间: 2023 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10064155
• 关键词: Advanced; process; control; AAV; manufacture

The therapeutic potential for gene therapies is transformative, where many largely intractable diseases will be treated safely, effectively and durably. Therapies are now starting to get approved (e.g. Roctavian, Zolgensma, and Luxturna), but cost remains a large barrier to entry and widespread patient access, partly due to suboptimal manufacturing processes. If gene therapies are ever going to be used to treat highly prevalent diseases, then breakthroughs need to be made in manufacturing.One area in need of improvement is process analytical technology (PAT) and continuous process monitoring. Current methods rely on laborious off-line analysis performed by highly trained operators at discrete time-points which introduces significant costs, delays and increases the chances of contamination. This project will use next-generation automation and novel PAT technologies to enable real-time characterisation and control of the manufacturing process.

基因疗法的治疗潜力是变化的，其中许多棘手的疾病将被安全，有效，持久地治疗。现在，疗法开始获得批准（例如Roctavian，Zolgensma和Luxturna），但成本仍然是一个很大的入境障碍和广泛的患者进入，部分是由于次优的制造过程所致。如果要使用基因疗法来治疗高度普遍的疾病，则需要在制造中取得突破。需要改进的一个领域是过程分析技术（PAT）和持续过程监测。当前的方法依赖于训练有素的运营商在离散的时间点上进行的费力离线分析，从而引入了巨大的成本，延误并增加了污染的机会。该项目将使用下一代自动化和新颖的PAT技术来实时表征和控制制造过程。