Large-scale microfluidic bioprocessing platform for cell and gene therapy manufacturing

用于细胞和基因治疗制造的大型微流控生物处理平台

• 批准号: 105770
• 金额: $ 61.77万
• 依托单位: MICROFLUIDX LTD
• 依托单位国家: 英国
• 项目类别: Study
• 财政年份: 2020
• 资助国家: 英国
• 起止时间: 2020 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=105770
• 关键词: Large; scale; microfluidic; bioprocessing; platform

MicrofluidX develops and manufactures a system for the production of cell and gene therapies (CGTs). CGTs are an advanced therapy which relies upon complex biological systems (cells, viruses or genetic vector systems) to perform corrective action in patients. This technology has demonstrated an unparalleled healing potential, from multiple types of cancers, to diabetes, and rare diseases. These emerging therapies substantially differ from existing drugs or biologics in that they are a live product with unique development and manufacturing considerations. Existing manufacturing solutions have largely relied on modifying existing biopharma approaches which do not always meet the conditions these therapies require for effective manufacturing. Typically, these therapies are extremely expensive and difficult to produce and average a list price of \>$300k per treatment. These cost constraints, together with the limited production capacity, force public health systems like the NHS to restrict the use to a limited number of patients, and will prevent or delay certain treatments reaching the market if the price points are too high. Key to enabling access to these treatments is therefore to develop a low-cost, robust and scalable manufacturing platform. MicrofluidX is developing a system that cuts costs by up to 90%, enables faster scale-up from discovery to market, and provides more control over manufacturing parameters for a safer end product.

MicrofluidX开发和制造用于生产细胞和基因疗法（CGT）的系统。CGT是一种先进的疗法，它依赖于复杂的生物系统（细胞、病毒或遗传载体系统）来对患者进行纠正。这项技术已经证明了无与伦比的治疗潜力，从多种类型的癌症到糖尿病和罕见疾病。这些新兴疗法与现有药物或生物制剂有很大不同，因为它们是具有独特开发和制造考虑的活产品。现有的制造解决方案在很大程度上依赖于修改现有的生物制药方法，这些方法并不总是满足这些疗法有效制造所需的条件。通常情况下，这些疗法是非常昂贵的，很难生产，平均价格为每治疗30万美元。这些成本限制，加上有限的生产能力，迫使像NHS这样的公共卫生系统将使用限制在有限数量的患者中，如果价格过高，将阻止或延迟某些治疗进入市场。因此，实现这些治疗的关键是开发一个低成本，强大和可扩展的制造平台。MicrofluidX正在开发一种系统，可将成本降低高达90%，实现从发现到市场的更快扩展，并为更安全的最终产品提供更多的制造参数控制。