Scalable AAV production in high density suspension HEK293SF-3F6 cell culture using Nanocin transfection

使用 Nanocin 转染在高密度悬浮 HEK293SF-3F6 细胞培养物中大规模生产 AAV

• 批准号: 10004533
• 金额: $ 12.21万
• 依托单位: TECREA LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2021
• 资助国家: 英国
• 起止时间: 2021 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=10004533
• 关键词: Scalable; AAV; production; density; suspension

Viral vectors are central to the gene therapy revolution of medicine and have demonstrated transformative patient benefits. The manufacturing of viruses is inherently complex, where a key challenge is the introduction of virus encoding DNA into producer cells grown in culture. This area of high-value manufacturing is exciting, strategically important, and currently transforming as manufacturers develop innovative processes. This project joins a rapidly growing UK SME with a world-leading bioprocess development centre. This partnership complements strengths and a joint motivation to develop an improved solution for adeno- associated virus (AAV) production, which can be considered to be a "work horse" within the gene therapy area. The aim is to develop flexible processes that are more efficient, less expensive, resilient to supply challenges, and compatible with customers' existing facilities and long-term manufacturing plans. We will reach our goals by combining and building on previous work in the area-field.The project will use the industry-leading PEI product (Polyplus;EU/US), as a benchmark, where we aim to exploit the advantageous properties of Nanocin and develop production processes in a scalable format that is compatible with clinical applications. The low toxicity properties of Nanocin enable repeat transfection (DNA delivery), and such advances, integrated into commercial bioreactors can provide step changes in manufacturing efficiencies and cost reductions.Benefits will flow to Tecrea and the UK high-value advanced medicines manufacturing industry, both regionally (in terms of potential clustering) and nationally. NRC will benefit by advancing its bioprocessing capacities and most importantly improve the quality and cost of gene therapies to the extent that they can have a substantial public health benefit within Canada.

病毒载体是医学基因治疗革命的核心，已经证明了对患者的变革性益处。病毒的制造本身就很复杂，其中一个关键的挑战是将编码DNA的病毒引入培养的生产细胞中。这个高价值制造领域令人兴奋，具有重要的战略意义，目前随着制造商开发创新工艺而发生变化。该项目加入了一个快速发展的英国中小企业与世界领先的生物工艺开发中心。这种伙伴关系互补了双方的优势和共同动机，以开发一种改进的腺相关病毒（AAV）生产解决方案，这可以被认为是基因治疗领域的“主力”。其目标是开发更高效、更便宜、对供应挑战有弹性的灵活工艺，并与客户现有设施和长期制造计划相兼容。我们将通过结合和发展以前在该领域的工作来实现我们的目标。该项目将使用业界领先的PEI产品（Polyplus；欧盟/美国）作为基准，我们的目标是利用Nanocin的优势特性，并以可扩展的格式开发与临床应用兼容的生产工艺。Nanocin的低毒特性使其能够重复转染（DNA传递），并且将这些进步整合到商业生物反应器中可以在制造效率和成本降低方面提供逐步变化。利益将流向Tecrea和英国的高价值先进药品制造业，无论是区域性的（就潜在集群而言）还是全国性的。NRC将受益于提高其生物处理能力，最重要的是提高基因疗法的质量和成本，使其能够在加拿大境内产生重大的公共卫生效益。