A shared scalable suspension platform for cost effective manufacture of novel AAV gene therapies

一个共享的可扩展悬浮平台，用于经济有效地制造新型 AAV 基因疗法

• 批准号: 104518
• 金额: $ 143.78万
• 依托单位: GYROSCOPE THERAPEUTICS LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2018
• 资助国家: 英国
• 起止时间: 2018 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=104518
• 关键词: shared; scalable; suspension; platform; cost

"The rapid development of Gene Therapy Medicinal Products (GTMPs) requires new manufacturing processes and facilities. Gyroscope Therapeutics Ltd and Freeline Therapeutics Ltd are UK-based SMEs developing gene therapies for different indications but which are both delivered via adeno-associated viral (AAV) vectors. Gyroscope and Freeline intend to co-develop an innovative, suspension-based viral vector manufacturing process, which will provide a shared platform for the manufacture of each company's GTMP products. The major challenge facing commercialisation of gene therapies is the manufacture and supply of products at a commercial scale. Gyroscope and Freeline will develop a proprietary, serum-free, scalable, suspension based manufacturing platform, which will improve product quality, safety, robustness and reduce the costs of goods at commercial scale. Working together with process development specialists at partner organisations the Centre for Clinical Biotechnology (CBC) and the Centre for Process Innovation (CPI), Gyroscope and Freeline will:1. Establish ways to reduce risk of genetic instability of plasmid starting materials in an integrated approach;2. Establish a proprietary animal component free and 'chemically defined' suspension cell culture manufacturing process which is scalable and which minimises product heterogeneity while maximising safety, quality and yield;3. Supply batches of material to confirm comparability of product made using suspension platform material to that produced by current adherent cell culture platforms and so enable process changes to be implemented4. Generate data to facilitate technology transfer of the developed process at a representative scale to GMP manufacturing facilities for subsequent qualification as a GMP manufacturing process.Ultimately, a successful outcome will address a significant manufacturing challenge for GTMPs, strengthen the UK position as a leader in scientific innovation, offer scope for investment and employment into UK Pharmaceutical manufacturing and bring healthcare benefits to patients."

“基因治疗药品（GTMPs）的快速发展需要新的生产工艺和设施。Gyroscope Therapeutics Ltd和Freeline Therapeutics Ltd是总部位于英国的中小企业，为不同的适应症开发基因疗法，但都是通过腺相关病毒（AAV）载体提供的。Gyroscope和Freeline打算共同开发一种创新的、基于悬浮液的病毒载体制造工艺，这将为两家公司的GTMP产品的制造提供一个共享平台。基因疗法商业化面临的主要挑战是以商业规模生产和供应产品。Gyroscope和Freeline将开发一个专有的、无血清的、可扩展的、基于悬浮液的制造平台，这将提高产品质量、安全性、稳健性，并降低商业规模的商品成本。临床生物技术中心（CBC）和工艺创新中心（CPI）、Gyroscope和Freeline与合作组织的工艺开发专家合作，将：1。建立以综合方法降低质粒起始材料遗传不稳定性风险的方法;2.建立专有的无动物成分和“化学定义”的悬浮细胞培养生产工艺，该工艺可扩展，并在最大限度地提高安全性、质量和产量的同时最大限度地减少产品异质性;3.提供材料批次，以确认使用悬浮平台材料生产的产品与当前贴壁细胞培养平台生产的产品的可比性，从而实现工艺变更4。生成数据，以促进技术转让的开发过程中，在一个代表性的规模，以GMP生产设施，随后作为一个GMP生产工艺的资格。最终，一个成功的结果将解决一个重大的生产挑战，GMP，加强英国作为一个领导者的地位，在科学创新，提供投资和就业的范围到英国制药业，并带来医疗保健的好处，病人。"