Development of an Innovative Supply Chain Model to Expedite Delivery of Viral Vector ATIMPs to Clinical Trials

“被称为先进疗法的新疗法的快速发展，如细胞疗法和基因疗法，需要全球范围内的新制造设施和技术。人类使用的基因治疗病毒载体的生产需要专门的高质量生产设施，目前英国和全球的这种能力不足。依赖海外合同管理组织在能力和缺乏灵活性方面都带来了挑战。细胞和基因治疗Catapult最近在Stevenage开设了制造中心，为Freeline等小型基因治疗公司提供创新的成本分摊模式，以建立其药物的内部制造能力。该项目的目的是通过建立CGTC-MC和Symbiosis（一家专门从事药品生产的英国公司）之间的综合、精益合作伙伴关系，进一步发展英国的供应链（灌装完成）用于临床无菌药品和小体积，利基商业无菌产品。Freeline，CGTC-MC和Symbiosis之间的合作旨在建立一个高效，安全的供应链，可靠地将病毒载体基因治疗药物产品交付临床试验。这将加快新的基因疗法向患者提供的步伐。该项目提供的临床供应链将为未来提供基因治疗产品的商业供应奠定基础，从而建立一个安全的英国嵌入式商业供应链。三家英国合作伙伴将共同努力创建新的流程和分析技术，以实现合作伙伴之间快速无缝的材料转移，同时保持质量。该项目将包括调整各自的质量体系和流程，以及投资于内部化关键质量控制测试和建立新的新型快速方法。内部化的质量控制将带来流程效率，增加供应链的安全性。新的整合运营模式将通过生产和供应Freeline基因治疗药物的GMP批次用于治疗溶酶体贮积症的I/II期临床试验来证明。由此产生的供应链和技术将转化为标准流程，CGT-MC和Symbiosis可以向其他未来的病毒载体客户提供，从而显着提高英国的病毒载体制造能力。

"The rapid development of novel treatments called Advanced Therapies, such as cell therapies and gene therapies have required new manufacturing facilities and technologies worldwide. Manufacturing of gene therapy viral vectors for human use requires specialised high-quality manufacturing facilities and there is currently insufficient such capacity in the UK and globally. Reliance on overseas CMOs presents challenges in terms of both capacity and lack of flexibility. The Cell and Gene Therapy Catapult has recently opened its manufacturing centre in Stevenage offering an innovative cost-sharing model for small gene therapy companies, such as Freeline, to establish an internal manufacturing capability for their drug substance. The aim of this project is to further develop the UK based supply chain by establishing an integrated, lean partnership between CGTC-MC and Symbiosis, a UK company that specialises in drug product manufacture (fill finish) for clinical sterile drug products and small volume, niche commercial sterile products.This collaboration, between Freeline, CGTC-MC and Symbiosis aims to establish an efficient, secure supply chain to reliably deliver viral vector gene therapy drug products to clinical trials. This will speed the pace at which new gene therapies can be made available to patients. The clinical supply chain delivered by this project will provide a foundation on which to deliver commercial supplies of gene therapy products in the future, thus establishing a secure UK-embedded commercial supply chain.The three UK collaborators will work together to create the new processes and analytical technologies to achieve rapid and seamless transfer of materials between the partners whilst maintaining quality. The project will include alignment of respective quality systems and processes as well as investment in internalising critical quality control tests and establishing new novel rapid methods. Internalising QC will bring about process efficiencies, adding to the security of the supply chain.The new integrated operating model will be demonstrated with the manufacture and supply of a GMP batch of a Freeline gene therapy drug product to a Phase I/II clinical trial for the treatment of a lysosomal storage disorder. The resulting supply chain and technologies will be translated into standard processes which can be offered by CGT-MC and Symbiosis to other future viral vector customers, thus significantly enhancing the UK viral vector manufacturing capability"