Analysis and quality control of novel mixed cell population for therapeutic development

用于治疗开发的新型混合细胞群的分析和质量控制

• 批准号: 10089851
• 金额: $ 2.1万
• 依托单位: VIDEREGEN LIMITED
• 依托单位国家: 英国
• 项目类别: Collaborative R&D
• 财政年份: 2024
• 资助国家: 英国
• 起止时间: 2024 至 无数据
• 项目状态: 未结题
• 来源: https://gtr.ukri.org/projects?ref=10089851
• 关键词: Analysis; quality; control; novel; mixed

This project will focus on developing a critical quality control (QC) method that will allow manufacturers to test and ensure the quality of an immune therapy (called BioThymus).The BioThymus therapy is designed to treat a life-threatening disease in infants. Children born without a thymus gland (called complete DiGeorge Syndrome or cDGS) have a poorly functioning immune system, and without treatment may die within 3 years.The research that lead to the BioThymus was invented in the UK at University College London and the Francis Crick Institute, and is now being developed by Videregen to treat children with cDGS and in the future it could be used to treat autoimmune diseases.The BioThymus combines mixed cell populations (from stem cells) within a scaffold made from thymus tissue. This provides a copy of a thymus gland structure, which is capable of regenerating the cells of the immune system.Videregen (project lead), will focus on developing the industrial manufacture process compliant with UK and International regulations and standards. The BioThymus's unique cellular aspects are key to its safety and efficacy, but because of the uniqueness of the therapy there are no off-the-shelf methods or kits to measure and quantify the cells used. A standardised, robust and accurate QC test method is required to ensure final product quality, safety and efficacy control.Videregen will collaborate in the project with the cell metrology lab of the National Measurement Laboratory (NML). NML have the necessary expertise and facilities to help address the cell measurement problem, by understanding how the multiple cell properties/attributes in the BioThymus can be harnessed and streamlined into a reproducible QC method that Videregen can incorporate into their manufacture process.BioThymus manufacture for preclinical studies and transition to clinical trials cannot be achieved without reliable QC methods that quantify and identify the cells in the product, which links to product effectiveness. QC methods to define BioThymus's cellular aspects is a critical activity/milestone for therapy development.* Method will be used as a QC measure and final release test.* Method will allow further research to understand relationships between thymic cell phenotype and their function to educate T-cells in-vivo, allowing manufacturing optimisation and therapy effectiveness.* Method will provide regulatory bodies, clinicians and investors a high degree of confidence to adopt or invest in the technology.

该项目将重点开发一种关键的质量控制 (QC) 方法，使制造商能够测试并确保免疫疗法（称为 BioThymus）的质量。BioThymus 疗法旨在治疗婴儿危及生命的疾病。出生时没有胸腺的儿童（称为完全性迪乔治综合症或 cDGS）的免疫系统功能较差，如果不进行治疗，可能会在 3 年内死亡。BioThymus 的研究是在英国伦敦大学学院和弗朗西斯·克里克研究所发明的。研究所，目前正在由 Videregen 开发，用于治疗患有 cDGS 的儿童，未来可用于治疗自身免疫性疾病。BioThymus 将混合细胞群（来自干细胞）结合在由胸腺组织制成的支架。这提供了胸腺结构的副本，能够再生免疫系统的细胞。Videregen（项目负责人）将专注于开发符合英国和国际法规和标准的工业制造工艺。 BioThymus 独特的细胞方面是其安全性和有效性的关键，但由于该疗法的独特性，没有现成的方法或试剂盒来测量和量化所使用的细胞。需要标准化、稳健且准确的 QC 测试方法来确保最终产品的质量、安全性和功效控制。Videregen 将与国家测量实验室 (NML) 的细胞计量实验室合作开展该项目。 NML 拥有必要的专业知识和设施，通过了解如何利用 BioThymus 中的多种细胞特性/属性并将其简化为可重复的 QC 方法，Videregen 可以将其纳入其制造过程，从而帮助解决细胞测量问题。BioThymus 临床前制造如果没有可靠的质量控制方法来量化和识别产品中的细胞（这与产品的有效性相关），就无法实现研究和向临床试验的过渡。定义 BioThymus 细胞方面的 QC 方法是治疗开发的关键活动/里程碑。*方法将用作 QC 测量和最终释放测试。*方法将允许进一​​步研究以了解胸腺细胞表型与其教育 T 功能之间的关系。 -体内细胞，允许制造优化和治疗效果。*方法将为监管机构、临床医生和投资者提供采用或投资该技术的高度信心。