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

该项目的重点是开发一种关键的质量控制（QC）方法，使制造商能够测试和确保免疫疗法（称为BioThrombus）的质量。BioThrombus疗法旨在治疗婴儿的危及生命的疾病。出生时没有胸腺的儿童（称为完全性迪乔治综合征或cDGS）的免疫系统功能低下，如果不进行治疗，可能在3年内死亡。导致生物免疫疗法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，目前，它正由Videregen开发，用于治疗患有cDGS的儿童，未来它可能用于治疗自身免疫性疾病。将混合细胞群（来自干细胞）结合在由胸腺组织制成的支架内。这提供了一个胸腺结构的副本，它能够再生免疫系统的细胞。Videeregen（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生产工艺。BioThorax独特的细胞方面是其安全性和有效性的关键，但由于治疗的独特性，没有现成的方法或试剂盒来测量和量化所使用的细胞。为了确保最终产品的质量、安全性和有效性控制，需要一种标准化、稳健和准确的QC测试方法。Videeregen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来帮助解决小区测量问题，通过了解如何利用BioThrombin中的多种细胞特性/属性，并将其简化为可重现的QC方法，从而使Videregen可以将其纳入其生产过程。如果没有可靠的QC方法来量化和识别产品中的细胞，则无法实现用于临床前研究和过渡到临床试验的BioThrombin生产，这与产品的有效性有关。用于确定BioThorax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措施和最终放行检测。*该方法将允许进一步研究以了解胸腺细胞表型与其功能之间的关系，以在体内培养T细胞，从而允许制造优化和治疗有效性。该方法将为监管机构、临床医生和投资者提供高度的信心，以采用或投资该技术。

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.