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.

该项目将着重于开发一种关键质量控制方法（QC）方法，该方法将使制造商能够测试并确保免疫治疗的质量（称为Biothymus）。该生物疗法旨在治疗婴儿中威胁生命的疾病。没有胸腺的孩子（称为完整的Digeorge综合征或CDG）的免疫系统功能较差，没有治疗可能会在3年内死亡。导致Biothymus的研究是在英国在伦敦大学的英国发明的，在伦敦大学和弗朗西斯·克里克研究所（Francis Crick Institute在胸腺组织制成的支架内（来自干细胞）的种群。这提供了胸腺结构的副本，该副本能够再生免疫系统的细胞。Videregen（项目负责人）将着重于开发符合英国和国际法规和标准的工业制造过程。 Biothymus的独特细胞方面是其安全性和功效的关键，但是由于治疗的独特性，没有现成的方法或试剂盒来测量和量化所使用的细胞。需要一种标准化，健壮和准确的QC测试方法，以确保最终产品质量，安全性和效率控制。Videregen将与国家测量实验室（NML）的细胞计量实验室合作。 NML具有必要的专业知识和设施来帮助解决细胞测量问题，通过了解如何将生物素中的多个细胞性能/属性（可以利用和简化为可重复的QC方法）中，即Videregen可以将其纳入其制造过程中。 效力。 QC定义生物素细胞方面的方法是治疗发展的关键活动/里程碑。