Clinical translation of bioengineered thymus for complete DiGeorge syndrome

生物工程胸腺治疗完全迪乔治综合征的临床转化

• 批准号: 10005465
• 金额: $ 49.13万
• 依托单位: VIDEREGEN LIMITED
• 依托单位国家: 英国
• 项目类别: Feasibility Studies
• 财政年份: 2021
• 资助国家: 英国
• 起止时间: 2021 至 无数据
• 项目状态: 已结题
• 来源: https://gtr.ukri.org/projects?ref=10005465
• 关键词: Clinical; translation; bioengineered; thymus; complete

This project will focus on the translation of world-leading UK research into robust manufacturing processes ready for a future clinical trial to treat a life-threatening disease in infants. Babies born without a thymus gland (called complete DiGeorge Syndrome) have a fatally deficient immune system, and without treatment will die within 2-3 years.The project uses ground-breaking technology to create a new thymus using biological tissue scaffolds and thymus cells. Laboratory research has shown that this bioengineered thymus can provide the immune system functions that are lacking in infants born without a functioning thymus. During the project research work will generate data to support the manufacture, quality, safety and efficacy of a bioengineered thymus, which will be reviewed by the UK regulatory body, MHRA (Medicines and Healthcare products Regulatory Agency). The MHRA is responsible for ensuring all new and existing medicines and therapies are safe. Outcomes from reviews with the MHRA will be used by the project team to ensure the correct data is generated to support future application for a clinical trial to treat infants born without a thymus.

该项目将专注于将世界领先的英国研究转化为强大的制造工艺，为未来治疗婴儿致命疾病的临床试验做好准备。出生时没有胸腺的婴儿（称为完全DiGeorge综合征）具有致命的免疫系统缺陷，如果不进行治疗，将在2-3年内死亡。该项目采用突破性技术，使用生物组织支架和胸腺细胞创造新的胸腺。实验室研究表明，这种生物工程胸腺可以提供出生时没有功能性胸腺的婴儿所缺乏的免疫系统功能。在项目期间，研究工作将产生数据，以支持生物工程胸腺的生产，质量，安全性和有效性，这些数据将由英国监管机构MHRA（药品和保健产品监管机构）进行审查。MHRA负责确保所有新的和现有的药物和疗法是安全的。项目团队将使用MHRA审查的结果，以确保生成正确的数据，以支持未来申请治疗出生时没有胸腺的婴儿的临床试验。