Clinical translation of bioengineered thymus for complete DiGeorge syndrome

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.