The UCL Technology Fund (UCLTF) has approved an investment of up to £1 million to support researchers Professor Adrian Thrasher, Professor Bobby Gaspar, and Dr GiorgiaSantilli in developing a gene therapy approach for treating p47phox-deficient chronic granulomatous disease (p47-CGD).
p47-CGD is a rare genetic disorder in which the immune system is compromised. Patients with p47-CGD suffer from serious recurrent infections, as well as widespread tissue inflammation.
The gene therapy approach, which was developed by Professor Thrasher and colleagues funded by the NIHR Great Ormond Street Biomedical Research Centre, could potentially restore the immune system in children with the condition. The funding provided by the UCLTF will support completion of final pre-clinical steps and take the therapy to a first-in-child, proof-of-concept clinical trial. Results from pre-clinical studies and from clinical trials in the US and Europe appear promising.