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Lentiviral gene therapy for p47-CGD

DNA Sequence
Developing a novel gene therapy for the p47phox-deficient variant of chronic granulomatous disease (CGD)

Prof Adrian Thrasher and his team at the UCL Great Ormond Street Institute for Child Health have developed a novel gene therapy for the p47phox-deficient variant of chronic granulomatous disease (CGD). A significant unmet need exists for patients who inherit this disease, as there is no existing curative gene therapy, stem-cell transplantation is not indicated for most patients and, in the absence of a cure, expensive lifetime prophylaxis against infection is required – and even then, recurrent infections and extended hospitalisations are common. With direct funding from the UCL Technology Fund, Prof Thrasher and colleagues will be completing the final preclinical and manufacturing steps required to take this gene therapy into the clinic; as well as supporting a first-in-man clinical trial which itself has the potential to transform the lives of patients.