Gene-modified cell therapy for children’s rare diseases

A team led by clinician scientists Professors Bobby Gaspar and Adrian Thrasher at Great Ormond Street Hospital NHS Trust and UCL have pioneered the use of gene therapy for rare diseases in children. With a focus on primary immune deficiencies (PIDs), the team has developed the technique of ex vivo gene therapy of patient haematopoietic stem cells in order to correct a disease’s underlying genetic defect.

The combination of UCL’s world-class academic environment, GOSH’s unique patient population and GOSH NIHR Biomedical Research Centre (BRC) support has enabled the team to progress the innovative technology through in vitro and in vivo proof-of-concept (POC) studies and into clinical trials. The team have now led seven early phase clinical trials for PID, restoring immune function to patients with minimal side effects in the majority of cases. Further therapies are in the clinical pipeline, and extending the technology to gene-modify other cell types such as T-cells and fibroblasts has widened the scope to treat diseases, including haematological disorders and skin disease.

Professors Gaspar, Thrasher and Professor Waseem Qasim founded spinout company Orchard Therapeutics in 2016 to advance and commercialise the group’s ex vivo gene therapy platform. Orchard was created by UCL Business PLC and F-Prime Capital Partners, securing £21 million in Series A financing. The company has partnerships with UCL, GOSH, University of Manchester, University of California Los Angeles, and Boston Children’s Hospital.