Steve is a Director of the Gene Medicine Research Group at Oxford University and his research is focused on the application of gene therapy technologies to provide therapeutic interventions for human disease. During his post-doctoral research at Oxford he was the first to demonstrate that gene transfer could correct the Cystic Fibrosis defect in transgenic mice. This led to an extensive pre-clinical and clinical programme of gene therapy. Products developed in the Gene Medicine Research Group have been evaluated in four gene therapy clinical trials. The most recent was a phase IIb clinical study involving 136 participants that showed a clinically relevant improvement in lung function in those receiving gene therapy over those receiving a placebo treatment. Steve is one of the founding principle investigators of the UK Cystic Fibrosis Gene Therapy Consortium, a rolling research programme to realise a gene therapy for cystic fibrosis. The Consortium has been at the forefront of respiratory gene therapy for over 20 years.
Steves current research is focused on the development of CpG-free non-viral gene transfer formulations; adeno-associated virus and lentiviral gene transfer vectors pseudotyped for efficient lung gene transfer and precise genome engineering to enhance vector production and performance. His particular expertise is in the design and assessment of viral and non-viral gene therapy vectors, transgene expression cassette design, CRISPR/Cas9 genome engineering, characterisation of aerosol properties for efficient lung delivery, and GMP compliant viral and non-viral vector production. Steve also works with other organisations to improve the quality of gene therapy research.