Gene Therapy, from Hollywood to the bench to the clinic

Dr Simon Waddington will present a seminar on 11 November 2025 from 15h00 - 16h30.

Inherited genetic diseases are individually rare but, collectively, affect over 400 million people worldwide; over 75% are children. For many young people with these conditions there is little hope. Most rare genetic diseases are untreatable, causing significant disability, with almost a third dying before their fifth birthday. Licensed genetic treatments are currently available for less than 0.1% of patients with these diseases, leading to significant morbidity and premature mortality. In this lecture, He will explore the public perception of gene therapy and detail one of our clinical-trajectory gene therapy programmes.

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