Episode 13: Professor Amit Nathwani’s journey to create a one-shot haemophilia A cure

To mark World Haemophilia Day, we sit down with Professor Amit Nathwani of the UCL Cancer Institute to explore his journey to commercialising the groundbreaking haemophilia A treatment, Roctavian.

Amit’s lab to market story, working with UCLB, led to the creation of Roctavian, a one-time gene therapy used for the treatment of adults with severe haemophilia A. The therapy, which was licensed to Biomarin Pharmaceutical Inc, received marketing approval in Europe in 2022 and US FDA approval in 2023 and is now available to patients commercially.

Host Nigel Campbell is joined by Amit and UCLB Director of Biopharm Rick Fagan to explore how Amit’s work for the NHS looking after patients with haemophilia instilled a mission to develop a 'one and done' vaccine type approach to the condition.