Professor Amit Nathwani’s journey to create a one-shot cure for haemophilia A

UCL Minds

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. Host Nigel Campbell is joined by Prof Nathwani and Dr. Rick Fagan, UCLB Director of Biopharm, to explore how Amit’s work looking after patients with haemophilia in the NHS instilled a mission to develop a 'one and done' vaccine type approach to the condition. 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. Date of episode recording: 2024-04-10T00:00:00Z Duration: 00:23:58 Language of episode: English Presenter: Nigel Campbell Guests: Prof Amit Nathwani; Dr Rick Fagan Producer: Nora Amin