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

  • Apr 17 2024
  • Length: 24 mins
  • Podcast

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

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

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  • Summary

  • 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

    Visit out website for the transcript: https://www.uclb.com/

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