Roctavian: the journey to developing a one-shot gene therapy cure for haemophilia

Professor Nathwani will sit down with UCLB’s Director of Biopharm, Rick Fagan, to discuss how his work for the NHS looking after patients with haemophilia instilled a mission to develop a ‘one and done’ vaccine type approach to the condition.

He worked with UCLB to patent his technology, Roctavian, a one-time gene therapy used for the treatment of adults with severe haemophilia A, which was licensed to Biomarin Pharmaceutical Inc. Roctavian received marketing approval in Europe in 2022 and US FDA approval in 2023 and is now available to patients commercially. More about this life transforming gene therapy here.

A light lunch and refreshments will be provided.

Professor Amit Nathwani’s biography

Professor Nathwani is a renowned serial innovator and entrepreneur. He has an international reputation for his work in cancer research, immunology, and gene therapy and has been the recipient of a number of awards

Over the last 20 years Professor Nathwani has coordinated numerous Phase II and III trials in the treatment of refractory chronic lymphocytic leukaemia and is an author of more than 200 publications. He is the Principal Investigator on several early phase studies and an author of several patents. He continues to serve as the Director of the Katharine Dormandy Hemophilia Centre at the Royal Free Hospital and as a Senior NIHR Investigator.

Additionally, he has also been a Professor of Hematology at University College London since November 2011. Professor Nathwani graduated with MBChB from the University of Aberdeen in 1984 and is a fellow of the Royal College of Physician as well as the Royal College of Pathologist since June 1995. He holds a PhD in Molecular Biology.

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This talk is brought to you by UCLB BioPharm ‘Lab to Market’ series, a networking event for academics to informally discuss topics relevant to research commercialisation with UCLB .

The rationale for launching these sessions is to encourage academics, new to the idea of research commercialisation to come and ask us questions and hopefully be inspired to look for opportunities for market impact from their own research portfolios.

These sessions are designed for academics and research staff/fellows.

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