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Professor Amit Nathwani, UCL Cancer Institute, has seen gene therapy for haemophilia become a reality.

In a UCL TRO supported project, Professor Amit Nathwani and colleagues successfully treated adults with haemophilia B, caused by mutations affecting the blood clotting protein factor IX. For some trial participants, the therapy has been life-changing. Even low-level expression – 2 per cent of normal – can do away with the need for regular injections of protein IX, the regular treatment.

Professor Nathwani’s work has significant economic importance. It currently costs £140,000 a year to provide a haemophilia B patient with the protein they are missing through regular injections. If this can be replaced with a single dose of gene therapy, in addition to the huge commercial opportunity (a global market worth £8bn), the new technology would also be economically beneficial to the health service. To date, Professor Nathwani estimates that the haemophilia B trial alone has saved around £2m in health service costs.

This work has since led to the spin out of Freeline - a company focussing on the development of innovatice gene therapies to create better lives for patients with chronic diseases. 

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