Novel gene therapy could reduce bleeding risk for haemophilia patients

For the paper, published in the New England Journal of Medicine, experts from UCL, Royal Free Hospital and biotechnology company Freeline Therapeutics trialled and continue to evaluate a new type of adeno-associated virus (AAV) gene therapy candidate, called FLT180a, to treat severe and moderately severe cases of the condition.

Professor Geraint Rees (UCL Vice-Provost Research, Innovation and Global Engagement, and Non-Executive Director of UCL Business), said: “The results of this important study are a perfect example of how UCL research and innovation can translate to real-world and life-changing impact.”

The Phase I/II trial was sponsored by UCL and funded by Freeline Therapeutics.

Freeline was supported by UCL Business (UCLB), UCL’s commercialisation company.

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