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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