UCL researchers have received a boost of over £88 million to continue their ground-breaking research into gene therapies for disorders like haemophilia.
£85 million of the funding for UCL spin-out company Freeline Therapeutics comes from healthcare technology investment company Syncona. A further £3.4 million in funding comes from The University College London Technology Fund.
The funds will enable researchers to drive forwards work on next-generation gene therapies for haemophilia B, a blood clotting disorder, and Fabry Disease, a rare genetic disease caused by an enzyme deficiency.
Freeline was set up by Syncona and UCL Business in 2015, to build on successful research into novel gene therapies to treat haemophilia by BRC-supported Professor Amit Nathwani, Professor of Haematology at UCL.
People with haemophilia B are unable to produce the protein factor IX, which is critical for blood clotting. Normally, patients are treated with injections which contain factor IX, but patients sometimes have to receive injections multiple times per week, which is costly and inconvenient.
Professor Nathwani, who is supported by the BRC, developed a gene therapy in which a modified virus ‘infects’ the body with the genetic blueprint needed to produce factor IX. Professor Nathwani found that, after just one round of this treatment, levels of factor IX remained at safe, sustained levels in all patients.
Professor Nathwani’s work has been described by doctors as potentially life-changing for people with haemophilia B.
Professor Nathwani’s research, published in the New England Journal of Medicine, can be read via the links below.
Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B (November 2014)
Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B (December 2011)
