Gene therapy has the potential to transform lives for people with severe diseases by providing a long-lasting, safe and reliable source of enzymes to the blood. The Company’s next-generation AAV gene therapy platform has been developed by Professor Amit Nathwani, Professor of Haematology at UCLB, and it builds on the successful haemophilia B phase I/II trial conducted by him with St. Jude Children’s Research Hospital, Memphis. The results of the study, published in the New England Journal of Medicine, demonstrated that all ten treated haemophilia B patients showed safe and sustained expression of blood clotting Factor IX after a single administration. The company will develop and commercialise gene therapies for bleeding and other debilitating disorders. Freeline’s shareholders include Syncona LLP, UCLB and UCL Technology Fund.
Freeline Therapeutics is a bio-pharmaceutical company focused on the development of liver-directed gene therapies