UK investment group Syncona and UCL Business (UCLB), the wholly-owned technology transfer company of University College London, have created the biopharmaceutical company, Freeline Therapeutics, to develop and commercialize gene therapies for bleeding and other debilitating disorders.
London, UK-based Syncona will invest £25 million in a Series A financing. Gene therapy has the potential to provide long-term benefit to patients from a single treatment by carrying a therapeutic gene to a target cell in the body.
The company’s next-generation AAV gene therapy platform has been developed by Amit Nathwani, Professor of Hematology at UCL, and it builds on the successful hemophilia B Phase I/II trial conducted by him with St Jude Children’s Research Hospital, Memphis, USA. The results of the study, published in the New England Journal of Medicine, demonstrated that all 10 treated haemophilia B patients showed safe, sustained expression of blood clotting Factor IX from a single treatment.
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