Syncona and UCLB launch Freeline Therapeutics

Syncona LLP and UCL Business, the wholly-owned technology transfer company of UCL, announce the creation of the biopharmaceutical company, Freeline Therapeutics, to develop and commercialise gene therapies for bleeding and other debilitating disorders. 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. Syncona, which is an independent subsidiary of the Wellcome Trust, will invest £25 million in a Series A financing. The company's next-generation AAV gene therapy platform has been developed by Professor Amit Nathwani, Professor of Haematology at UCL, 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, sustained expression of blood clotting Factor IX from a single treatment. Professor Nathwani joins Freeline Therapeutics as Founder and Chief Scientific Officer and will split his time between the Company and his roles as Professor of Haematology at UCL and Director of the Katharine Dormandy Haemophilia Centre and Thrombosis Unit of Royal Free London NHS Foundation Trust. Dr Christian Groendahl, Partner with Syncona LLP, joins Freeline Therapeutics as CEO.