London, 10 December 2015 - Syncona LLP and UCL Business PLC (UCLB), the wholly-owned technology transfer company of UCL (University College London), 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 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. Chris Hollowood, Partner with Syncona LLP and Chairman of Freeline Therapeutics, said: "It is exciting to be building a company on this ground-breaking technology that has the potential to transform the lives of patients with a single administration. It is another example of where Syncona is partnering with world leaders in their respective fields to provide them with a platform to deliver the greatest possible impact with their clinical research. Professor Nathwani is a thought leader in the development of novel treatments using gene transfer technology and we look forward to working with him to build Freeline into a sustainable biopharmaceutical company over the long-term." Cengiz Tarhan, Managing Director of UCLB, commented: "UCL is a world leader in the biomedical sciences, with a commitment to outstanding research and translation into healthcare benefits for patients. We have considerable interests in advanced therapies and I am pleased that Professor Nathwani's work is being taken forward in a commercial environment in a way that may benefit patients globally. The formation of Freeline represents the culmination of several years of research in the laboratories of Professor Nathwani and his collaborators, drawing on funding from multiple funding sources. UCLB are delighted to be able to partner with Syncona to launch the Company." Amit Nathwani, Founder and CSO of Freeline Therapeutics, said: "Our initial study has shown the potential of AAV gene therapy. Building on the success of that study, we partnered with Syncona to form Freeline to provide an opportunity to realise our ambition of bringing multiple therapeutics to the patients that need them. Freeline will bring industry leading development people and our expertise together to ensure our clinical translation is rapidly converted to registered therapeutics." Professor Bryan Williams, director of the National Institute for Health Research University College London Hospitals Biomedical Research Centre which supports Professor Nathwani's work, added: "This is a wonderful example of how ground-breaking research from our University can be used to develop life changing treatments for our patients. Our NIHR BRC is committed to supporting and speeding up these kinds of transformative scientific breakthroughs." – Ends – Further information: Freeline Therapeutics Chris Hollowood Christian Groendahl +44 (0)20 7611 2031 JW Communications Julia Wilson +44 (0) 781 8430877 UCL/UCLB – Margaret-Anne Orgill Media Relations Manager – UCL Enterprise +44 (0)7572 602345 [email protected] Notes for Editors: About Freeline Therapeutics Freeline Therapeutics is a biopharmaceutical company based at the UCL Royal Free London Hospital focused on the development of gene therapies for bleeding and other disorders. 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 human adapted AAV vector platform is being utilised to advance therapies through the clinic and its shareholders include Syncona LLP and UCL. For further information please visit the Company's website at: www.freelinetx.com About Syncona Syncona LLP was founded in 2012 and operates as an evergreen investment company, taking an active role in identifying, developing and funding technologies with the potential to significantly impact the healthcare market of the future. Syncona can take the long view when necessary, able to concentrate investment into opportunities as technology is validated. Syncona is an independent subsidiary of the Wellcome Trust that invested the initial £200m capitalisation. For further information please visit the Company's website at: www.synconapartners.com About UCL Business PLC UCL Business PLC (UCLB) is a leading technology transfer company that supports and commercialises research and innovations arising from UCL, one of the UK's top research-led universities. UCLB has a successful track record and a strong reputation for identifying and protecting promising new technologies and innovations from UCL academics. UCLB has a strong track record in commercialising medical technologies and provides technology transfer services to UCL's associated hospitals; University College London Hospitals, Moorfields Eye Hospital, Great Ormond Street Hospital for Children and the Royal Free London Hospital. It invests directly in development projects to maximise the potential of the research and manages the commercialisation process of technologies from laboratory to market. For further information, please visit: www.uclb.com About UCL Founded in 1826, UCL was the first English university established after Oxford and Cambridge, the first to admit students regardless of race, class, religion or gender, and the first to provide systematic teaching of law, architecture and medicine. We are among the world's top universities, as reflected by performance in a range of international rankings and tables. UCL currently has almost 29,000 students from 150 countries and in the region of 10,000 employees. For further information, please visit: www.ucl.ac.uk About The National Institute for Health Research (NIHR) University College London Hospitals Biomedical Research Centre This is a partnership between University College London Hospitals NHS Foundation Trust and UCL, and is funded by the NIHR. The centre, set up in 2007, is at the forefront of research into major causes of illness and disease-related death and has already invested over £100m in research projects and infrastructure. Awarded a further £100m in government funding from the NIHR in 2011, the centre helps take innovations in basic science and turn them into therapies that directly benefit patients. In particular the centre supports experimental medicine research which tends to be 'first in man' studies such as research into new therapies and devices or the mechanisms of disease.