Syncona and UCLB launch Freeline Therapeutics

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.

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."

Links

• Freeline Therapeutics
• Professor Amit Nathwani's academic profile
• UCL Business
• Syncona LLP
• National Institute for Health Research University College London Hospitals Biomedical Research Centre

Image

• Freeline Therapeutics logo