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news.UCL Business, the technology-transfer company of University College London, and Syncona have launched Freeline Therapeutics, a biopharmaceutical firm focused on the development and commercialization of gene therapies for bleeding and other debilitating disorders.
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Syncona, an independent subsidiary of the Wellcome Trust, will invest £25m in a Series A financing.
UCL professor of haematology Amit Nathwani developed Freeline's next-generation AAV gene therapy platform.
In the phase I/II study, all ten treated haemophilia B patients experienced safe, sustained expression of blood clotting Factor IX from a single treatment.
Nathwani is the founder and chief scientific officer of Freeline Therapeutics, which will be based at the UCL Royal Free London Hospital. Syncona partner Christian Groendahl will serve as CEO.
Nathwani 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."
UCLB managing director Cengiz Tarhan said: "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."
Gene therapy has the potential to transform lives for people with severe diseases by offering a long-lasting source of enzymes to the blood.