Syncona, an independent subsidiary of the Wellcome Trust, and UCL Business have announced the creation of biopharmaceutical company Freeline Therapeutics to develop and commercialize gene therapies for bleeding and other debilitating disorders.
Syncona will invest £25 million ($27.5 million) in a Series A financing.
The company’s next-generation AAV gene therapy platform has been developed by professor 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.
The results of the study, published in the New England Journal of Medicine, demonstrated that all 10 treated hemophilia B patients showed safe, sustained expression of blood clotting Factor IX from a single treatment.
Nathwani joins Freeline as founder and chief scientific officer and will split his time between the company and his roles at UCL, and director of the Katharine Dormandy Hemophilia Center and Thrombosis Unit of Royal Free London NHS Foundation Trust.
Dr. Christian Groendahl, partner with Syncona, joins Freeline as CEO.