A new company, set up by a doctor at the Royal Free Hospital, aims to combine academic creativity with private sector investment and experience to bring new, more effective treatments to patients sooner.
Freeline Therapeutics, which will be based at the Royal Free Hospital, is focused on developing gene therapies for haemophilia and other single-gene disorders, such as Gaucher’s disease.
The company has been set up by Professor Amit Nathwani, a consultant haemotologist at the Royal Free London and professor of haematology at UCL. It will build on the successful haemophilia B trial conducted by Professor Nathwani at the Katharine Dormandy Haemophilia and Thrombosis Centre at the Royal Free Hospital.
People with haemophilia A and haemophilia B are missing a gene which means they cannot produce a protein vital in the blood-clotting process. The current treatment involves two or three weekly injections of the missing protein. Professor Nathwani has developed a one-off gene therapy treatment for haemophilia B which involves delivering the missing gene via an infusion. He is now hoping to develop a similar treatment for patients with haemophilia A, who are missing a different gene from those with haemophilia B.
Professor Nathwani, said: “This company is based around our invention and supported by highly talented people from industry with experience and know-how of how to develop drugs for the world-wide market. Its location at the Royal Free Hospital will mean close collaboration between researchers and clinical staff and their work will be supported by a team from industry with special skills in drug development.
“With a £25 million investment from Syncona, an investment arm of the Wellcome Trust, and support from UCL Business, we will have an opportunity to build a world class company that will accelerate research and the development of new drugs. Additionally, the creation of new jobs and a rich seam of specialist skills at the Royal Free Hospital will cement the trust’s reputation for creating cutting edge treatments for patients.”
Image: Professor Amit Nathwani
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About the Royal Free London NHS Foundation Trust
The Royal Free began as a pioneering organisation and continues to play a leading role in the care of patients. Our mission is to provide world class expertise and local care. In the 21st century, the Royal Free London continues to lead improvements in healthcare.
The Royal Free London attracts patients from across the country and beyond to its specialist services in liver and kidney transplantation, haemophilia, renal care, HIV, infectious diseases, plastic surgery, immunology, Parkinson's disease, vascular surgery, cardiology, amyloidosis and scleroderma and we are a member of the academic health science partnership UCLPartners.