Freeline’s mission is to become a leading biopharmaceutical company founded on the successful development and commercialisation of liver directed gene therapies for bleeding disorders and other severe diseases.
These therapies are based on our next-generation proprietary AAV vector platform, with our lead programme being a gene therapy to treat haemophilia B. This gene therapy treatment, pioneered by Professor Amit Nathwani, Professor of Haematology at UCL and CSO of Freeline Therapeutics, has transformed the lives of patients by providing a safe, reliable source of the blood clotting protein Factor IX (Ref to NEJM papers 2011 and 2014; links are mentioned below). Clinical relevant blood levels of Factor IX have been obtained in ten out of ten treated patients and long-term sustained levels have been observed following a single gene therapy treatment.
The successes seen to date gives us a solid platform for developing a gene therapy approach for the treatment of Haemophilia B and other conditions.