Freeline is a privately-held clinical-stage biotechnology company based in the UK and in Germany. Our vision is to create better lives for people suffering from chronic systemic diseases using the potential of gene therapy as a one-off curative treatment. Freeline has a clear focus on AAV-based gene therapy targeting the liver with the aim to provide treatments for diseases with significant unmet needs.
Our gene therapy treatment builds upon the pioneering work by the Freeline CSO, Professor Amit Nathwani, Professor of Haematology at UCL, which has already transformed the lives of patients by providing safe, effective and reliable gene therapy for people with Haemophilia B.
Our proprietary AAV capsid is optimised to effectively transduce human liver cells and thus provide sustainable, high levels of the desired protein. This strong capsid performance is being validated in the clinic for haemophilia B patients and sets the stage for potentially curative products in a wide range of diseases which have not, to date, been treatable with a one-time treatment.