Freeline is a clinical-stage, fully integrated, next generation, systemic AAV-based gene therapy company with the ambition of transforming the lives of patients suffering from inherited systemic debilitating diseases.
A life-changing breakthrough
Our ambitious vision is to create better lives for people suffering from inherited, systemic debilitating disease.
We aim to deliver one-time gene therapy treatments that provide functional cures through permanently sustained physiological protein levels, leveraging the high expression enabled by our proprietary gene therapy platform. Our initial focus is on developing treatments for monogenic diseases with high unmet need followed by expansion to address diseases requiring higher protein expression.
Our next generation gene therapy uses AAV technology to deliver safe and effective gene replacement to the liver to produce sustained therapeutic protein expression for diseases like haemophilia B and Fabry disease. Our treatment builds on the pioneering work of our founder, Amit Nathwani, Professor of Haematology at UCL. His award-winning scientific research was the first to show successful and sustained correction of bleeding symptoms in patients with severe haemophilia B.
We plan to use our novel proprietary capsid across our entire pipeline, leveraging faster and cost-effective pathways to clinic for our different indications. We are working hard on our vision to build and advance a pipeline of curative treatments to make a life-changing impact.