- Theresa Heggie
- Prof. Amit Nathwani
- % Shareholding
- Number of employees
- Clinical programmes
Unless stated all data at 31 December 2020
Freeline is a clinical-stage biotechnology company developing transformative gene therapies for patients suffering from inherited systemic debilitating diseases.
Targeting the liver, Freeline has the potential to treat a wide range of chronic diseases. It aims to leverage the high expression enabled by its proprietary gene therapy platform, initially focusing on developing treatments for monogenic diseases with high unmet need as well as addressing diseases requiring higher protein expression which are more challenging to tackle with conventional gene therapy platforms.
Freeline’s next generation gene therapy uses Adeno-Associated Virus (AAV) technology that aims to deliver best in class safe and effective gene replacement to the liver to produce sustained therapeutic protein expression for diseases like Haemophilia B and Fabry disease.
The Company’s treatment builds on the pioneering work of founder and CSO, 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.
Beyond the leading target indications, Freeline plan to also use this novel proprietary capsid across their entire pipeline, which we believe reduces the risks of each subsequent pipeline programme. The company’s vision is to build and advance a pipeline of curative treatments that have the potential to restore protein production to normal levels in order to make a life-changing impact.
- To deliver therapies for a broad pipeline of systemic diseases which require the delivery of high protein expression levels, with the aim of curing and transforming patients’ lives.
Unmet medical need
- Significant number of systemic diseases with genetic drivers which have poor or no treatment options
- Current standard of care in clinical programmes of Haemophilia B and Fabry disease is Enzyme Replacement Therapy (ERT); requires regular administration, protein activity does not remain stable
- 9,000 patient opportunity in lead programme in Haemophilia B
- 9,000 patient opportunity in Fabry’s disease
- 6,000 patient opportunity in Gaucher’s
- 38,000 patient opportunity in Haemophilia A
- Highly competitive environment
- Differentiated product required
- Complex manufacturing
*Freeline corporate presentation
The Freeline Therapeutics pipeline
B-AMAZE - Haemophilia B