Michael Parini
Prof. Amit Nathwani
% Shareholding
Number of employees
(June 2020)
Clinical trials

Unless stated all financials at 30 June 2021

Freeline website

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.

Investment opportunity

  • 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

Market opportunity*

  • 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

Key risks

  • Highly competitive environment
  • Differentiated product required
  • Complex manufacturing 

*Freeline corporate presentation

14.05.18Syncona 0040 Freeline
14.05.18Syncona 0116 Freeline

The Freeline Therapeutics pipeline

Research Pre-clinical Clinical
  • B-AMAZE - Haemophilia B

  • Fabry disease

  • Gaucher

  • Haemophilia A

Syncona team

Chris Hollowood

Chris Hollowood

Chris is the Chief Investment Officer of Syncona Investment Management Ltd. He has been instrumental in the foundation and development of Syncona's gene therapy strategy, where it has founded companies focused on the eye, the liver, the kidney, and central nervous system. He is Chairman of Freeline, Gyroscope, SwanBio and Purespring and was also previously Chairman of Nightstar, a retinal gene the…
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Michael Kyriakides

Michael Kyriakides

Michael Kyriakides is a Partner of Syncona Investment Management Ltd. He works closely with Syncona portfolio companies Freeline Therapeutics and Gyroscope Therapeutics Previously, Michael was a member of the Life Sciences team at L.E.K. Consulting, where he worked in projects for pharmaceutical, biotech and private equity clients. Prior to joining L.E.K. Consulting, he was a clinical project and…
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Dominic Schmidt

Dominic Schmidt

Dominic is a Partner of Syncona Investment Management Ltd. He is a a Director on the board of Anaveon, and Purespring Therapeutics. Prior to joining Syncona, Dominic worked in the life science practice of strategy consultancy L.E.K Consulting. He received his PhD from the Department of Oncology at the University of Cambridge where he was a Cancer Research UK scholar. He also holds a German Diplom…
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Freeline Therapeutics leaders and founders

Professor Amit Nathwani

Professor Amit Nathwani

Founder, Clinical and Scientific Adviser and Director

Freeline Therapeutics publications & presentations

Show presentations from

Freeline corporate presentation - June 2021

Download PDF (1.80Mb)

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