Freeline Therapeutics

Michael Parini
Prof. Amit Nathwani
Markus Hörer
% Shareholding
Number of employees
Clinical trial

Unless stated all financials at 31 December 2023.

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 Gaucher.

The Company’s treatment builds on the pioneering work of founder and CSO, Amit Nathwani, Professor of Haematology at UCL. 

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 thesis

  • ­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

Market opportunity*

  • 18,000 patient opportunity in Gaucher disease

*Freeline corporate presentation

14.05.18Syncona 0040 Freeline
14.05.18Syncona 0116 Freeline

The Freeline Therapeutics pipeline

Best ideas Pre-clinical Clinical Late Clinical BLA
  • FLT201 - Gaucher disease type 1

Syncona team

Chris Hollowood

Chris Hollowood

Chris is the Chief Executive 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 Beacon, Freeline, Purespring and Forcefield and was also previously Chairman of retinal gene therapies Nightst…
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Michael Kyriakides

Michael Kyriakides

Michael Kyriakides is an Investment Partner of Syncona Investment Management Ltd. He works closely with Syncona portfolio companies Freeline Therapeutics, Purespring Therapeutics and Beacon Therapeutics. He previously also supported Nightstar Therapeutics, Gyroscope Therapeutics and Clade Therapeutics. Prior to joining Syncona, Michael was a member of the Life Sciences team at L.E.K. Consulting an…
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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

Syncona Investor Webinar: Freeline Therapeutics

Download PDF (1.46Mb)

Freeline Corporate Presentation - October 2023

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Freeline Corporate Presentation - August 2023

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Freeline Corporate Presentation - January 2023

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