Spur Therapeutics

Michael Parini
% Shareholding
Number of employees
Clinical trials

Unless stated all financials at 31 March 2024.

Spur website

Spur Therapeutics (formerly Freeline Therapeutics) is a clinical-stage biotechnology company focused on developing life-changing gene therapies for debilitating chronic conditions.

By optimising every component of its product candidates, Spur aims to unlock the true potential of gene therapy to realise outsized clinical results. Spur is advancing a breakthrough gene therapy candidate for Gaucher disease and a potential first-in-class gene therapy candidate for adrenomyeloneuropathy (AMN), as well as a research strategy to move gene therapy into more prevalent diseases, including forms of Parkinson’s, dementia, and cardiovascular disease.

Spur’s lead programme, FLT201, is a highly differentiated gene therapy candidate that delivers a novel transgene to treat Gaucher disease Type 1, for which there is currently no cure. Spur’s next generation gene therapy uses AAV technology that aims to deliver best-in-class safe and effective gene replacement to the liver to produce sustained therapeutic protein expression. Spur has additional pre-clinical research programmes in central nervous system (CNS) and cardiovascular diseases, including one focused on GBA1 Parkinson’s disease that leverages the same novel transgene as FLT201.

In 2024 Spur acquired Syncona portfolio company SwanBio Therapeutics, creating a consolidated adeno-associated virus (AAV) gene therapy pipeline, bringing synergies around clinical capabilities and manufacturing know-how, as well as driving cost and operating efficiencies. Through acquiring SwanBio, Spur incorporated SBT101, a potentially first-in-class AAV gene therapy in Phase I/II clinical trials for the treatment of AMN, into its pipeline. AMN is a devastating neurodegenerative disease for which there are currently no approved treatments. The integration of the SBT101 programme enhances Spur’s growing focus on CNS disorders, including for its pre-clinical research programme in Parkinson’s.

Investment thesis

  • Spur has two potential first-in-class gene therapy programmes

  • Its lead programme is a highly differentiated gene therapy candidate, FLT201, for Gaucher disease type 1, and has shown positive safety and efficacy data to date

  • SBT101, Spur’s second gene therapy programme, is a potential first-to-market therapy in an area of high unmet medical need

Unmet medical need

  • There are a significant number of systemic diseases with genetic drivers and single gene neurological disorders which have poor or no treatment options

  • Gaucher disease type 1 and adrenomyeloneuropathy are both debilitating, chronic and progressive disorders

Market opportunity*

  • 18,000 patient opportunity in Gaucher disease
  • AMN impacts 8,000-10,000 male patients in the US and EU5

*Spur corporate presentation

14.05.18Syncona 0040 Freeline
14.05.18Syncona 0116 Freeline

The Spur Therapeutics pipeline

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

  • SBT101 – AMN

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 Spur and Purespring, and is a Board member of Beacon and Yellowstone. He was also previously Chairman of retin…
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John Tsai

John Tsai

John Tsai is an Executive Partner of Syncona Investment Management Limited He is currently the CEO of Forcefield Therapeutics and a Board member of Mosaic Therapeutics, Purespring Therapeutics and Spur Therapeutics. As an Executive Partner at Syncona, John works closely with Lead Partners and the wider investment team to support evaluation and execution of new and existing opportunities at Syncona…
View John's biography

Spur Therapeutics leaders and founders

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