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Eye examination

Normal Retina

Eye Test Light Box

Retina showing rods & cones

Retinal eye surgery


October 2011
First patient to receive gene therapy for Choroideremia is treated by Prof Robert MacLaren at the University of Oxford in a Phase I/II study
January 2014
Initial meetings between University of Oxford, Prof. MacLaren and Syncona about the AAV2-REP1 gene therapy
January 2014
Preliminary results from the Phase I/II study published in the Lancet.
January 2014
Oxford University and Syncona found NightstaRx with a Series A subsequently expanded to $30m, Syncona partner Chris Hollowood appointed Chairman.
August 2014 to August 2015
Dave Fellows appointed as CEO and management team recruited including Chief Medical Officer and heads of manufacturing and regulatory.
June 2015 to November 2015
NightstaRx announces the start of AAV2-REP1 gene therapy studies in Miami, USA and Alberta, Canada
November 2015
NightstaRx close Series B round raising $35M, and licenses a further 5 retinal dystrophies programmes from the University of Oxford
September 2016
NightstaRx recruits a Chief Scientific Officer

Company Details


Gene therapy for inherited retinal dystrophies

Fully diluted ownership


Value of Syncona holding


% of nav



Nightstar is a biopharmaceutical company focused on the development and commercialisation of therapies for retinal dystrophies.

Nightstar’s most advanced program, NSR AAV2-REP1, is in clinical trials for the treatment of choroideremia, a rare disease which causes permanent loss of eyesight and for which there is no other treatment. The gene therapy is delivered by injection into the retina, providing a working copy of the disease-causing faulty gene locally in the eye. The disease modifying technology has the potential to maintain and restore sight in patients, from a single administration.

There are multiple inherited forms of blindness that are addressable with Nightstar’s gene therapy technology. Nightstar is building a pipeline of products based on its manufacturing, gene therapy and retinal surgery capability.


David Fellows

Syncona Representatives
  • Chris Hollowood
  • Iraj Ali
  • Robert MacLaren
“Working with Syncona has provided a partner capable of accelerating our research into becoming approved treatments for patients. I was impressed with Syncona’s plan and drive to build a company to develop multiple therapeutics for people suffering from debilitating retinal diseases.”

Robert MacLaren - Nightstar