Nightstar is a leading clinical-stage gene therapy company focused on developing and commercialising novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness

Nightstar’s lead product candidate, NSR-REP1, is currently in Phase 3 development for the treatment of patients with Choroideremia, a rare, degenerative, genetic retinal disorder that has no current treatments and affects approximately one in every 50,000 people.

Positive results from a Phase 1/2 trial of NSR-REP1 were published in The Lancet in 2014 and in The New England Journal of Medicine in 2016. Nightstar's second product candidate, NSR-RPGR, is currently being evaluated in a Phase 1/2 clinical trial for the treatment of patients with X-linked Retinitis Pigmentosa, an inherited X-linked recessive retinal disease that affects approximately one in every 40,000 people.


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.”