Nightstar is a leading clinical-stage gene therapy company focused on novel one-time treatments for patients suffering from rare inherited retinal diseases. Nightstar’s lead product candidate, NSR-REP1, a AAV2 vector designed to produce REP1 inside the eye, 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 Phase 1/2 trials of NSR-REP1 were published in The Lancet in 2014 and in NEJM in 2016. In data from 32 patients treated with NSR-REP1 across four open-label Phase 1/2 clinical trials, over 90% of treated patients maintained or improved their visual acuity over a two-year follow-up period. Nightstar’s second product candidate, NSR-RPGR, is currently being evaluated in a clinical trial known as the XIRIUS trial for the treatment of patients with X-linked retinitis pigmentosa.

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