Nightstar Therapeutics (formerly known as NightstaRx) is a clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases. The Company’s lead product candidate includes NSR-REP1 that has completed Phase 1/2 clinical trial for the treatment of patients with choroideremia. It is also developing NSR-RPGR that is in Phase 1/2 clinical trial for the treatment of patients with X-linked retinitis pigmentosa, an inherited X-linked recessive retinal disease; and NSR-BEST1, which is in preclinical development for the treatment of Best vitelliform macular dystrophy.