Nightstar Therapeutics PLC, formerly Nightstar Therapeutics Limited, is a clinical-stage gene therapy company. The Company is focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness. The Company is developing a pipeline of proprietary product candidates that are designed to substantially modify or halt the progression of inherited retinal diseases. The Company's lead product candidate, NSR-REP1, for the treatment of choroideremia, or CHM, is entering Phase III clinical development. The Company is also conducting a Phase I/II clinical trial with its second product candidate, NSR-RPGR, for the treatment of X-linked retinitis pigmentosa, or XLRP. The Company's third product candidate, NSR-BEST1, is in preclinical development for the treatment of Best vitelliform macular dystrophy, or Best disease.
