Corporate Profile

At Nightstar, our mission is to maintain and restore sight in patients with inherited retinal diseases.


We are a clinical-stage company focused on developing and commercializing a pipeline of novel and potentially curative, one-time retinal gene therapies for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness, and, for which, there are no currently approved treatments.

Our lead retinal gene therapy product candidate, NSR-REP1, is being developed for the treatment of choroideremia (CHM), a rare, degenerative, X-linked genetic retinal disorder primarily affecting males that is caused by a mutation in the CHM gene.

We are currently conducting a prospective, natural history study, known as the NIGHT study, across multiple clinical sites in the United States, Europe and Canada. We plan to initiate a Phase 3 registrational clinical study, the STAR study, of NSR-REP1 for CHM in the first half of 2018. NSR-REP1 has been granted orphan drug designation for the treatment of CHM from both the U.S. Food and Drug Administration and the European Medicines Agency.

We are developing NSR-RPGR for the treatment of X-linked retinitis pigmentosa (XLRP), an inherited X-linked recessive retinal disease characterized by mutations in the RPGR gene, leading to a lack of protein transport and a loss of photoreceptors, the specialized cells in the eye that convert light into visual signals. NSR-RPGR is currently being evaluated in a dose-ranging Phase 1/2 clinical trial for the treatment of XLRP. We plan to initiate a natural history trial across multiple clinical sites by the end of 2018.

Our NSR-BEST1 gene therapy product candidate is being developed for the treatment of Best vitelliform macular dystrophy and is currently in preclinical development.

We have licensed three additional retinal gene therapy preclinical programs and are evaluating other in-licensing opportunities to broaden our pipeline and drive future growth.