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Nightstar is developing and testing new therapies in patients to treat serious retinal diseases.

The Company applies 100% of its resources and industry-leading expertise in gene therapy, ophthalmology R&D, and surgical delivery to developing novel treatments for the eye.

The Company’s lead therapeutic candidate – NSR AAV-REP1 – is an AAV gene therapy advancing toward late-stage, pivotal clinical testing in patients with choroideremia. This disease is a rare inherited cause of blindness, affecting around 1 in 50,000 people. Nightstar’s treatment has the potential to deliver a one-time cure for patients with this disease.

All of the Company’s efforts focus on retinal dystrophies with currently few or no effective approved therapies. In the next 2-3 years, Nightstar will be progressing earlier-stage research programs to clinical testing for these rare retinal diseases.