NightstaRx is a clinical-stage company developing a potentially sight restoring therapy for a rare degenerative disease causing blindness known as Choroideremia.
Beyond Choroideremia we will utilise our gene therapy expertise to bring to market therapies that treat sight threatening retinal diseases.
Choroideremia is a form of inherited blindness that affects 1 in 50,000 people. This disease is caused by a faulty gene which results in degeneration of the light-sensing retinal cells at the back of the eye. Nightstar is developing a way of delivering a working copy of the faulty gene to potentially prevent further loss of sight and to restore the vision of our patients.