NightstaRx Ltd

Nightstar is a private biopharmaceutical company, funded by Syncona LLP, an independent subsidiary of the Wellcome Trust, focused on the development of therapies for retinal dystrophies. The Company’s lead programme is a retinal gene therapy for choroideremia, a rare inherited cause of blindness that affects around 1 in 50,000 people. There are a number of other retinal degenerative disorders, some due to a genetic mutation, with others, such as age-related macular degeneration (AMD), with age being the major risk factor. Gene therapy treatment is extraordinarily complex involving both scientific expertise alongside ground-breaking surgical techniques and has the real potential to deliver a one-time cure for these conditions.