Gene therapy for inherited retinal dystrophies
Fully diluted ownership
Value of Syncona holding
£174.6m (as at 29 September 2017)
Nightstar is a clinical-stage gene therapy company focused on developing and commercializing novel one-time treatments for patients suffering from rare inherited retinal diseases that would otherwise progress to blindness
Nightstar’s most advanced programme is in clinical trials for the treatment of choroideremia, a rare disease which causes permanent loss of eyesight for which there is no treatment. Nightstar’s treatment would deliver a gene therapy by injection into the retina, providing a working copy of the disease-causing faulty gene locally in the eye. The disease modifying technology has the potential to maintain and restore sight in patients, from a single administration.
There are multiple inherited forms of blindness that are addressable with Nightstar’s gene therapy technology. Nightstar is building a pipeline of products based on its manufacturing, gene therapy and retinal surgery capability. Nightstar is listed on NASDAQ under the ticker ‘NITE’.
- Chris Hollowood
- Iraj Ali
- Robert MacLaren