Gene Therapy

Nightstar is a biopharmaceutical company with substantial funding provided by Syncona Partners, the healthcare investment company of the Wellcome Trust. The Company has been established to initially focus on developing and registering an AAV.REP1 retinal gene therapy for choroideremia in the US and Europe. Choroideremia is a rare inherited cause of blindness and the development program builds on the successful Phase I/II study published in the Lancet Medical Journal in January 2014.