NightstaRx develops one-time gene therapies for rare inherited retinal diseases, focusing on preventing progression to blindness. Its lead program, NSR-REP1 for choroideremia, produced positive Phase 1/2 results published in The Lancet (2014) and NEJM (2016) and is advancing toward a pivotal Phase 3 registrational trial. A second program, NSR-RPGR, is in an ongoing Phase 1/2 trial for X-linked retinitis pigmentosa. The company is also planning a Phase 1/2 trial for a gene therapy targeting an inherited form of macular dystrophy and is evaluating additional retinal candidates for clinical entry. NightstaRx was founded in 2014 by ophthalmology researchers at the University of Oxford and operates from Boston and London. The recent financing provides capital to move multiple programs forward through clinical development. NightstaRx is developing gene therapies delivered via AAV vectors to treat inherited retinal diseases, initially focusing on choroideremia with its lead program AAV.REP1. The company reported Phase I results in which AAV.REP1 improved vision in six patients, data published in The Lancet. NightstaRx is advancing AAV.REP1 through a Phase II trial while licensing five additional retinal programs from Oxford’s tech transfer department to expand its pipeline. Management has bolstered the team with ophthalmology veterans—Aniz Girach as CMO and David Fellows as CEO—and plans to invest in the infrastructure needed to take multiple programs through clinical development. Financially, the company was seeded by the Wellcome Trust’s Syncona and previously raised about $25 million in Series A funding; it has now secured an additional $35 million in new financing. The platform is intended to have broad applications across genetic forms of blindness such as retinitis pigmentosa, according to founder Robert MacLaren.

Total raised

$80M

Funding rounds

2

Latest round

Series C

Latest activity

Jun 2017