Syncona, the venture arm of London-based charity Wellcome Trust, in January invested £12 ($20) million in an Oxford startup, the first program of which is a gene therapy for inherited blindness. NightstaRx—pronounced 'Nightstar'—is a spin-out from the University of Oxford and its research commercialization unit Isis Innovation. The company is pursuing a therapy for choroideremia (CHM), an inherited X-linked form of progressive blindness, caused by mutations to the gene encoding Rab-escort protein 1 (REP1). The first symptoms occur in childhood with reduced night vision as the retina degenerates. The gene therapy—an adeno-associated viral (AAV) vector encoding REP1 designed to deliver the correct version to the cells in the retina—was developed by Robert MacLaren at Oxford's Nuffield Laboratory of Ophthalmology. Vision improvements achieved by six patients were published in the Lancet (doi:10.1016/S0140-6736(13)62117-0; 16 January 2014), and a 12-patient phase 1 trial is underway. If approved, the price for this one-time treatment could fall between $83,000 and $110,000 per quality-adjusted life year, says Sander van Deventer, managing partner for Naarden, The Netherlands–based Forbion Capital Partners, the investors who backed the first FDA-approved gene therapy UniQure. The figures paid out for gene therapies are likely to match those for severe, untreated, Crohn's disease or rheumatoid arthritis, which are on a par with blindness for reduced quality of life, he says. Final payout will depend largely on whether the therapy brings full sight recovery or just restores light perception—and how long it lasts. Another gene therapy company Voyager Therapeutics of Cambridge, Massachusetts, raised $45 million in a series A round from investors Third Rock Ventures in February. Voyager's lead gene therapy program, an AAV serotype 2 vector encoding the dopa decarboxylase gene, is currently in phase 1 trials for Parkinson's disease.