NEA-backed Nightstar Therapeutics sets sights on an $86M IPO to fund PhIII gene therapy study
Two months after banking a $45 million crossover round from some top biotech investors, Oxford gene therapy spinout Nightstar Therapeutics has penciled in an $86 million IPO to push its lead drug through Phase III testing.
Nightstar is a retinal gene therapy company— officially set up as a new subsidiary to NightStaRx — advancing new therapies aimed at diseases that cause blinding. First up is NSR-REP1, designed to treat choroideremia by correcting mutations in the CHM gene. According to the F-1, the biotech has data on 32 patients showing their gene therapy either maintained or improved visual acuity in 90% of the patients in the study. The Phase III program, set to start in early 2018, will recruit 140 patients and divide it into high- and low- and no-dose groups, while running a parallel natural history study to detail expected rates of decline.
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