After posting some early clinical success with its first gene therapy project, University of Oxford spinout NightstaRx is looking to widen its pipeline of projects, recruiting venture giant New Enterprise Associates to pitch in on a $35 million funding round.
The company, seeded by the Wellcome Trust's Syncona subsidiary, is at work on a potential one-time treatment for the rare choroideremia, an inherited form of progressive blindness that stems from a genetic defect. NightstaRx made headlines early last year when its gene therapy, AAV.REP1, improved the vision of 6 patients with the incurable disease in a Phase I study published in The Lancet. That early success helped the company raise about $25 million in Series A funds.
Now, with its new cash, NightstaRx is licensing 5 more retinal programs from Oxford's tech transfer department while working through a Phase II trial on AAV.REP1. The plan is to build NightstaRx into a commercial-scale gene therapy company, management said, plotting to invest in the infrastructure necessary to get multiple projects through clinical development.
Since making a splash in choroideremia last year, NightstaRx has expanded its ranks, bringing in Aniz Girach, a former ophthalmology exec at Novartis ($NVS) and Merck ($MRK), to serve as chief medical officer and appointing David Fellows, a veteran of Johnson & Johnson ($JNJ) and Allergan ($AGN), as CEO. Fellows serves on the company's board alongside Syncona's Chris Hollowood, and the two will now be joined by NEA partner and biotech VC fixture David Mott.
The company grew out of the work of Oxford's Robert MacLaren, an award-winning ophthalmology professor who has done innovative work in retinal reconstruction, cell therapy and other approaches to improving vision. NightstaRx's technology is designed to deliver corrective genes by hitching them onto a genetically modified virus that carries its payload into the retina, and the platform has wide applications beyond choroideremia, MacLaren said.
"Gene therapy has huge potential as a treatment for many patients who are suffering from retinitis pigmentosa and other genetic forms of blindness," MacLaren said in a statement. "We have established an internationally renowned team around the choroideremia program and I am delighted that this additional funding will allow us to develop our other retinal gene therapy projects into real treatments for patients."
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