- Sanofi will invest up to $60 million in Gyroscope Therapeutics, a five-year-old U.K.-based gene therapy startup that is advancing a treatment to prevent a common form of blindness. The funding comes just five weeks after the company reported positive early safety and biological data from the gene therapy, called GT005.
- According to the deal's terms, Sanofi will invest $40 million initially, with another $20 million tranche due when Gyroscope assembles another venture capital investment round. In March, the company completed a Series C venture round worth $148 million.
- The new cash infusion is smaller than the one Gyroscope sought in May, when it filed for and then postponed an initial public offering that would have raised at least $135 million. Executives blamed "market conditions" at a time when 18 biotechs successfully priced IPOs in two months.
Eye diseases are an attractive field for gene therapy developers because the eye can be targeted more directly and potential immune responses are limited compared to other organs. The first gene therapy approved in the U.S., Roche's Luxturna, treats a rare, genetic cause of blindness, but other developers like Adverum Biotechnologies, RegenXbio, Kriya Therapeutics and Nanoscope Technologies are working on treatments for more common eye conditions.
Novartis and Johnson & Johnson also have shown interest in this area. A year ago, the Swiss group bought Vedere Bio for $150 million and Johnson & Johnson licensed a gene therapy from Hemera that could treat the same condition as Gyroscope's lead project.
Rather than replace a defective gene, Gyroscope is using its therapy to stimulate proteins that block immune activity that contributes to a type of vision loss in people who have dry age-related macular degeneration, or AMD.
The most advanced clinical trial of GT005 is an all-comers trial in patients with the vision loss — called "geographic atrophy" — but a separate trial is recruiting patients with a rare mutation that drives the immune activity behind the vision loss.
That first trial has reported early safety and biomarker data. In 28 patients with up to two years of follow up, researchers didn't report any serious adverse events, although 16 patients experienced mild-to-moderate adverse events related to the eye injection.
Of 13 patients who've been followed for at least 29 weeks after the injection, 11 had elevated levels of a key protein that is though to block the immune activity, as well as other biological signs the immune activity had declined.
That trial is primarily designed to assess safety and determine the best dose for clinical use, but has secondary goals of measuring whether the gene therapy stops further vision loss. Two more advanced trials — one in patients with the rare mutation as well as a second broader study — will assess progression of vision loss as their primary goals.
Gyroscope was founded by U.K.-based healthcare investment group Syncona Ltd. Before the attempted IPO, Syncona held a 62.2% stake. With the Sanofi investment, Syncona's share drops to 49%, which it values at 154 million British pounds, or about $208 million.