Ann Arbor, MI's RetroSense Therapeutics announced a $6 million Series B funding round to fund a Phase I/II study of its gene therapy candidate for vision restoration, as well as a "a second promising gene therapy candidate." The financing brings its total haul to $13 million, including a $7 million Series A round completed earlier this year.
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.
As has become the norm for biotechs going public, gene therapy player Dimension Therapeutics priced its IPO below the expected range, raising $71.5 million as the industry continues to struggle on Wall Street.
Shares of bluebird bio took a hit midday Monday after the closely watched biotech noted that one of the patients in a pioneering gene therapy study for beta-thalassemia required two blood transfusions after demonstrating symptoms of anemia following 7 years of being transfusion-free.
Using an AAV vector, a scientific team funded by the NIH has developed a gene therapy that preserved the vision of a group of dogs which share a malfunctioning retinitis pigmentosa GTPase regulator gene. And they believe the same type of treatment will also work in the human eye.
Voyager Therapeutics evidently isn't all that alarmed about widespread fears that the IPO window for biotech may be grinding shut. The biotech became the ninth Fierce 15 company from the class of 2014 to file for an IPO, looking to the markets to deliver $86 million or so to continue funding for its early-stage gene therapy work.
A mouse model has been established for Leber hereditary optic neuropathy, a vision disorder caused by mutations within genes in the "battery packs" of our cells--the mitochondria. And investigators at the NIH say they were able to develop a gene therapy that could be used to treat it.
After repeatedly batting back doubts about its lead gene therapy, Spark Therapeutics says its pivotal study for SPK-RPE65, an FDA-designated "breakthrough" for sight-blighting inherited retinal dystrophies, came through with positive data. The biotech says that the treatment hit the primary and two of three secondary endpoints in the late-stage study, setting up a biologics license application at the FDA next year.
UniQure is pressing forward with its gene therapy for a rare and deadly disease after charting a positive signal in a small yearlong trial, news that sent the Dutch company's share price soaring.
The biotech IPO window is still wide open and ready for new business, especially if it relates to a hot new tech like gene therapy. RegenX Bio proved that point today, raising $138 million from the sale of 6.3 million shares at $22 a pop.