A pair of early-stage biotech companies has joined the thinning queue of drug developers trying to go public amid a painful market correction, angling to raise more than $135 million combined as the industry sputters.
When GlaxoSmithKline veteran Amber Salzman started up Paris-based AAVLife and ramped up its first gene therapy program for Friedreich's ataxia, she started working early on with adenovirus vector pioneer Ron Crystal at Weill Cornell Medicine. And now, less than two years after rounding up the $12 million Series A for the biotech, Crystal is delivering three new gene therapy programs that the company--now renamed Annapurna--plans to put into the clinic as Salzman hunts up a Series B.
The Wellcome Trust's investment arm is putting up £25 million ($38 million) to launch a new company developing a potential one-time treatment for hemophilia and planning to build a pipeline of gene therapies.
Bluebird bio, at work on a promising gene therapy for a rare blood disease, has shifted its clinical strategy after learning that its treatment doesn't work the same way for all patients with the disorder, planning to split up the population in future studies.
UniQure has dropped plans to seek FDA approval for its €1.1 million ($1.2 million)-per-course gene therapy Glybera. The decision comes three months after FDA told uniQure it would need to see data from two clinical trials of the lipoprotein lipase deficiency therapy before making a decision.
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.