gene therapy

Latest Headlines

Latest Headlines

Proteostasis and AveXis test the troubled IPO waters as biotech valuations wane

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.

Annapurna adds three new gene therapy programs from Ron Crystal's lab at Weill Cornell

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.

Wellcome invests $38M in another gene therapy startup, targeting hemophilia

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 adjusts to a new reality with its much-scrutinized gene therapy

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 abandons ambition to win FDA approval for €1.1M gene therapy

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.

RetroSense bags $6M in funding for its viral vector gene therapy candidate for a rare eye condition

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.

NEA joins a $35M round for a Wellcome-backed gene therapy player

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.

Dimension pulls off a downsized $71.5M IPO amid biotech's Wall Street swoon

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.

Bluebird shares dented after gene therapy patient experiences a setback

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.

Gene therapy study in dogs demonstrates success in preserving vision

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.