Celladon's lead program--as well as its stock price--crashed after the biotech announced Sunday evening that its heart therapy Mydicar failed the primary and secondary endpoints in a Phase IIb trial, losing out in a full lineup of efficacy tests.
A biotech with deep roots in the European gene therapy field has successfully tested a new approach to creating a personalized treatment for a rare disorder of the immune system.
Two months after tying the Big Pharma collaboration knot with Sanofi, Cambridge, MA-based gene therapy startup Voyager Therapeutics has rolled out a $60 million venture round that appears to set the stage for an upcoming IPO.
MultiVir, a developer of viral vectors to deliver anticancer gene therapy, just filed for a $70 million IPO with the Securities and Exchange Commission as it seeks funding for clinical trials of its Phase I/II lead candidates for colorectal cancer and head and neck cancer, and to take the first FDA-approved gene therapy to the market.
Sanofi subsidiary Genzyme is tying up with gene therapy upstart Voyager Therapeutics, gambling $100 million upfront on the hot field and promising up to $745 million in milestones for their partnership on a full slate of development programs.
Bluebird bio nabbed the FDA's breakthrough drug title for LentiGlobin BB305 as a new treatment for beta-thalassemia major, putting one of the industry's top experimental gene therapies back squarely in a promising spotlight.
Spark Therapeutics, at work on one-time treatments for rare diseases, pulled off a $161 million IPO, pricing above its range and keeping biotech's Wall Street hot streak rolling.
Cashing in on the current enthusiasm in venture circles for gene therapy upstarts, ReGenX has snagged a $30 million round that will finance a round of new hires as it sets sail for the clinic with lead programs for rare diseases of the central nervous system.
Just weeks after Novartis and Atlas Venture announced the launch of an upstart player in the fast-emerging field of CRISPR-Cas9 gene editing, the pharma giant has stepped back up to formally hammer out a partnership development deal and throw its enormous resources behind the biotech.
Spark Therapeutics jumped into existence late in 2013 with a pipeline of gene therapy projects spawned by investigators at the Children's Hospital of Philadelphia and quickly followed up this year with a $73 million round, breakthrough drug status and a $280 million partnership with Pfizer. Now it's ending its freshman year by filing to go public.