Tag:

gene therapy

Latest Headlines

Latest Headlines

UPDATED: Celladon shares blasted as gene therapy for heart failure bombs in PhIIb

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.

Small European study highlights promise of gene therapy for rare immune disease

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.

Big Pharma partner: Check. Crossover round: Check. Hot tech: Check. IPO...

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.

Developer of viral vectors for anticancer gene therapy files for $70M 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 embarks on an $845M gene therapy R&D odyssey with Voyager

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.

Gene therapy pioneer bluebird bags a 'breakthrough' title for lead program

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 nails a $161M IPO to fund its 'breakthrough' gene therapy

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.

Gene therapy pioneer ReGenX fuels R&D, hiring spree with $30M VC round

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.

Novartis adopts a CRISPR-Cas9 partner and jumps into the hot new R&D field

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.

Upstart Spark is ready to ring in the New Year with $86M gene therapy IPO

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.