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.
Pfizer is the latest pharma heavyweight to get behind the promise of gene therapy, as the New York drugmaker has laid out plans to set up a dedicated R&D operation and signed a deal with startup Spark Therapeutics to kick-start the effort.
Gene therapy startup Spark Therapeutics has picked up the FDA's coveted breakthrough-therapy designation for its lead candidate, a one-time treatment that promises a permanent solution some rare eye diseases.
Japan's Astellas Pharma is the latest big drugmaker allured by the promise of gene therapy, mounting an R&D effort that could lead to a new treatment for an inherited eye disease.
Using a new type of cell transplantation, researchers at Cincinnati Children's Hospital Medical Center corrected a lung disease in mice that mimics a similar rare disease in humans.