Late last year San Francisco-based gene therapy upstart Audentes Therapeutics gathered a $42.5 million venture round from some A-list investors with plans to use the money to bankroll some new deals. Today, it made good on the plan and pulled back the covers from a buyout that gives the little biotech player a third preclinical program as it tackles a hot field.
After years of outlicensing its gene therapy technology, ReGenX Bio is pressing forward with treatments of its own, filing to raise $100 million in an IPO to fund in-house R&D.
Avalanche Biotechnologies, months removed from a costly Phase II dud, is calling off plans for further study of its lead gene therapy candidate, heading back to the lab to regroup before trying another clinical trial.
Researchers at the Harvard Stem Cell Institute have built a viral vector based on an ancient virus that is highly effective at delivering gene therapies to the liver, muscle and retina. The findings may allow the use of viruses as a safe and more potent method for gene therapy.
A global team of scientists working with Johns Hopkins' Jordan Green has developed a new nonviral gene therapy for brain cancer, successfully testing it in the lab in cell lines as well as rat models for the disease.
The rapid maturation of gene therapy technologies has galvanized a wide range of projects throughout industry and academia, and now a team of researchers from Harvard and Boston Children's Hospital believe they may have come up with a one-time treatment for a genetic cause of deafness.
Parisian drug developer Gensight Biologics is swinging for a $100 million U.S. IPO to fund its work on potential one-time treatments for serious retinal diseases, angling to take advantage of a bullish market for biotechs.
Celladon is circling the drain, suspending all research tied to its failed gene therapy and again halving its payroll as management searches for a sale and flirts with liquidation.
Baxalta won't formally debut as an independent Baxter drug spinoff for a few more days, but the company isn't waiting to discuss an early peek at efficacy results from a Phase I/II study of their long-term gene therapy for hemophilia.
Immuno-oncology innovator Kite Pharma is joining gene therapy luminary bluebird bio to craft new treatments for HPV-related cancers, combining two high-profile technologies in hopes of developing targeted therapies.