gene therapy

Latest Headlines

Latest Headlines

UniQure beats a path to Phase III after a 'promising' gene therapy trial

UniQure is pressing forward with its gene therapy for a rare and deadly disease after charting a positive signal in a small yearlong trial, news that sent the Dutch company's share price soaring.

RegenX IPO raises $138M, renewing hopes for a big Q4 biotech run

The biotech IPO window is still wide open and ready for new business, especially if it relates to a hot new tech like gene therapy. RegenX Bio proved that point today, raising $138 million from the sale of 6.3 million shares at $22 a pop.

Gene therapy player Dimension catches the biotech IPO wave, files $115M offering

Dimension Therapeutics is preparing to execute the next carefully planned step in its evolution, filing for a $115 million IPO close to 5 months after its crossover round landed. The filing came just days after the FDA accepted Cambridge, MA-based Dimension's IND for its lead gene therapy program, targeting a genetic fix for hemophilia B.

Roche backs AveXis in a $65M round for SMA gene therapy

Gene therapy biotech AveXis raised $65 million in Series D cash to advance a potential one-time treatment for the rare spinal muscular atrophy, a debilitating disease with no approved therapies.

Gene therapy upstart Audentes bags a new program in buyout

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.

Gene therapy stalwart ReGenX plots a $100M IPO to strike out on its own

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 hits the brakes on its gene therapy program after a Phase II misstep

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.

Harvard researchers reconstruct ancient virus into a 'next-gen' vector for gene therapy

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.

Nonviral gene therapy shrinks brain tumors in rats

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.

Gene therapy signals hope for inherited deafness

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.