Susan Young for MIT Technology Review 2013-09-30 14:11:37 UTC

Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials.

A big step forward already came in November 2012, when the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. That approval came as a relief to many in the field, who had been waiting for a break in the clouds hanging over the technology since failed and fatal trials in the 1990s. You see a resurgence in terms of investors, and in truth, a number of problems have been solved, says Katherine High, a medical researcher at Childrens Hospital of Philadelphia, who is overseeing a late-stage clinical trial for a different gene therapy.

Still, experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have a good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

Like most gene therapies, UniQures treatment uses a modified virus to deliver a working copy of a gene to patients who lack a healthy version. In this case, the gene is needed for the body to break down fats; without it, patients can develop painful and even fatal inflammation of the pancreas. UniQure uses a modified version of a virus that most of us already carry. The choice of virus used to deliver a gene therapy depends in part on where the treatment needs to go in the body and whether the viruses are intended to replicate themselves. Some viruses, for instance, are designed to spread throughout the body to kill cancer cells.

There are several groups that could be the first to develop a U.S.-approved gene therapy (see table). Highs team is one; they are enrolling patients in a late-stage trial of a treatment for a disorder that causes blindness at an early age. The patients in this trial have previously been given the gene therapy in one eye, and now the other will be tested.

In the experimental treatment, doctors inject a virus-based particle just behind a patients retina. The treatment improved some patients vision to the point that they were no longer legally blind. Some patients have been stable for nearly six years. The trial is scheduled to end in April 2015.

Another possibility comes from Massachusetts-based Bluebird Bio, which has published results from patients who have seemingly been cured of a genetic blood disease. The company is about to start testing its approach in a hereditary neurological disorder that is often fatal in young boys.

SEE ALSO: Gene Therapy Combats Hereditary Blood Disease

The rest is here:
When Will Gene Therapy Come to the United States?

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