When Will Gene Therapy Come to the U.S.?

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 Children’s 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 good chance of also getting the first U.S...