Last month, Europe’s Committee for Medicinal Products for Human Use approved a gene therapy for a rare genetic disease, the first time a Western regulatory agency has okayed such a treatment, though gene therapies have been approved in China.
The virus-mediated treatment, called Glybera, which is being developed by Amsterdam-based UniQure, introduces a normal version of a gene needed to properly break down fats in the blood. Though the condition is rare, patients with dysfunctional copies of this gene have dramatically increased levels of fat in their blood, which can lead to fatal inflammation of the pancreas. The genetic repair lowers blood fat concentration and reduces the frequency of pancreatitis, according to clinical trial data.
The approval is just another sign that the field of gene therapy is quietly enjoying a resurgence after disastrous results in the late 1990s derailed testing of the technology. A number of companies are now testing various gene therapy products. For example, in 2010, Cambridge, Massachusetts-based Bluebird Bio reported it had successfully treated an 18-year-old patient for a rare blood disease (see “Gene Therapy Combats Hereditary Blood Disease”). Hemophilia, immune deficiencies, and blindness have all been treated with at least some benefit to the patients willing to try the experimental therapy. And large pharmaceutical companies such as Novartis, GlaxoSmithKline, and Baxter have taken on gene therapy projects in recent years.