Gene therapy nears regulatory approval in Europe

The New York Times reports that the European Medicines  Agency has recommended approval of gene therapy to treat a rare disease.  Should the European Commission follow the advice, it would become the first approval of a gene therapy drug in the Western world. 

According to the NYT:

Gene therapy involves providing the body with genes it needs, such as correct copies of defective genes that cause genetic disorders. Its use in the West so far has been confined to clinical trials.

The therapy recommended for approval in Europe, called Glybera, was developed by uniQure, a Dutch company. It treats lipoprotein lipase deficiency, a disease that affects only several hundred people in the European Union and a similar number in North America.

People with the disease have a genetic mutation that prevents them from producing an enzyme needed to break down certain fat-carrying particles that circulate in the bloodstream after meals. Without the enzyme, so much fat can accumulate that the blood looks white rather than red.

Glybera provides correct copies of the lipoprotein lipase gene, which allows patients to make some of the needed enzyme. A single treatment, consisting of injections into multiple spots on the leg muscles on the same day, is expected to last for several years, if not longer, said Jörn Aldag, chief executive of uniQure.

Read the full story here.