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Gene Therapy Nears Regulatory Approval in Europe

The European Medicines Agency (EMA) has recommended approval of a gene therapy to treat a rare genetic disease. If the European Commission agrees, it would mark the first regulatory approval of a gene-therapy drug in the Western world.

The drug recommended for approval in Europe, 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, large amounts of fat can accumulate in the blood.

Glybera provides correct copies of the lipoprotein lipase gene, which allows patients to make some of the needed enzyme.

The clinical efficacy of Glybera is open to question, however, since uniQure tested the drug in only 27 patients, without rigorous, controlled trials.

Read the full story on the New York Times Web site.

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