Gene Therapy Arrives in Europe

The European Commission approves the Western hemisphere’s first gene therapy, aimed at correcting a lipid-processing disorder.

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stock.xchng, tijman.The field of gene therapy has a long but frustrating history, with early safety concerns that threatened to shutter its clinical development altogether. Now, almost 10 years after China sanctioned the world’s first gene therapy in 2003, the strategy has arrived in Europe. (There are still no approved gene therapies in the United States.)

Approved last week (November 2) by the European Commission, uniQure’s Glybera utilizes viral vectors to deliver DNA encoding a lipid-processing enzyme to patients lacking a functional copy due to a gene mutation. The therapy, which is indicated for patients with severe symptoms such as life-threatening bouts of pancreatitis, will be sold in Europe beginning in late 2013.

“It’s really a milestone that this product has been approved,” said Katherine High, a pediatrician at the University of Pennsylvania and Children’s Research Hospital of Philadelphia, who was not involved in research on Glybera. It marks the first time that a gene therapy has been “judged safe and efficacious for human clinical use,” according to regulatory standards used in Europe and the United States, she noted.

“It’s really ...

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