UniQure drug first gene therapy to get approval in West

The European Commission has given the OK to the gene therapy from Dutch firm UniQure for an extremely rare condition, the first approval for gene therapy to make it past regulators in the West. The approval is seen as a boost for the whole field of gene therapy, which has languished in recent years. The drug is Glybera, which is for certain patients with lipoprotein lipase deficiency (LPLD), an enzyme deficiency that robs them of the ability to metabolize fat-laden particles in their blood. It can lead to inflammation of the pancreas and other complications, such as diabetes. Glybera, which is indicated for patients whose disease can be controlled with diet and who suffer from pancreatitis, is designed to mend the genetic defect underpinning the ailment. Regulators had turned down the therapy repeatedly before supporting its approval, and other experimental gene therapies have stumbled as well. Yet in recent years, groups such as uniQure and Cambridge, MA-based Bluebird Bio have generated upbeat results and renewed faith in the field among investors. Story | More

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