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Gene Therapy Gets the Go-Ahead in Europe



If doctors detect any negative issues with the genetic makeup of a patient, gene therapy simply dictates that the genetic code be changed.

In a successful clinical breakthrough, the commercial use of a treatment used to correct errors in a person’s genetic code has been commercially approved in Europe for the first time. The gene therapy has been developed to combat a rare disease that inhibits the successful digestion of fats in the body. Glybera has received marketing authorization from the European Commission allowing it to be sold across the EU. The manufacturers have said that is will be released into the market in 2013.

The Drug

Glybera is used to treat the very rare case of lipoprotein lipase deficiency. Only one in a million people are born with damaged copies of the gene, which is an essential component used for breaking down fats. The gene therapy specifically tackles that gene. The LPL gene is packaged in a delivery vector derived from adeno-associated virus (AAV), serotype 1, which has a natural propensity towards muscle cells In the United States and Europe the drug has only been used gene therapy in research labs. The drug is administered via a one-time series of small intramuscular injections in the legs.

The Disease

The failure to adequately break down the fat results in a severe build-up of fats in the blood that can lead to abdominal a potentially terminal case of pancreatitis – the inflammation of the pancreas. If one suffers from the disease the only way to keep the harmful effects at bay without gene therapy has been to live on a very low-fat diet.

The Company

UniQure is the company who developed the gene therapy makes use of a special virus designed to invade and infect muscle cells with a healthy copy of the damaged gene.

Because of the nature of such therapy, the use of Glybera was deemed to be very restricted. The European Medicines Agency recommended and thus it affected earlier this year to only patients with the most severe cases of the disease.

The Breakthrough

The chief executive officer at UniQure, Jorn Aldag announced that the concluding approval from the European Commission symbolizes a great progression in the motion for making gene therapy accessible to a host of other rare diseases – in addition to the lipoprotein lipase deficiency – that have medical needs yet to be met by other treatments.