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Gene therapy approved for first time to treat disease
Last reviewed: 01.07.2025
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A landmark event in European medical history is about to occur: the first approval for the practical use of gene therapy to treat a hereditary disease.
The European Medicines Agency has recommended the treatment for a rare genetic disorder that leaves patients unable to digest fat.
Based on this recommendation, the European Commission must make a final decision on the admissibility of this treatment method.
The principle of gene therapy is simple: if a defect is found in some part of a person’s genetic code, it is replaced with laboratory-created genetic material with the desired properties.
However, in reality, things are not so simple. During clinical trials in the US, teenager Jesse Gelsinger died, and other patients fell ill with leukemia.
Currently, gene therapy methods are not used in Europe or the USA.
The first gene medicine
The European Medical Commission's Committee on Medicinal Products has reviewed the use of the medicinal product Glybera for the treatment of Buerger-Grutz disease, an inherited deficiency of lipoprotein lipase.
This syndrome affects one in a million people. Sufferers have a damaged gene structure that is responsible for breaking down fats in the digestive tract.
This leads to accumulation of fats in the blood, groin pain and acute pancreatitis.
Until now, the only way to alleviate the condition of such patients was a strict fat-free diet.
The new treatment uses a virus that infects muscle tissue and introduces a copy of the intact gene into the body.
It is recommended for patients who suffer from acute inflammation of the pancreas, pancreatitis, and who do not respond to diet.
Replacement of a defective gene
The new drug's maker, pharmaceutical company UniQure, said the decision was a landmark event for patients and for medicine as a whole.
The company's CEO, Jorn Aldag, said: "Patients with lipoprotein lipase deficiency are afraid to eat normal food because it can lead to acute and extremely painful inflammation of the pancreas, which often ends in hospitalisation."
"Now - for the first time in history - a treatment method has been created that not only reduces the risk of inflammation, but also has a long-term beneficial effect on the patient's condition," he added.
China has become the first country in the world to officially approve the use of gene therapy methods.
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