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27.11.2012

First gene therapy approval

On November 2, 2012, a drug referred to as Glybera (produced by uniQure) became the first ever gene therapy to be approved for market, in the western world. The news of this gene therapy approval has generated a lot of excitement, as it is hoped that it will pave the way for other gene therapy approvals.

It is often commented that gene therapy is always 5 years away. Despite the fact that this approach to the treatment of human genetic diseases was deemed to have great promise over 25 years ago, it is a field that has been frustratingly slow to come to fruition. While there are many gene therapies for various conditions currently undergoing testing in clinical trials, no gene therapy is as yet used in routine healthcare in either Europe or the US. For one rare condition however, the news that a gene therapy has been approved for use in Europe, means that the wait is almost over.

On November 2, 2012, a drug referred to as Glybera (produced by uniQure) became the first ever gene therapy to be approved for market, in the western world. The drug will be used to treat patients with a form of the rare condition, lipoprotein lipase deficiency (LPLD). Due to a faulty LPL gene, people with LPLD are unable to break down the large fat particles that accumulate in the blood after a meal, leading to a range of complications, including inflammation of the pancreas and diabetes. The active ingredient in Glybera is a good copy of the LPL gene, which has been packaged into a virus (a viral vector) to facilitate its delivery to muscle cells.  It will be administered to patients on a one-off basis, by injection into muscle.

The news of this gene therapy approval has generated a lot of excitement, as it is hoped that it will pave the way for other gene therapy approvals. People familiar with EB research will know that, in addition to a focus on protein, cell and small molecule therapies, enormous effort has been directed towards potential gene therapies, over the last decade. Gene therapy is considered by many the 'holy grail' of the different treatment approaches for EB as it has the potential to treat, not the symptoms, but the underlying cause of the condition.

Unfortunately, while gene therapy holds enormous promise, it has shown itself to be one of the most challenging forms of EB therapy to develop. Technical difficulties at various points in the development pathway and concerns over the safety of the viral vectors have hampered progress. Despite the difficulties however, a number of research groups, most notably in the US, France and Italy, have persisted in their efforts to develop a gene therapy for EB. This recent news of the first approval for a gene therapy will be very welcome to those research groups and should give heart to people living with EB that gene therapy holds realistic promise.

For more information on Glybera and gene therapy see this article:

The Scientist (Nov 6, 2012): Gene Therapy Arrives in Europe

Hand holding a pipette with drop of liquid

Image courtesy of ponsulak at FreeDigitalPhotos.net