Gene Therapy Goes On Sale Mid-2013

The West's First Gene Therapy Goes On Sale Mid-2013We're tampering with genes to cure a rare metabolic disorder--and that's likely just the beginning.
By Clay DillowPosted 11.06.2012 at 2:45 pm

Gene Therapy: Tampering With DNA Using genes to generate protein therapies in the body. National Institutes of Health




The first gene therapy to be approved in the West will hit the market by the middle of next year, opening the masses to a controversial treatment that directly alters a patient’s own DNA. Dutch biotech uniQure’s Glybera was approved for sale by the European Commission late last month.

Gene therapies emerged--appropriately--about the same time the first human genome was being mapped during the 1990s, though the study of gene therapies goes back as far as the 1970s. They work by actually tampering with a person’s DNA--usually by encoding a functioning gene to replace a mutated one, or by introducing DNA that encodes a therapeutic protein into the body. Clinical trials have gone on for years. Early on, failures caused many to dismiss the idea of tampering with genes. But later--in the following decade--many in the medical community changed their minds after witnessing a series of successes. Among them: China, which approved its first gene therapy in 2003.

In the West, however, gene therapy has been a source of constant debate, and that’s what makes this development notable. Gene therapies carry a lot of promise, including the ability to treat any number of inherited diseases that have few treatment options. They are a way to literally tinker with the fundamental material that tells our cells how to function, so their potential is indeed vast--if we can make them work.

Glybera will treat lipoprotein lipase deficiency (LPLD), an extremely rare inherited disorder affecting the metabolism of certain fat particles. It affects something like one or two people in a million, so it’s not like we’re curing blindness here. Gene therapy is still limited to single-gene disorders--and most common diseases are more complex multi-gene problems.

Still, the acceptance of the first gene therapy into Western medicine could mark a turning point for gene medicine, provided nothing goes wrong. Many labs are still working on gene therapies for a number of conditions, and uniQure itself is working on additional genetic therapies for everything from hemophilia to Parkinson’s. Even limited success there would naturally be a huge leap forward for medical science.

News
First gene therapy to go on sale in Europe in 2013
Tuesday, 6 November 2012

Agençe France-Presse


Gene therapy, approved in China as long ago as 2003, will be made available in Europe from mid-2013. It works by modifying a patient's DNA, offering the theoretical hope of blocking inherited diseases.

Credit: Veer Images

THE HAGUE: Dutch biotech company uniQure said it would start selling the first human gene therapy to be approved in the West by mid-2013 and predicted an explosion of similar therapies to come.

The European Commission approved Glybera on October 25, making the drug for treating the extremely rare disorder lipoprotein lipase deficiency (LPLD) the first to be approved for sale in Europe or North America.

"We believe that after Glybera's approval gene therapy is at the beginning of a period of rapid growth similar to the development of the antibody business in the last decade," uniQure chief executive Joern Aldag said in a statement.

Blocking inherited disease

Gene therapy works by modifying a patient's DNA to combat a specific disease, and has been experimented with to treat everything from blindness to depression and brain wasting diseases.

But the relatively unknown treatments have struggled to obtain regulatory approval in the West, although authorities in China approved a gene therapy for treating head and neck cancer as long ago as 2003.

Gene medicine burst on the medical scene in the late 1990s and is one of the most alluring areas of biotechnology, offering the theoretical promise of blocking or reversing inherited disease.

But this new frontier has also been hit by occasional setbacks, notably an unexpected or uncontrollable response from the immune system.

Successes few, limited to single-gene disorders

So far, successes have been few, limited to single-gene disorders – as opposed to complex multi-gene disorders that account for the most common diseases.

Setbacks included the tragic death of an 18-year-old U.S. volunteer, Jesse Gelsinger, in 1999, and the development of cancer among two French children treated for ‘bubble baby’ syndrome, a chronic lack of immune defences.

Glybera treats LPLD, an inherited disease that affects around one or two people in a million, by preventing them from metabolising certain fat particles in the blood.

"First commercial sales of Glybera in Europe are expected in summer 2013," uniQure said, with the company also seeking approval for the drug in North America.

The company said it was developing a raft of other gene therapies to treat diseases including blood clotting disorder haemophilia B, metabolic disorder acute intermittent porphyria, central nervous system disorder Parkinson's and enzyme disorder Sanfilippo B.