According to George Dvorsky in We come from the future: The idea of treating genetic diseases by replacing a defective gene with a working copy has been around for nearly three decades, but the road to creating an actual therapy that's safe and effective has proven to be a long and difficult one. Now, thanks to the efforts of the Dutch biotech company uniQure, the Western world's first therapy to correct errors in a person's genetic code has finally been approved. While the therapy is extremely expensive and limited to a rare genetic disorder, the approval is set to open the door for similar interventions.
The drug is called Glybera and it's intended for the treatment of a genetic disorder called Lipoprotein Lipase Deficiency (LPLD), a rare, inherited disease that makes it impossible to metabolize fatty acids found in blood, what results in inflammation of the pancreas. The disorder affects about 1 in every one million people.
"This provides higher benefit to patients than the classical protein replacement strategy" he told Reuters. The European Commission gave uniQure its consent to start selling Glybera next summer.
Glybera works by introducing a normal, healthy LPL gene into the patient's body so that it can make functional LPL protein. 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.
The company is also currently working on regulatory approval for Glybera in Canada and the United States.
In future, it's thought that similar therapies will be able to remedy other genetic disorders, including Severe Combined Immunodeficiency Disorder (the so-called "bubble boy" disease).
It's worth noting that Glybera is not the world's first gene therapy drug. Back in 2003, China's Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer (though not for use in Europe or North America).
Sources: Reuters, uniQure. Here is a link to the article in We come from the future.
NanoBioCorp
The drug is called Glybera and it's intended for the treatment of a genetic disorder called Lipoprotein Lipase Deficiency (LPLD), a rare, inherited disease that makes it impossible to metabolize fatty acids found in blood, what results in inflammation of the pancreas. The disorder affects about 1 in every one million people.
"This provides higher benefit to patients than the classical protein replacement strategy" he told Reuters. The European Commission gave uniQure its consent to start selling Glybera next summer.
Glybera works by introducing a normal, healthy LPL gene into the patient's body so that it can make functional LPL protein. 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.
The company is also currently working on regulatory approval for Glybera in Canada and the United States.
In future, it's thought that similar therapies will be able to remedy other genetic disorders, including Severe Combined Immunodeficiency Disorder (the so-called "bubble boy" disease).
It's worth noting that Glybera is not the world's first gene therapy drug. Back in 2003, China's Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer (though not for use in Europe or North America).
Sources: Reuters, uniQure. Here is a link to the article in We come from the future.
NanoBioCorp
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