Glybera is a human gene therapy developed by uniQure to treat lipoprotein lipase deficiency (LPLD). LPLD is a rare genetic disorder that prevents the breakdown of fats in the blood, leading to potentially life-threatening pancreatitis. Glybera was the first gene therapy to be approved for use in Europe and North America. It was approved by the European Commission in 2012 and by Health Canada in 2015.
Glybera consists of a modified adeno-associated virus (AAV) that carries the human gene for lipoprotein lipase (LPL). The AAV is injected into a muscle, where it infects cells and expresses the LPL gene. The expressed protein then breaks down fats in the blood, reducing the risk of pancreatitis.
In clinical trials, Glybera was shown to be effective at reducing the incidence and severity of pancreatitis attacks in patients with LPLD. The most common side effects were mild injection site reactions and flu-like symptoms. Glybera is administered as a single infusion and has been shown to be safe and efficacious with long-term follow-up out to 4 years.
uniQure is currently working on expanding the indications for Glybera beyond LPLD, including other lipid disorders such as familial chylomicronemia syndrome (FCS) and familial partial lipodystrophy type 2 (FPLD2). In addition, uniQure is developing an next-generation AAV platform that will enable more widespread use of gene therapy.