European regulators have approved the first therapy in the western world that can correct errors in a person’s genetic code, according to Amsterdam-based uniQure (formerly Amsterdam Molecular Therapeutics).
Europe has approved Glybera for treatment of Lipoprotein Lipase Deficiency (LPLD), a very rare, inherited disease. Patients with LPLD are unable to metabolize the fat particles carried in their blood, which leads to inflammation of the pancreas (pancreatitis).
Glybera introduces a normal, healthy LPL gene into the 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.
Glybera will be available in the second half of 2013, according to uniQure, which is preparing to apply for regulatory approval in the U.S., Canada, and other markets.
First therapy in the western world to correct errors in a person’s genetic code approved