Glybera's clinical program

Lipoprotein lipase (LPL) deficiency is characterized by severe hypertriglyceridemia leading to acute pancreatitis. uniQure B.V.'s gene therapy Glybera alipogene tiparvovec is designed to replace the absent enzyme and thus restore proper clearance of triglycerides from plasma. The product comprises an AAV vector encoding the LPL gene for one-time delivery by intramuscular injection.