Amsterdam, The Netherlands, June 4, 2010 – Amsterdam Molecular Therapeutics (Euronext: AMT) a world leader in gene therapy, today reports new data showing that it’s lead product Glybera results in break-down of chylomicrons in lipoprotein lipase deficient (LPLD) patients. The data will be presented on June 5 at the Second International Symposium on Chylomicrons in Disease, Sint Franciscus Gasthuis, Rotterdam, The Netherlands.
Glybera is a gene therapy product that induces functional lipoprotein activity. New data from an ongoing Canadian clinical study indicate that a single administration of Glybera in LPLD patients results in a remarkable improvement in the ability to break down the chylomicrons that transport dietary fat (triglycerides). Lipoprotein-lipase-deficient (LPLD) patients are incapable of clearing chylomicrons which are responsible for causing significant morbidity and mortality.
“The long-term improvement in chylomicron handling following Glybera administration is very impressive”
said Dr. André Carpentier, co-investigator from the University of Sherbrooke, Quebec, Canada, who designed and analyzed the chylomicron sub-study.
“These data are important, because the major complications observed in LPLD patients, including pancreatitis, are a consequence of chylomicron overload. They also constitute evidence for a long term clinically relevant lipoprotein lipase activity induced by Glybera”
noted the principal investigator, Prof. Daniel Gaudet, from the University of Montreal, and ECOGENE-21 clinical study center, Chicoutimi, Quebec, Canada.
These new data provide a basis for explaining the mechanism of action of Glybera in LPLD patients, and in general for continued pharmacologic activity after one time gene therapy.
LPLD is a seriously debilitating, and potentially lethal, orphan disease, for which no approved therapy exists today. The disease is caused by mutations in the LPL gene, resulting in highly decreased or absent LPL activity in patients. LPL activity is needed in order to break down Chylomicrons, large fat-carrying particles that are formed in the gut and enter the circulation after each meal. When such particles are not adequately broken down they accumulate in the blood, they may obstruct small blood vessels, which in turn can lead to pancreatitis. Recurrent pancreatitis in LPLD patients can result in difficult-to-treat diabetes.
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