Gene therapy improves chylomicron metabolism in LPLD patients

Mar 29 2012

Amsterdam Molecular Therapeutics (Euronext: AMT), a leader in the field of human gene therapy, announced today data demonstrating that one-time administration of the gene therapy Glybera® (alipogene tiparvovec) is able to markedly improve chylomicron (fat particles in the blood) metabolism following consumption of a low fat meal. This results in a much reduced level of newly-formed chylomicrons in the bloodstream, which are considered to be the cause of the acute and recurring bouts of pancreatitis seen in lipoprotein lipase deficiency (LPLD) subjects. LPLD is a very rare inherited condition that is associated with increased levels of chylomicrons. These particles carry certain types of fat in the blood, which because they are not removed from the body can cause recurrent pancreatitis. Data were published online in the Journal of Clinical Endocrinology & Metabolism (JCEM, Mar 2012).

"These data show that Glybera has a profound impact on chylomicron metabolism 14 weeks after a single administration. Although the patient cohort is small, due to the rare nature of LPLD, these results are very encouraging," explained Dr. André Carpentier, Division of Endocrinology at the Université de Sherbrooke, Quebec, Candada. "LPLD patients often suffer from extremely painful bouts of pancreatitis, which is believed to be caused by the accumulation of chylomicron particles in the blood."

"This publication provides additional, independent support on the ability of Glybera to restore chylomicron metabolism in LPLD patients. We believe by restoring the body's ability to metabolize these particles in LPLD patients, Glybera treatment results in fewer pancreatitis attacks," stated Carlos Camozzi, Chief Medical Officer at AMT. "LPLD patients are under constant risk of these attacks and the associated excruciating pain."