The article "Gene Therapy in Lipoprotein Lipase Deficiency (LPLD): Case Report on the First Patient Treated with Alipogene Tiparvovec under Daily Practice Conditions" was coauthored by Ursula Kassner, Tim Hollstein, Thomas Grenkowitz, Marion Wühle-Demuth, Bastian Salewsky, Ilja Demuth, Elisabeth Steinhagen-Thiessen, from Chari-té - Universitätsmedizin Berlin, and Michaela Dippel, MD Medscript, Bad Dürheim, Germany.
‘Gene therapy with alipogene tiparvovec offers a potential cure for LPLD, which is currently treated with plasmapheresis.’
LPLD is a rare inherited disorder associated with an increased incidence of inflammation of the pancreas due to elevated triglyceride levels. Gene therapy
with alipogene tiparvovec offers a potential cure for LPLD, which is currently treated with plasmapheresis when maintenance of an ultra-low fat diet, use of fibrates, and other triglyceride-lowering therapies prove inadequate. Alipogene tiparvovec is an adeno-associated virus 1 (AAV1) gene therapy administered via intramuscular injections together with immunosuppression.
Uniqure, the manufacturer of alipogene tiparvovec (marketed as Glybera), has ceased post-marketing studies and withdrawn the product from the market in the European Un-ion due to very limited demand for its use. The withdrawal of Glybera was not related to any risk-benefit concerns.
"While Glybera has not continued in commercial sales, it is very gratifying to gene therapy translational researchers to see a case report of a safe and effective gene therapy product in clinical use," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA.