title: Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency.
abstract: Alipogene tiparvovec (AAV1-LPL(S447X)) gene therapy is developed to prevent complications and decrease the clinical morbidity of lipoprotein lipase deficiency (LPLD). LPLD is an autosomal recessive disease associated with severe hypertriglyceridemia (hyperTG), severe chylomicronaemia, and low HDL. Acute pancreatitis, the most frequent serious clinical LPLD complication, is a complex and heterogeneous inflammatory condition having many causes including hyperTG and chylomicronaemia. In many patients, low fat diet and currently available lipid lowering drugs are ineffective to prevent hyperTG or pancreatitis in LPLD. The clinical development program of alipogene tiparvovec includes observational studies as well as phase I/II and II/III clinical trials. Pooled data are collected on safety and efficacy issues, including the incidence of pancreatitis.
authors.affiliation: Department of Medicine, Université de Montréal, ECOGENE-21 Clinical Research Center, Chicoutimi Hospital, Canada. daniel.gaudet@umontreal.ca
authors.forename: Daniel
authors.initials: D
authors.lastname: Gaudet
authors.forename: Janneke
authors.initials: J
authors.lastname: de Wal
authors.forename: Karine
authors.initials: K
authors.lastname: Tremblay
authors.forename: Stéphane
authors.initials: S
authors.lastname: Déry
authors.forename: Sander
authors.initials: S
authors.lastname: van Deventer
authors.forename: Andreas
authors.initials: A
authors.lastname: Freidig
authors.forename: Diane
authors.initials: D
authors.lastname: Brisson
authors.forename: Julie
authors.initials: J
authors.lastname: Méthot
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics; D019317:Evidence-Based Medicine; D015316:Genetic Therapy / Q000009:adverse effects / Q000379:methods*; D005822:Genetic Vectors; D006801:Humans; D008072:Hyperlipoproteinemia Type I / Q000201:enzymology / Q000235:genetics / Q000628:therapy*; D007273:Injections, Intramuscular; D008071:Lipoprotein Lipase / Q000096:biosynthesis / Q000235:genetics*; D010195:Pancreatitis / Q000201:enzymology / Q000235:genetics / Q000517:prevention & control; D018570:Risk Assessment; D016896:Treatment Outcome
chemical_list: C548261:LPL protein, human; D008071:Lipoprotein Lipase