Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency

Atheroscler Suppl. 2010 Jun;11(1):55-60. doi: 10.1016/j.atherosclerosissup.2010.03.004. Epub 2010 Apr 27.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Dependovirus / genetics
  • Evidence-Based Medicine
  • Genetic Therapy / adverse effects
  • Genetic Therapy / methods*
  • Genetic Vectors
  • Humans
  • Hyperlipoproteinemia Type I / enzymology
  • Hyperlipoproteinemia Type I / genetics
  • Hyperlipoproteinemia Type I / therapy*
  • Injections, Intramuscular
  • Lipoprotein Lipase / biosynthesis
  • Lipoprotein Lipase / genetics*
  • Pancreatitis / enzymology
  • Pancreatitis / genetics
  • Pancreatitis / prevention & control
  • Risk Assessment
  • Treatment Outcome


  • LPL protein, human
  • Lipoprotein Lipase