Small-Molecule Modulators of Lipid Storage for Treatment of Obesity, Atherosclerosis, Metabolic Syndrome and Lipid Storage Diseases

Lipid droplets are key organelles involved in lipid homeostasis. In normal physiology, these droplets are formed in response to elevated fatty acid levels, and are broken down when needed for energy production. Imbalances in lipid homeostasis trigger compensatory alterations in metabolism that can lead to diseases such as obesity, atherosclerosis, and metabolic syndrome. There are also a number of inherited lipid storage diseases that result in harmful buildup of various lipids, such as Gaucher disease, Fabry disease, and others. Reducing the accumulation of lipid droplets is a promising potential strategy for treatment of such disorders.

This technology describes three novel structural classes of small-molecule compounds that significantly reduce the accumulation of lipid droplets. These compounds hold promise for the treatment of diseases associated with aberrant lipid deposition.

Potential Commercial Applications: Competitive Advantages:
  • Treatment of inherited metabolic diseases such as Gaucher disease, Fabry disease, and Tay Sachs disease.
  • Treatment of obesity and metabolic disease.
  • Treatment of atherosclerosis.
  • Modulation of lipid droplet accumulation is a novel mechanism for treatment of lipid storage diseases.

Ya-Qin Non Zhang (NCATS)
Matthew Boxer (NCATS)
Mathias Beller
Kirsten Tschapalda
Min Shen (NCATS)
Li Liu (NCATS)
Carole Sztalryd
Zhuyin Li (NCATS)

Intellectual Property:
US Application No. 61/562,894
PCT Application No. PCT/US2012/066394

Licensing Contact:
Lauren Nguyen-Antczak , Ph.D., J.D.
NIH Office of Technology Transfer
Phone: 301-496-7057

OTT Reference No: E-277-2011/0

Updated: Sep-30-2013