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.
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Ya-Qin Non Zhang (NCATS)
Matthew Boxer (NCATS)
Min Shen (NCATS)
Li Liu (NCATS)
Zhuyin Li (NCATS)
US Application No. 61/562,894
PCT Application No. PCT/US2012/066394
Tara Kirby , Ph.D.
NIH Office of Technology Transfer
6011 Executive Blvd. Suite 325
Rockville , MD 20852
OTT Reference No: E-277-2011/0