Lysosomal phospholipase A2 to study and treat bis(monoacylglycero)phosphate-related disorders

This technology is an enzyme that is involved in synthesizing a precursor to bis(monoacylglycero)phosphate (BMP) and can be used to modulate BMP levels and treat BMP-related disorders.

Unmet Need: A means to modulate endogenous bis(monoacylglycero)phosphate (BMP) levels

Bis(monoacylglycero)phosphate (BMP) is a lipid involved in many cellular processes such as endosomal and lysosomal trafficking. BMP dysregulation is implicated in a variety of neurodegenerative diseases and viral infections, but there are currently no therapeutics to modulate endogenous BMP levels, and little is known about its biosynthetic enzyme.

The Technology: BMP precursor enzyme for studying and treating BMP-related disorders

This technology identifies an enzyme, lysosomal phospholipase A2 (LPLA2), that is localized to late endosomes and lysosomes, involved with converting phosphatidylglycerol to lysophosphatidylglycerol, a precursor for the synthesis of BMP. LPLA2 has been shown to modulate BMP levels in human cell lines and to decrease cholesterol levels in fibroblasts of Niemann-Pick disease patients, a storage disorder in which cholesterol accumulates in lysosomes.

This technology has been validated with a cell model of Niemann-Pick disease type C.

Applications:

  • Therapeutic for disorders in which bis(monoacylglycero)phosphate (BMP) is dysregulated
  • Research tool for modulating and characterizing BMP-related disease pathways
  • Research tool for investigating cellular cholesterol levels

Advantages:

  • Aids understanding of BMP biology
  • Reveals a precursor in BMP synthesis
  • Treats disorders with BMP-dependent pathways
  • Treats Niemann-Pick disease type C

Lead Inventor:

Catherine Marquer, Ph.D.

Patent Information:

Patent Pending

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