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Fatty Acid Amides Treatment of Osteoporosis for Prader-Willi Syndrome

Tam Joseph, HUJI, School of Medicine - IMRIC, School of Pharmacy- Institute for Drug Research

 

Categories:

Life Science & Biotechnology

Technology Keywords:

Rare diseases, Osteoporosis

Development Stage:

TRL 3 - In vivo POC

Patent Status:

PCT

 

Among a multitude of hormonal and metabolic complications, individuals with Prader-Willi syndrome (PWS) rare disease exhibit significant bone abnormalities, including decreased bone mineral density, osteoporosis, and subsequent increased fracture risk.

Our Innovation

Our innovation is directed to methods of treating bone diseases associated with Prader-Willi syndrome (PWS). The treatment is based on fatty acid amide, oleoyl serine (OS), or with a synthetic derivative of OS.

Clinical Results

  • Low skeletal and circulating levels of the endogenous bone homeostasis regulator, N-Oleoyl serine (OS), were found in a mouse model for PWS.
  • Reduced blood OS levels were detected in individuals with PWS and Schaaf-Yang syndrome (SYS), another genetic disorder caused by a disruption of the MAGEL2 gene. These reduced levels of OS were positively linked with their reduced bone mineral density.
  • Attenuating the skeletal abnormalities in the mouse model for PWS was achieved with chronic administration of a novel synthetic derivative of OS.

Nest Development Stage

Testing the efficacy of OS or its synthetic derivative in reversing bone loss in PWS and SYS patients.

Opportunity

Prader-Willi syndrome (PWS) is a genetic disorder that occurs in approximately one out of every 15,000 births. Diminished bone mineral density (BMD) and osteoporosis are common in PWS individuals, especially in adolescence and adulthood. 

Patent Status

Published WO 2017/179058 A1

Contact for more information:

Shoshana Keynan
VP, Head of Business Development, Healthcare
+972-2-6586683
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