Technology ID
TAB-3538

Treatment for Wolfram Syndrome and Other Endoplasmic Reticulum Stress Disorders with Endoplasmic Reticulum Calcium Modulators

E-Numbers
E-095-2017-0
Lead Inventor
Urano, Fumihiko (Washington University School of Medicine)
Co-Inventors
Mahadevan, Jana (Washington University School of Medicine)
Jadhav, Ajit (NCATS)
Yang, Shyh-Ming (NCATS)
Maloney, David (NCATS)
Henderson, Mark (NIDA)
Harvey, Brandon (NIDA)
Applications
Therapeutics
Therapeutic Areas
Endocrinology
Lead IC
NCATS
ICs
NIDA
NCATS
This technology includes the use of JTV-519 and oxidized form of JTV-519, as a novel treatment for Wolfram syndrome and other diseases associated with endoplasmic reticulum (ER). JTV-519 can prevent the leakage of ER calcium to the cytosol and abnormal activation of a pro-apoptotic enzyme, calpain 2, in cell models of Wolfram syndrome. Further, these compounds can prevent cell death in beta cell models of these diseases.
Commercial Applications
Wolfram syndrome and other diseases associated with endoplasmic reticulum.

Competitive Advantages
Novel treatment for diseases currently lacking therapeutic options.
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