Technology Bundle ID
TAB-3601

Development of a Therapy for the Treatment of Zellweger Spectrum Disorder

Applications
Linked ID
E-230-2016-0
Lead Inventors
James Inglese (NCATS)
Co-Inventors
Patricia Dranchak (NCATS)
ICs
This technology includes a method for selecting a therapeutic effective amount of one of two compounds (including naltriben and naltrin) for the treatment of Zellweger Spectrum Disorder (ZSD), or any disease associated with peroxisome dysfunction. The compounds were identified using a cell-image based high-content screening (HCS) assay to identify small molecules that enhance peroxisome assembly in immortalized skin fibroblasts obtained from a ZSD patient.
Commercial Applications
Clinical work with the identified compounds may result in the establishment of new therapeutic agents effective for Zellweger Spectrum Disorder.
Competitive Advantages
Current treatment options are palliative in nature and there is a need to develop targeted therapies that address the peroxisome assembly defects responsible for disease development and progression.

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