Development of a Phenotypic High-Content Assay to Identify Pharmacoperone Drugs for the Treatment of Primary Hyperoxaluria Type 1 by High-Throughput Screening

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Michael Conn
Dr. P. Michael Conn is the Sr. Vice President for Research, Associate Provost and Professor of Internal Medicine and Cell Biology.
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Cameron Smith
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Provisional Patent Application Filed

Description:

Hyperoxalosis is a deadly disease for which the best current therapy is dialysis or organ transplant.  The underlying cause of some forms of the disease is the misrouting of a specific enzyme; alanine:glyoxylate aminotransferase (AGT) to the mitochondira inststead of the peroxisome.  Pharmacoperones are small molecules that can restore proper function to the peroxisome to restore organ function and potentially cure the disease.

The discovery of target-specific pharmacoperones by high throughput screening (HTS) requires the design of automation-friendly, microtiter plate-compatible assays. Previously reported assays measuring AGT activity are not fully compatible with HTS requirements or are effectively too far removed from the phenotypic and physiological relevance of this target.  This invention describes a phenotypic, microscopy-based assay that detects the co-localization of AGT with the peroxisomes in a mammalian cell based system expressing a pathophysiologically-relevant mislocated mutant form of AGT; AGT-170. This assay has been successfully miniaturized to a 384-well plate format.  This assay is HTS ready.

Reference Number: D-1154

Market Applications:

  • Pharmaceuticals
  • Biotechnology

Features, Benefits & Advantages:

  • Assay to determine AGT activity
  • Assay to determine possible drugs for hyperoxaluria Type 1

Intellectual Property:

  • A U.S. Provisional Patent application Serial 62/103,889 was filed on 1/15/15, and a PCT application, PCT/US16/13610 was filed 01/15/16.

Development Stage:

  • This invention has been reduced to practice, tested and proved ready for HTS applications.

Researcher:

  • Michael Conn, Ph. D., Professor and Vice President of Research, Texas Tech University Health Sciences Center, Lubbock, Texas.

Keywords: pharmacoperones, chemical chaperones, hyperoxaluria Type 1, phenotypic assay