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Biotech, Pharma Partner on Neuro Drug Discovery

Brain wiring illustration

Brain wiring illustration (Courtesy, Human Connectome Project and NIH)

3 January 2017. A collaboration between a biotechnology enterprise tracking defective proteins and pharmaceutical company plans to discover new treatments for neurodegenerative disorders. The partnership between Sharp Edge Labs Inc. in Pittsburgh and Sumitomo Dainippon Pharma in Osaka, Japan involves sharing scientific resources between the companies and screening for new drug candidates, but financial and intellectual property aspects of the agreement were not disclosed.

Sharp Edge Labs discovers and develops therapies for diseases that result from defects in protein trafficking, movements of proteins to surfaces of cells and their subsequent internalization by cells, resulting from genetic mutations. The company’s technology uses biosensors called fluorogenic activating modules, made with fragments of antibodies in short chains of amino acids that bind and activate fluorescence in specially designed dyes. Once detected and tracked, properties of these defects can be quantified and analyzed as potential therapy targets, and screened against drug candidates.

Sharp Edge Labs licensed the technology patent from Carnegie Mellon University, with two of the inventors — Marcel Bruchez and Alan Waggoner — becoming the company’s scientific founders. The company is developing drugs for cystic fibrosis, rare diseases from lysomal storage disorders such as Gaucher’s and Tay-Sachs disease, and Parkinson’s disease, associated with glucocerebrosidase or GBA mutations also linked to Gaucher’s disease.

The company is harnessing neurons created with stem cells from Parkinson’s disease patients with GBA mutations to validate compounds discovered through its screening process using fluorogenic activating modules. One advantage of its technology, says Sharp Edge Labs, is the ability to identify compounds that cross the blood-brain barrier, which enzyme replacement therapies, the current treatments for lysomal storage disorders, cannot.

The agreement calls for Sumitomo Dainippon Pharma to make its compound library available for screening by Sharp Edge Labs to discover lead candidates for protein trafficking defects associated with neurodegenerative disorders. A key objective of the screening exercise is to identify small molecule, or low molecular weight, compounds with the ability to penetrate the blood-brain barrier. Sharp Edge Labs is also expected to describe the cellular mechanisms of lead compound candidates, from assessments based on activity in cells from actual patients.

Sumitomo Dainippon Pharma says one of its corporate goals is to enhance its fundamental drug discovery capabilities by making use of advanced state-of-the-art technologies. The company designated neurology and psychiatry, along with cancer, as key therapeutic areas for new pharmaceutical products.

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