Developing therapies for specific subsets of Parkinson's patients may hold the key to bringing drugs through the clinic and to market.
Pittsburgh, PA and New York, NY (PRWEB) September 15, 2015
Sharp Edge Labs, Inc., a drug discovery biology firm (http://www.sharpedgelabs.com), today announced that they have been awarded a grant from The Michael J. Fox Foundation for Parkinson’s Research to apply their proprietary technology to a drug discovery effort for Parkinson’s disease. The grant covers the development of a high-throughput compound screening (HTS) assay for a protein trafficking defect related to Parkinson’s: mutation and misfolding of the enzyme glucosocerebrosidase.
Mutations in the gene coding for this enzyme have been strongly correlated with the onset of Parkinson’s disease. The most common mutations cause a “trafficking defect,” which prevents the protein from reaching its proper destination, a compartment inside cells called the lysosome. The defect makes patients up to 20 times more likely to develop Parkinson’s disease. Trafficking defects in this same enzyme are also the primary cause of Gaucher’s disease (a lysosomal storage disorder).
Using funds from the grant, Sharp Edge Labs has developed a new high throughput assay for glucosocerebrosidase trafficking that will expose molecular targets not previously probed in drug screens. The company will then use this assay to screen for potential new Parkinson’s drugs. This work will have impact for Parkinson’s and other neurodegenerative disorders including Alzheimer’s, as well as for lysosomal storage disorders such as Gaucher’s disease.
Scott Sneddon, PhD, JD, President and CEO of Sharp Edge Labs, noted that this was a unique opportunity to apply Sharp Edge Labs’ trafficking technology. “We’re excited to start the hunt for Parkinson’s drugs using this new technology,” Sneddon said. “These assays probe novel biology and expose molecular targets not previously exploited in drug discovery programs. By targeting this specific patient population within Parkinson’s we also believe that we’re setting ourselves up for clinical success in what has traditionally been a very difficult disease area.”
Prior to co-founding Sharp Edge Labs, Sneddon was a co-founder of the Drug Discovery program at Genzyme Corp., a company well known for developing drugs for lysosomal storage disorders. Before that, Dr. Sneddon was in the New Leads Discovery program at Pfizer Central Research in Groton, Connecticut.
About Sharp Edge Labs
Founded in 2010, Sharp Edge Labs, Inc. is a privately held company focused on the role of molecular trafficking in disease, bringing novel trafficking assay technology to drive drug discovery programs across a wide range of disease application. Sharp Edge Labs is the exclusive licensee of assay technology from Carnegie Mellon University, and is located in Pittsburgh Pennsylvania. For more information visit http://www.sharpedgelabs.com
About The Michael J. Fox Foundation for Parkinson’s Research
As the world’s largest nonprofit funder of Parkinson’s research, The Michael J. Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research program coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers. In addition to funding more than $450 million in research to date, the Foundation has fundamentally altered the trajectory of progress toward a cure. Operating at the hub of worldwide Parkinson’s research, the Foundation forges groundbreaking collaborations with industry leaders, academic scientists and government research funders; increases the flow of participants into Parkinson’s disease clinical trials with its online tool, Fox Trial Finder; promotes Parkinson’s awareness through high-profile advocacy, events and outreach; and coordinates the grassroots involvement of thousands of Team Fox members around the world.