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Medicinal chemistry professor awarded NIH grant to develop a new therapy for cystinuria

Longqin Hu, Professor in the Department of Medicinal Chemistry, Ernest Mario School of Pharmacy, and Amrik Sahota, Research Professor in the Department of Genetics, School of Arts and Sciences, are the two principal investigators of a new five-year multi-PI R01 grant, totaling $2,878,846, to develop "L-Cystine Diamides as Inhibitors of L-Cystine Stone Formation in Cystinuria". Cystinuria is a rare genetic disorder that is characterized by high concentrations of the poorly soluble amino acid cystine in the urine. It is a chronic, lifelong condition and affected patients have a >50% chance of stone formation in the kidneys or the bladder during their lifetime. Currently, there is no effective therapy available for cystinuria. The goal of this research project is to develop an effective small molecule drug that can be used to prevent recurrent stone formation in patients with cystinuria. This project is supported by grant R01 DK112782-01 starting on March 1, 2017 through the National Institute of Diabetes and Digestive and Kidney Diseases.