Federal Grant Award Summary

The University of California, San Francisco received a $736,509 Project Grant from the National Institute of Diabetes and Digestive and Kidney Diseases under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847), awarded June 24, 2026, with completion targeted for March 31, 2030. This international collaborative research initiative, structured as a multi-PI (Principal Investigator) grant mechanism involving researchers from UCSF, University of Ulster, and Dublin City University, delivers novel translational research on nephropathic cystinosis (NC), a rare genetic kidney disorder characterized by cysteine accumulation in lysosomes. The grant funds preclinical and mechanistic research investigating the previously unexplored interaction between the CTNS and ATP6V0A1 genes to identify rational drug design opportunities for mitigating kidney injury in NC patients who experience progressive renal failure despite current cysteine depletion therapy.

The research deliverables encompass multi-modal imaging studies, pH and mechanistic analyses of disease-relevant mutations in their endogenous context, and development of innovative therapeutic approaches targeting ATP6V0A1 to address the underlying causes of kidney damage in nephropathic cystinosis. The collaborative team leverages combined expertise in translational nephrology, molecular medicine, cellular imaging, preclinical disease modeling, and drug development to advance beyond current symptomatic management toward disease-modifying treatments for this pediatric kidney disorder.