Federal Project Grant Award Summary

Oregon Health & Science University (OHSU) received a $785,283 Project Grant award from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) under the Diabetes, Digestive, and Kidney Diseases Extramural Research program (CFDA 93.847) on June 4, 2026. This grant supports the development of adeno-associated virus (AAV) vector-mediated gene therapy for Cystinuria Type B, a rare genetic kidney disorder affecting approximately 1 in 7,000 births worldwide. The project will focus on addressing the root cause of this monogenic disease by developing a clinically applicable therapeutic approach to replace lost function of the SLC7A9 gene, which encodes a critical amino acid transporter in the kidney's proximal tubules.

The research aims to deliver an innovative gene therapy solution to replace current conservative treatments—including hyperhydration, urinary alkalinization, and dietary restrictions—that have limited efficacy and poor patient tolerance. Cystinuria Type B patients currently suffer from recurrent cystine stone formation, progressive kidney damage, and repeated surgical interventions. The project period extends from the award date through March 31, 2030, with the research being conducted at OHSU's Portland, Oregon facility. By developing this breakthrough therapy, the project seeks to provide patients with a potentially curative treatment that addresses the underlying genetic defect rather than managing symptoms.