Engineered Cyst(e)inase for Cystinuria Treatment
An enzyme that reduces plasma and urine cystine to prevent the formation of cystine stones for treatment of the rare disease cystinuria
Background
Cystinuria is a pediatric onset genetic disorder characterized by excessive amounts of cystine in urine that leads to recurrent stone formation. Approximately 70% of cystinuria patients develop chronic kidney disease that progresses to end-stage renal disease in about 5% of patients. The current standard of care for cystinuria is the prescription of adequate fluids, low sodium and protein diet, urinary alkalinization and cystine-binding thiol medications. These thiol medications provide modest efficacy in halting new stone formation; however, patients commonly discontinue treatment due to side effects and still require surgical removal of kidney stones, with the average cystinuria patient undergoing seven stone removal surgeries by 45-65 years. Researchers at the University of Texas at Austin have developed a novel enzyme that degrades cystine, directly removing it from circulation and reducing the need for surgical stone removal.
Technology Overview
This technology is an engineered enzyme that degrades cystine to remove it from circulation. This enzyme exhibits good efficacy in mouse cystinuria model; treatment with the enzyme provides a rapid reduction in plasma and urine cystine/cysteine and a concomitant reduction of cystine kidney stones and urine crystals. The cystine/cysteine degrading enzyme is anticipated to provide the same therapeutic benefit in humans.
Benefits
- Improves symptoms in a mouse cystinuria model
- Well tolerated
- Enzyme has been significantly de‑risked and is being further developed by UT Austin
Applications
- Treatment of the rare disease cystinuria
Opportunity
Cystinuria affects approximately 40,000 people in the United States, and the available treatment options are largely ineffective leading to high unmet need. Approximately 25% of cystinuria patients discontinue treatment with small molecule thiol binders due to side effects. This enzymatic therapeutic treatment represents a significant improvement over existing treatments. This therapeutic is eligible for Orphan Product designation. This technology is currently being developed by the UT Austin Life Science Technology Development Program with the goal of preparing a data package ready for an Investigational New Drug application by an appropriate sponsor.
UT Austin is seeking partners to participate in development of this program.