RESEARCH PROGRAM

With our platform approach of reengineering human enzymes, we are designing solutions for a range of rare and high-burden diseases.

We believe there is a better way to pursue the drug development process, with a focus on purpose and continuous learning throughout the development cycle to reach patients faster with high-impact therapies. With expertise in rare genetic disease and enzymology, we thoughtfully select enzymes based on their metabolite targets and functionality, evaluate in highly translatable models and reduce risks by leveraging established engineering and manufacturing processes.

Each of our development programs is purposely selected because of its potential to transform the lives of patients and their families who have few options today.

Our most advanced research program looks to lower cystine levels in patients with Cystinuria, a serious metabolic disease that causes recurrent kidney stones and puts patients at increased risk of hypertension and chronic kidney failure. Our preclinical data show that treatment with an engineered human enzyme designed to reduce levels of cystine in the blood results in a reduction in kidney stone formation.

Advancing Multiple Development Programs