Unlocking the Potential of Next Generation Human Enzymes

Engineering human enzymes to provide solutions for diseases with high unmet medical need is the cornerstone of our work.
Using these innovations to improve the lives of patients is our passion.

Discover with us.…

Rare Genetic Diseases

Enzyme Therapy for Arginase 1 Deficiency

Arginase 1 Deficiency, sometimes called hyperargininemia, leads to excessively high levels of arginine in the blood. It is due to a rare, hereditary, inborn error of metabolism causing patients to be deficient in the enzyme Arginase 1. This devastating, life-threatening urea cycle disorder presents in early childhood with a range of neurological symptoms significantly affecting the child and, in turn, their family.

Aside from severe dietary protein restriction, there is no treatment that addresses the root cause of the disease to help prevent the progressive spasticity, seizures, impaired growth, intellectual disability, and other serious symptoms of this condition.

We’re testing the ability of our lead product candidate, pegzilarginase, to lower blood arginine levels and address the underlying cause of this devastating disease.