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 Replacement 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.


A cancer diagnosis is a life-changing, potentially devastating event. While there are many cancer treatments available, existing treatments don’t fit every situation and many patients are still in need of effective treatment options.

To attempt to address this unmet medical need, scientists at Aeglea are unlocking the potential of engineered human enzymes to provide targeted therapy to fight cancer.

Our enzymes take advantage of certain cancers’ dependence on specific amino acids for survival. By depriving these cancers of this critical nutrient, we see the potential for a novel treatment that may deliver clinical benefits for patients.

Using engineered human enzymes, we aim to deplete amino acid concentrations in blood to levels that tumors can’t tolerate – but normal tissues can. Going hand-in-hand with this work is the development of diagnostic tools to identify cancer patients who will benefit most from amino acid metabolism therapy.