Unlocking the Potential of Engineered Human Enzymes

Engineering human enzymes to modulate the extremes of amino acid metabolism in rare genetic diseases and cancer is the cornerstone of our work. Using these innovations to improve lives and address unmet needs in serious human disease is our passion.

As a leader in this field, we’re developing treatments with the potential to reduce toxic levels of certain amino acids in the blood, such as arginine for the treatment of Arginase I deficiency, and to selectively target and deprive tumors of amino acids they depend on for survival.

Discover with us.…

Rare Genetic Diseases

Enzyme Replacement Therapy for Arginase 1 Deficiency

Hyperarginaemia, or excessively high levels of arginine in the blood, is due to a rare, hereditary, inborn error of metabolism causing patients to be deficient in the enzyme Arginase I. Also known as Arginase I deficiency, 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 diet 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 currently conducting clinical trials using an engineered human enzyme Arginase I to serve as an enzyme replacement therapy in patients with Arginase I deficiency.

We’re testing the ability of our lead product candidate (AEB1102) to return elevated blood arginine levels to normal, getting to the underlying cause of this disease, with potential clinical benefits for patients.

Cancer

Oncology, Hematology

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 alleviate this unmet medical need, scientists at Aeglea are unlocking the potential of engineered human enzymes to provide targeted cancer therapy to fight advanced solid tumors and hematological malignancies (cancers that begin in the blood).

Our enzymes take advantage of certain cancers’ dependence on specific amino acids for survival. By depriving these cancers of this critical fuel, 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. Phase 1 Clinical Trials are underway.