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.