Rare Genetic Diseases

Focusing on modulating the extremes of amino acid metabolism, we use novel engineered human enzymes to reduce amino acid toxicity in the blood. For patients with rare genetic diseases (like Arginase 1 Deficiency) who currently have limited treatment options, we believe in a better future through enzyme therapy.

Discover Our Therapeutic Human Enzymes

Pegzilarginase

Arginase 1 Deficiency

ACN00177

Homocystinuria

AEB5100 Program

Cystinuria

RESEARCH PROGRAMS

Undisclosed Rare Diseases

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