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.


We’re unlocking the potential of enzyme therapy to deprive certain cancers of the key amino acids they depend on. It’s a well described but not yet broadly applied concept with the potential to transform lives for a variety of diseases associated with abnormal amino acid metabolism.

Discover Our Therapeutic Human Enzymes

 Pegzilarginase (AEB1102)

Arginase 1 Deficiency
Cutaneous Melanoma, Uveal Melanoma, SCLC - Single Agent Expansion
SCLC - Combination with Checkpoint Inhibitor


Homocystinuria: Homocystine


Cystinuria: Cystine


Cancer Metabolism: Cysteine / Cystine


Cancer Metabolism: Methionine


Undisclosed Rare Diseases

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Rare Genetic Disease Clinical Trials

Patients, families, and healthcare providers play a crucial role in helping us to make our prospective therapies available to those who need them. Learn about current clinical trials in rare genetic diseases such as inborn errors of metabolism.

Cancer Clinical Trials

Engineered human enzymes for targeted cancer therapy have great potential and clinical studies are underway. Thank you for considering getting involved. Find out more about our enzyme therapy cancer clinical trials.

Inspiring Careers

Talented, dedicated team members are in good company at Aeglea. If you’re passionate about using your experience and expertise to help others – join us.