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What We Do

Traditional pharmaceutical approaches focus on one biological target or gene. At Axcella, we are taking the next evolutionary step in disease treatment through a multi-targeted approach. We view disease as deviation from a balanced state, impacting not just the function of one biological pathway but rather an entire system of pathways throughout the body. By addressing the multiple dimensions of disease, we can achieve a systems-wide therapeutic effect and fully treat the multi-causal nature of disease.

Furthermore, we recognize amino acids as more than just building blocks of proteins. In fact, amino acid homeostasis (balance) plays a central regulatory role in cell health. More than 2,000 diseases are linked to a dysregulation of amino acid homeostasis. Our technology capitalizes on orally digestible, recombinant proteins, peptides, amino acids and metabolites to release amino acid signatures of interest into the blood stream.

Our approach—the application of systems pharmacology to treat the multiple causes of disease and prevent disease onset—represents a paradigm shift to the current standard of drug treatment. Systems pharmacology is based on the intersection of comprehensive diagnostic approaches (like omics, genotyping, and imaging) and pathophysiological-based disease treatment and prevention.

In multiple clinical studies our candidates have demonstrated a statistically significant impact on the restoration of metabolic functions measured by well-established biomarkers of health. We are currently focused on developing therapeutics for metabolic diseases (including liver and muscle) and neurological diseases, but we intend to meet the opportunity to treat a wide range of diseases through private development and partnerships.

We also leverage our understanding of the critical role of amino acids in cell health through our subsidiary: Acora. Our subsidiary will forge a new frontier for nutritional products in healthcare. All of our modalities are evaluated for their nutritional benefits and potential as nutritional products.