A validated molecule.
A new approach to neurological diseases.

Ageronix is developing Alpha-1 Antitrypsin (AAT)-based therapies for rare neurological disorders with high unmet medical need. The company's lead program focuses on Charcot–Marie–Tooth disease (CMT), one of the most common inherited neuromuscular disorders and a condition for which no approved disease-modifying treatment currently exists.
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The foundation of the program is Alpha-1 Antitrypsin, a clinically established molecule with a well-characterized safety profile and broad anti-inflammatory and immunomodulatory properties. Ageronix recognized the opportunity to apply these biological mechanisms to neurodegenerative processes underlying CMT and initiated a focused translational development program.
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Preclinical studies in a transgenic CMT1A mouse model, published in the International Journal of Molecular Sciences in 2022, demonstrated that AAT slowed disease progression and improved key measures of peripheral nerve health. These findings established the scientific rationale for further development and supported subsequent regulatory recognition by the FDA through Orphan Drug Designation and Rare Pediatric Disease Designation in 2023.
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Based in Geneva, Switzerland, Ageronix is advancing its lead CMT program toward clinical development while expanding its AAT platform to Alzheimer's disease and other neurodegenerative indications.