Developing Alpha-1 Antitrypsin-based therapeutics with programs spanning rare neurological disorders and Alzheimer's disease.
CMT is our lead development program. Building on our Alpha-1 Antitrypsin platform, we are advancing additional programs in Alzheimer's disease and broader neurodegeneration.
FDA-
Recognized
Experienced
Research Team
Commitment
to CMT Patients
Pioneering
AAT Therapies
Leading the
search for a cure
A progressive
genetic disorder
Charcot–Marie–Tooth (CMT) disease is a hereditary neuropathy that affects the peripheral nervous system. The most common form, CMT1A, results from a duplication of the PMP22 gene, causing damage to the myelin sheath that insulates nerves. Progressive nerve degeneration leads to muscle weakness, sensory deficits, and mobility impairment.
Alpha-1 Antitrypsin offers
new therapeutic hope
Alpha-1 Antitrypsin (AAT) is a naturally occurring protein with powerful anti-inflammatory properties. It is now emerging as a promising therapeutic approach for CMT disease. Developed by Ageronix, our AAT program has received key support from the U.S. Food and Drug Administration (FDA). Preclinical studies suggest that AAT may reduce neuroinflammation, protect nerve function, and slow disease progression. While clinical validation is still required, these findings provide a strong foundation for the development of a novel treatment for patients with CMT.
Our AAT therapy is evolving across three generations of innovation.
Together, they mark a shift toward more precise and effective treatments for rare diseases.
01.
Plasma-derived
02.
Recombinant
03.
Gene therapy
First Generation: Plasma-derived AAT established a strong clinical foundation, demonstrating an excellent safety profile and therapeutic potential
Second Generation: Recombinant AAT unlocked scalable production and expanded the possibilities for broader clinical application.
Third Generation: Ageronix is pioneering novel AAT-based therapies designed to precisely target inflammation and modify disease progression.
01.
plasma derived
02.
recombinant
03.
Gene THERAPY
Ageronix was granted the Orphan Drug Designation (ODD)
and the Rare Pediatric Disease (RPD) Designation by the FDA.
PRECLINICAL
VALIDATION
2023
FDA
ORPHAN DRUG
DESIGNATION
July 2023
FDA RARE
PEDIATRIC DISEASE
DESIGNATION
August 2023
TRANSLATIONAL
DEVELOPMENT
AND PIPELINE
EXPANSION
Since 2024
CLINICAL
DEVELOPMENT
PATHWAY
Upcoming
Orphan Drug
Designation
The FDA's Orphan Drug Designation (ODD) recognizes the potential of Ageronix's therapeutic approach to address a significant unmet need in rare disease. This important milestone validates the scientific foundation of our program and supports the advancement of innovative treatments for patients with limited therapeutic options.
Rare Pediatric
Disease
The Rare Pediatric Disease (RPD) designation further reinforces the momentum of our development program, recognizing its potential relevance for pediatric patients. It reflects growing confidence in the impact of our therapeutic approach and strengthens the role of Ageronix in advancing the next generation of treatments for rare diseases.
For more information
please get in touch at:
info@ageronix.com
Avenue de la Roseraie 64
1205, Geneva - Switzerland
+41 223 79 46 43
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