Our Science
At Vesalic, we have discovered a systemic metabolic dysfunction that creates a toxic exosome cargo in ALS patients, which is carried to the CNS, where it binds to and damages neurons. We are now pioneering a therapeutic to target and neutralize these toxins before they can damage motor neurons. This approach may be able to slow or halt progression of both monogenic and sporadic forms of ALS.
Vesalic is now conducting in vivo studies to establish preclinical proof of concept for its therapeutic and to support a planned regulatory filing in 2028 to initiate clinical study.
- Detecting ALS quickly and accuratelyWe have also developed a biomarker-based technology with >90% accuracy in detecting both monogenic and sporadic forms of ALS, and which could be deployed in third-party clinical trials.
- Guiding future therapeutic strategiesWe have preliminary evidence that our biomarker test could be used to monitor how effectively a therapy is working in individual ALS patients. Thus, as treatments emerge, this test could play a critical role in guiding therapeutic strategies for ALS.
Leveraging our AI-based platform, the systemic extracellular vesicle lipidomic signature we identified in ALS has revealed convergent patterns of lipid dysregulation relevant to Alzheimer’s and Parkinson’s disease, supporting a shared biological framework across neurodegenerative diseases.
Our platform provides a powerful foundation for developing circulating biomarkers that capture systemic disease biology and may support future disease-specific stratification and therapeutic monitoring. Interested in partnering?
ALS patients experience progressive degeneration and loss of neurons in the brain, brainstem and spinal cord, which results in, the brain losing its ability to initiate and control muscle movement, leading to paralysis and eventually death.
ALS remains largely untreatable, particularly for patients with sporadic disease, despite extensive investigation over many years of approaches targeting abnormalities in the brain and CNS.
Approaches like Vesalic’s, which explore systemic pathogenic drivers, may help finally unlock desperately needed advancements for ALS patients, as well as people with other NDDs.
July 2026
Extracellular Vesicle Lipid Alterations Define Systemic Biomarkers Of Disease Biology In ALS, Alzheimer’s, And Parkinson’s Disease
- Alzheimer’s Association International Conference (AAIC), London, United Kingdom
June 2026
Redefining Neurodegenerative Disease: From Systemic Biology to Transformative Therapeutics
- XX Conference of Italian Researchers in the World (Virtual), Rome, Italy
May 2026
Neurodegeneration Through the Lens of Longevity: From Systemic Biology to Transformative Therapeutics
- Milan Longevity Summit
