Vesalic is a biotechnology company developing therapeutics and diagnostics for ALS and other neurodegenerative diseases by targeting systemic metabolic dysfunction rather than only the central nervous system. Its lead investigational therapeutic addresses a newly characterized toxic exosome cargo mechanism, while its blood-based biomarker technology detects disease-specific lipid alterations in circulating exosomes. The platform is designed for both monogenic and sporadic ALS cases.
Funding
Funding not disclosed
Founders
Product
Problem
ALS remains a devastating neurodegenerative disease with no definitive non-invasive diagnostic method, especially in early stages. The root cause is understood in only 10% of patients with monogenic disease, while the other 90% of sporadic cases have seen little therapeutic progress. Most research has focused on the brain and central nervous system, where damage is observed, but this approach has yielded limited success.
Solution
Vesalic is pioneering a systemic biology approach to ALS and other neurodegenerative diseases, based on the discovery that a previously unknown systemic metabolic dysfunction creates toxic exosome cargo in ALS patients. This toxic cargo travels to the central nervous system, where it binds to and damages neurons, representing a novel druggable target with disease-modifying potential. The company is developing an investigational therapeutic that intercepts this root cause, applicable to both monogenic and sporadic ALS. Additionally, Vesalic has identified disease-specific alterations in the lipid composition of exosome membranes circulating in the blood, enabling a novel blood-based biomarker for quicker, more accurate ALS detection. This dual therapeutic and diagnostic platform extends beyond ALS to potentially address Alzheimer's and Parkinson's diseases through the same systemic framework.
Target Audience
Primary stakeholders are researchers and clinicians in neurodegenerative disease, particularly those focused on ALS, Alzheimer's, and Parkinson's, as well as pharmaceutical partners interested in systemic approaches to NDD therapeutics and diagnostics.
Features
- Novel therapeutic candidate targeting systemic metabolic dysregulation and toxic exosome cargo that damages CNS neurons
- Blood-based biomarker technology detecting disease-specific lipid alterations in circulating exosome membranes
- Platform applicable to both monogenic and sporadic ALS patient populations
- Discovery framework extends to Alzheimer's and Parkinson's disease pathogenesis through lysosomal and mitochondrial dysfunction
- Non-invasive diagnostic approach designed for early disease detection
- Research presented at international conferences including AAIC and Milan Longevity Summit