By Nuala Moran, Europe Editor
Originally published February 17, 2026, in BioWorld

Newco Vesalic Ltd. has formed to take forward research indicating extracellular vesicles secreted by skeletal muscle cells carry toxic payloads that are key drivers of motor neuron diseases, including amyotrophic lateral sclerosis (ALS).

The discovery of this process, which is largely external to the brain and the central nervous system, has opened up new targeting possibilities, and Vesalic is now working on in vivo studies to demonstrate preclinical proof of concept.

It also has been shown that the toxin-carrying exosomes extracted from blood samples of ALS patients have a distinctive lipid composition in their membranes and, based on this observation, Vesalic has developed a diagnostic biomarker.

The biomarker has shown more than 90% accuracy in detecting both the 90% of ALS cases that are sporadic and the 10% that are monogenic. The London-based company said it believes this biomarker has the potential to detect ALS before it becomes symptomatic, and could avoid the need for the battery of tests, including MRI scans, tests of nerve conduction and nerve and muscle electrical activity, and lumber punctures, that currently are required to confirm a diagnosis of ALS.

Numerous cell and tissue types, including skeletal muscle, secrete exosomes that carry functional proteins, mRNA, miRNA and lipids that are involved in cell-to-cell communication in the central nervous system (CNS).

Valeria Ricotti, CEO and
co-founder, Vesalic

There is mounting evidence implicating exosomes in spreading diseases that are characterized by misfolded proteins, and exosomes from the CNS of ALS patients have been shown to carry misfolded proteins, including SOD1, FUS and TDP-43, that can then be ferried into healthy neighboring neurons and glial cells.

The observation of toxic vesicles in muscle cell cultures from ALS patients was “very serendipitous,” said Valeria Ricotti, CEO and co-founder of Vesalic. “We took this observation and we fully upgraded the methodology, we fully upgraded the way of measuring the signature, so now it’s totally quantifiable … and it’s quantifiable from a very small blood sample. We analyze the content of these exosomes in a very precise way,” she told BioWorld.

“What we have observed is that in these exosomes, there is a cargo that is toxic to motor neurons. We have identified a druggable target, and by using a biologic, we can intercept that toxic cargo,” Ricotti said.

“The major difference, if this approach works, is that first of all, it’s for all forms of ALS, not just for monogenic conditions, which are the minority … and that we are targeting a systemic issue that should significantly slow down the progression of the disease, and at the root cause of the disease, rather than intervening when the avalanche of pathology has already happened.”

Neurotoxic vesicles

For now, no further details are being disclosed. However, two of the scientists involved in Vesalic, director Julie Dumonceaux and advisor Virginie Mariot, who are both research fellows at the National Institute of Health Research at University College London, are co-authors of a paper published in 2022 describing the discovery that muscle cells of sporadic ALS patients secrete neurotoxic vesicles.

They showed that vesicles from ALS muscle stem cells induced shortened, less branched neurites, promoted cell death and disrupted the localization of RNA and RNA-processing proteins in motor neuron cells derived from induced pluripotent stem cells from healthy donors.

Many of the peptides and proteins that were observed at higher levels in vesicles from ALS patients compared to controls are known protein-binding proteins and the researchers suggest the disrupted RNA processing, leads to RNA accumulation in the nucleus, and cell death.

The toxic vesicles also showed increased expression of CD63, a biomarker found on the surface membrane of exosomes. When CD63 levels were reduced by incubating the vesicles with an anti-CD63 monoclonal antibody, the level of motor neuron cell death in culture dramatically decreased.

Vesalic was formed in 2023 and is now showing its face as it completes preclinical proof of concept and works toward filing an IND in mid-2027. The preclinical research is revolving around an animal model of sporadic ALS the company has developed and patented.

To date, Vesalic has been funded by individual investors. It is now in the middle of raising more money, but is not yet looking for formal VC funding. “So far, we have just reached out to high net worth individuals, and we’re probably going to remain in the realm of angels or family offices,” said Ricotti.

The diagnostic biomarker has been assessed by third party academic groups. Although not regulatory approved, it could be used to monitor treatment response in clinical trials.

Separately from advancing its lead program to the clinic, the company is working with advisors on a strategy for validating and commercializing the diagnostic. The underlying principle also would be relevant to developing blood biomarker diagnostics for other neurodegenerative diseases, Ricotti said.

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