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SBT02 is a novel, brain-penetrant recombinant biological analogue of human Small Ubiquitin-like Modifier 2 (SUMO2) being developed as a disease-modifying therapy for neurodegenerative disorders, including Alzheimer's disease, Parkinson's disease, and multiple system atrophy. Developed through a collaborative effort by researchers at the University of Toronto, the Mario Negri Institute for Pharmacological Research, and Columbia University, SBT02 mimics the neuroprotective activity of endogenous SUMO2. It functions by facilitating the SUMOylation of pathological proteins such as microtubule-associated protein tau and alpha-synuclein, thereby maintaining them in a soluble state, preventing their aggregation, and preserving synaptic integrity. In preclinical models of amyloidosis, tauopathy, and synucleinopathy, systemic (subcutaneous) administration of SBT02 has demonstrated the ability to prevent and reverse synaptic dysfunction, cognitive decline, and motor deficits without altering amyloid-beta plaque burden.
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