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SRT-055 is a first-in-class, brain/CNS-penetrant small molecule that acts as a dual inhibitor of leucine-rich repeat serine/threonine-protein kinase 2 (LRRK2) and apoptosis signal-regulating kinase 1 (ASK1). It is being developed by Seal Rock Therapeutics for the treatment of neurodegenerative diseases, primarily Parkinson’s disease and amyotrophic lateral sclerosis (ALS). LRRK2 is genetically linked to both familial and idiopathic forms of Parkinson’s disease, with excessive activity leading to lysosomal dysfunction and neuronal damage. ASK1 is activated by oxidative stress and contributes to neuronal apoptosis. By inhibiting both kinases, SRT-055 aims to provide neuroprotection through improved lysosomal function, reduced cell death, and decreased inflammation. Preclinical studies have shown potent neuroprotective effects superior to other LRRK2 inhibitors in models of Parkinson’s disease. The drug has not yet entered clinical trials but has received funding for preclinical ALS research[1][4][5][7][8].
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