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BUK-001 is an early-stage small molecule drug candidate being developed by Bukwang Pharmaceuticals for the treatment of Parkinson's disease. Originally licensed from Gilead Sciences, the compound acts as an inhibitor of Leucine-rich repeat kinase 2 (LRRK2). LRRK2 is a large multidomain protein with kinase and GTPase activities; mutations in the LRRK2 gene, such as the G2019S mutation, are among the most common genetic risk factors for both familial and late-onset sporadic Parkinson's disease. These mutations typically result in increased kinase activity, which is linked to lysosomal dysfunction, neuroinflammation, and the pathological aggregation of alpha-synuclein. By targeting and reducing LRRK2 kinase activity, BUK-001 aims to provide a disease-modifying effect that could potentially slow or halt the neurodegenerative process in Parkinson's patients. The asset is currently in the preclinical stage of development.
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