SKL32276

SKL32276 is a small molecule drug candidate currently in preclinical development by SK Biopharmaceuticals for the treatment of Parkinson's disease. It targets glucosylceramidase (GCase), a lysosomal enzyme responsible for the hydrolysis of glucosylceramide. Mutations in the GBA1 gene, which encodes GCase, are the most significant genetic risk factor for Parkinson's disease, as reduced enzyme activity leads to lysosomal dysfunction and the accumulation of toxic alpha-synuclein aggregates. SKL32276 is designed to modulate GCase activity, thereby restoring lysosomal proteostasis and promoting the clearance of pathogenic protein aggregates to slow or prevent disease progression.