SEL-145,793

SEL-145,793 is an orally available, CNS-penetrant small molecule being developed by Sharp Therapeutics for the treatment of Niemann-Pick disease type C (NPC). Discovered using the company's proprietary Disco™ platform, the compound acts as an allosteric activator of the mutated Niemann-Pick C1 (NPC1) protein, restoring its function to facilitate the transport of cholesterol out of late endosomes and lysosomes. Preclinical studies in NPC patient-derived fibroblasts have demonstrated that SEL-145,793 significantly reduces intracellular accumulation of free cholesterol, increases lysosomal exocytosis, and reverses disease-associated hyperactivation of the mTOR signaling pathway.