MC16

MC16 is a first-in-class small molecule developed by MitoChem Therapeutics that targets the RNA-binding protein Fused in Sarcoma (FUS). It is designed to correct the mislocalization of FUS between the nucleus and cytoplasm, a pathological feature associated with neuronal energy loss and degeneration. By restoring FUS localization, MC16 up-regulates pro-survival and anti-oxidant pathways to maintain mitochondrial homeostasis and address mitochondrial dysfunction. The drug is currently in preclinical development for retinitis pigmentosa (RP), for which it received FDA Orphan Drug Designation in 2021, and is being investigated for Amyotrophic Lateral Sclerosis (ALS) and other neurodegenerative and metabolic disorders.