Science
MitoChem’s research focuses on restoring mitochondrial function, a key early failure point in neurodegenerative diseases. We have developed a library of mitochondrial protective small molecules. Our lead program, MC16, directly targets the RNA-binding protein Fused in Sarcoma (FUS), correcting its mislocalization between the nucleus and cytoplasm - a root cause of neuronal energy loss and degeneration.
Mitochondria are the cell’s power grid. Their dysfunction is one of the earliest hallmarks of ALS, FTD, and other neurodegenerative diseases.
FUS regulates transcription, RNA metabolism, and DNA repair. Chronic stress or mutation causes FUS to mislocalize to the cytoplasm, where it disrupts mitochondrial metabolism.
MC16 reverses both loss of function in the nucleus and gain of toxic function in the cytoplasm, restoring mitochondrial balance and enabling neuron survival.