PAV-615

PAV-615 is an investigational, small molecule tetrahydroisoquinolone (THIQ)-based assembly modulator developed by Prosetta Biosciences. It is designed to treat neurodegenerative disorders, specifically Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD), with a focus on cases associated with the C9orf72 G4C2 repeat expansion. The compound acts by targeting allosteric sites on aberrant multi-protein complexes within the host protein assembly machinery, aiming to restore these complexes to a healthy functional state. Preclinical studies in mouse models have demonstrated that PAV-615 can significantly improve motor performance, reduce anxiety-like behavior, and decrease pathological burdens including cortical dipeptide repeat proteins, phosphorylated TDP-43, and ataxin 2-positive stress granules.