OCT-980

OCT-980 is an oral small-molecule pharmacological chaperone (corrector) designed to treat rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP). Developed by Octant Biotech, the drug targets the rhodopsin (RHO) protein, which is frequently misfolded due to genetic mutations in patients with RHO-adRP. OCT-980 binds to these misfolded proteins to facilitate their proper folding and trafficking to the rod cell surface, thereby preventing the toxic accumulation of protein aggregates that leads to photoreceptor cell death. In preclinical models, OCT-980 has demonstrated the ability to restore retinal structure and improve low-light vision. It is currently undergoing IND-enabling studies.