Oral rhodopsin correction in adRP: Ester Carreño, MD, PhD, weighs in

Rhodopsin-associated autosomal dominant retinitis pigmentosa (RHO-adRP) is a progressive inherited retinal disease with no approved treatment. In October 2026, the US Food and Drug Administration (FDA) granted orphan drug designation to OCT-980 (Octant), an investigational oral small-molecule corrector designed to correct misfolded rhodopsin and restore its trafficking within photoreceptors. OCT-980 is being evaluated in a phase 1/2 trial (NCT07408232). The phase 1a portion, a single ascending dose study in healthy volunteers in Australia, is complete. The open-label, multicentre phase 1b/2 multiple ascending dose study is enrolling adults aged 18 to 65 years with genetically confirmed RHO-adRP at US sites, who will take oral tablets for up to 48 weeks.