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OCNT-980

Development stage: Preclinical
Lead developer: Octant
Modality: Small Molecules
Administration: Oral

Overview

OCNT-980 is a small molecule drug candidate developed by Octant for the treatment of RHO-associated autosomal dominant retinitis pigmentosa (RHO-adRP), an inherited retinal disease with no current disease-modifying therapies. The compound was discovered and optimized using deep mutational scanning and generative chemistry approaches, enabling the design of potent, stable, orally available correctors that cross the blood-retinal barrier. OCNT-980 acts by mitigating toxic accumulation of misfolded rhodopsin in the retina, thereby addressing cellular dysfunction caused by RHO mutations[4].

Targets

RHO (Rhodopsin)

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