Retinal pigment epithelium 65 (RPE65) also known as retinoid isomerohydrolase is a 65 kDa enzyme located within the human retinal pigment epithelium cells responsible for the chemical conversion of all-trans-retinyl ester to 11-cis-retinol in the visual cycle.

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Function

Structural Features of RPE65

Enzymatic Catalyzed Reaction

Protein-Ligand Interaction

Endogenous Ligands

Exogenous Ligands

Emixustat (ACU-4429)

Emixustat (ACU-4429) is an investigational small molecule inhibitor of RPE65 first invented by a British-American chemist, Ian L. Scott. In 2008, Acucela Inc. partnered with Otsuka Pharmaceutical Company for the development of emixustat as a potential inhibiter of RPE65. Currently emixustat is in Phase III clinical trials in the United States for the potential treatment of Stargard's disease, a juvenile form of atrophic (dry) age dependent macular degeneration (AMD).

Formulated as an hydrochloride salt, emixustat hydrochloride is taken by mouth and is known as a visual cycle modulator (VCM).

Mutations

Disease Implications

Medical Relevance

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