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emixustat (formerly ACU-4429)

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Company Kubota Pharmaceutical Holdings Co. Ltd.
DescriptionNon-retinoid visual cycle isomerase inhibitor targeting retinal pigment epithelium-specific 65 kDa protein (RPE65)
Molecular Target Retinal pigment epithelium-specific protein 65kDa (RPE65)
Mechanism of Action 
Therapeutic ModalitySmall molecule

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Disease CategoryStandard IndicationIndication DetailsPartnersMilestonesLatest Stage of DevelopmentRegulatory Designation
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