EYE-003

• Description :

  EYE-003 is an orally active RPE65 inhibitor with an IC50 of 102 nM. EYE-003 regulates the visual cycle in mice by reducing 11-cis-retinal (11cRAL) (HY-116711) synthesis and increasing all-trans-retinyl esters (atREs) . EYE-003 suppresses scotopic ERG b-wave amplitude and exerts protective effects against retinal degeneration in Abca4⁻/⁻ Rdh8⁻/⁻ mice (a STGD1 model) by reducing retinal autofluorescent puncta and preserving outer nuclear layer (ONL) thickness in a dose-dependent manner. EYE-003 can be used for the study of visual cycle-associated retinopathies, such as Stargardt disease type 1 (STGD1) [1].

• UNSPSC :

  12352100

• Target :

  RPE65

• Related Pathways :

  Metabolic Enzyme/Protease

• Field of Research :

  Metabolic Disease

• Smiles :

  O=CO.CCCC(CCC)C(OC1=CC=CC(C(O)CCN)=C1)=O

• Molecular Formula :

  C18H29NO5

• Molecular Weight :

  339.43

• References & Citations :

  [1]Bassetto M, et al. Rationally Designed, Short-Acting RPE65 Inhibitors for Visual Cycle-Associated Retinopathies. J Med Chem. 2025 Aug 28;68 (16) :17638-17652.

• Shipping Conditions :

  Room temperature

• Scientific Category :

  Reference compound1

• Clinical Information :

  No Development Reported