RPE65

Retinal pigment epithelium 65 kDa protein

RPE65 (Retinal pigment epithelium 65 kDa protein) is a critical enzyme in the visual cycle that is specifically expressed in retinal pigment epithelial (RPE) cells. As a retinoid isomerohydrolase, RPE65 catalyzes the conversion of all-trans-retinyl esters to 11-cis-retinol, a vital process for visual phototransduction ^[1].RPE65 facilitates retinoid isomerization through its iron-binding site, maintaining the continuous supply of 11-cis-retinal in the retina - the essential precursor for rhodopsin regeneration ^[2]. Deficiencies in RPE65 function disrupt the visual cycle and are associated with various inherited retinal disorders, including Leber congenital amaurosis type 2 (LCA2) and retinitis pigmentosa ^[3].

References:

[1]. Gu SM, et al. Mutations in RPE65 cause autosomal recessive childhood-onset severe retinal dystrophy. Nat Genet. 1997 Oct;17(2):194-7. [Content Brief]

[2]. Moiseyev G, et al. RPE65 is the isomerohydrolase in the retinoid visual cycle. Proc Natl Acad Sci U S A. 2005 Aug 30;102(35):12413-8. [Content Brief]

[3]. Russell S, et al. Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial. Lancet. 2017 Aug 26;390(10097):849-860. [Content Brief]

RPE65 Inhibitors (2)

RPE65 Related Products (3):

Cat. No.	Product Name	Effect	Purity	Chemical Structure

HY-19720

Emixustat		98.00%
Emixustat is an orally active RPE65 inhibitor with an IC₅₀ value of 4.4 nM. Emixustat is also a visual cycle modulator, capable of regulating visual cycle activity by inhibiting retinol isomerization, and holds potential for studying vision disorders such as age-related macular degeneration (AMD).

HY-175663

EYE-002	Inhibitor
EYE-002 is a RPE65 inhibitor with an IC₅₀ of 69 nM. EYE-002 promotes recovery of scotopic ERG a-/b-wave amplitudes. EYE-002 regulates the visual cycle in mice by reducing 11-cis-retinal (11cRAL) (HY-116711) synthesis and increasing all-trans-retinyl esters (atREs). EYE-002 exerts protective effects against photic retinal damage in mice. EYE-002 can be used for the study of visual cycle-associated retinopathies, such as Stargardt disease type 1 (STGD1).

HY-175664

EYE-003	Inhibitor
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).

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