Drug pipeline
Full profile accessExplore the programs pursuing this target and their development progress.
- Drug candidates
- Developers
- Development stage
Target intelligence / Profile preview
Retinoid Isomerohydrolase (RPE65)
- Target
- RPE65
- Molecular classification
- Enzyme, Carotenoid cleavage oxygenase
01
Overview
Retinoid isomerohydrolase (RPE65) is a critical enzyme in the vertebrate visual cycle, primarily expressed in the retinal pigment epithelium (RPE). It catalyzes the conversion of all-trans-retinyl esters to 11-cis-retinol, a vital step in regenerating visual pigments necessary for vision. Mutations in RPE65 cause severe inherited retinal diseases like Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa.
- Other names
- Retinol isomeraseRetinoid isomerase
02
Mechanism of action
Catalyzes the conversion of all-trans-retinyl esters to 11-cis-retinol, essential for regenerating visual pigments.
03
Biological functions
Retinoid isomerizationVisual cyclePhototransduction
04
Disease associations
Leber Congenital Amaurosis (LCA)Retinitis Pigmentosa Type 20Fundus Albipunctatus
05
Safety considerations
Loss-of-function mutations lead to severe visual impairment and retinal degeneration.
Beyond the preview
Go deeper on Retinoid Isomerohydrolase (RPE65).
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Clinical trials
Full profile accessFollow the clinical studies evaluating therapies directed at this target.
- Trial design
- Status
- Readouts
Competitive landscape
Full profile accessCompare approaches across drug candidates, modalities, and indications.
- Programs
- Modalities
- Indications
Literature & evidence
Full profile accessInvestigate the research and source evidence behind target biology and development.
- Publications
- Sources
- Analysis
Patents
Full profile accessExplore patent activity around therapies and technologies addressing this target.
- Patents
- Assignees
- Technologies
Research & analysis
Full profile accessConnect target biology, drug development, and emerging evidence in your research.
- Biology
- Development news
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on Retinoid Isomerohydrolase (RPE65).