RPE65 Overview
Molecule Name
Retinoid isomerohydrolase
Synonyms
BCO3, LCA2, RP20, mRPE65, p63, rd12, sRPE65
Drug Development Progress
Drugs in Clinical Trials5
Approved Drugs1
Highest Development StageApproved
Target Synonym
EC:5.3.3.22，Retinoid isomerohydrolase，All-trans-retinyl-palmitate hydrolase，Retinal pigment epithelium-specific 65 kDa protein，RPE65，RPE65， Retinoid Isomerohydrolase，Retinol Isomerase，Retinal Pigment Epithelium-Specific Protein 65kDa，EC 5.3.3.22，Retinoid Isomerohydrolase RPE65，Retinal Pigment Epithelium Specific Protein 65，EC 5.3.3.-，EC:3.1.1.64，Lutein Isomerase，Meso-Zeaxanthin Isomerase，BCO Family， Member 3，Retinitis Pigmentosa 20 (Autosomal Recessive)，RBP-Binding Membrane Protein，EC 3.1.1.64，MRPE65，SRPE65，BCO3，LCA2，Rd12，RP20，P63
Product Inquiry
Clinical Drug Information
| English Name | Research Code | Highest Development Stage | Company | Indication | Clinical Trial |
|---|---|---|---|---|---|
| Emixustat Hydrochloride | ACU-02; ACU-4429; ACU-4429 HCl | Phase 3 Clinical | Acucela Inc | Anetoderma; Geographic Atrophy; Stargardt Disease; Diabetic Retinopathy; Macular Degeneration | Details |
| LX-101 | LX-101 | Phase 3 Clinical | Shanghai Langsheng Biological Technology Co Ltd | Retinal Dystrophies; Retinal Dysplasia; Retinal Degeneration | Details |
| HORA-RPE65 (Horama) | HORA-RPE65 | Phase 2 Clinical | Horama Sa | Leber Congenital Amaurosis | Details |
| HG-004 | HG-004 | Phase 2 Clinical | HuiGene Therapeutics Ltd | Leber Congenital Amaurosis; Retinal Diseases | Details |
| FT-001(Frontera Therapeutics) | FT-001; FT-001(Frontera Therapeutics) | Phase 2 Clinical | Frontera Therapeutics Inc | Retinal Dysplasia | Details |
Marketed Drug Information
| English Name | Research Code | Highest Development Stage | Company | First Brand Name | First Approved Country | First Indication | First Approved Company | First Approved Date | Indication | Clinical Trial |
|---|---|---|---|---|---|---|---|---|---|---|
| Voretigene neparvovec-rzyl | 2SPI046IKD; rAAV.hRPE65v2; AAV2-hRPE65v2; SPKRPE-65; rAAV2-CBSB-hRPE65; AAV2.RPE65 | Approved | Childrens Hospital of Philadelphia | Luxturna | United States | Retinal Dysplasia | Spark Therapeutics Inc | 2017-12-19 | Vision Disorders; Retinal Dystrophies; Leber Congenital Amaurosis; Retinal Dysplasia | Details |