| Gene symbol | RPE65 | Synonyms | BCO3, LCA2, RP20, mRPE65, p63, rd12, sRPE65 | Type of gene | protein-coding |
| Chromosome | 1 | Map location | 1p31.3 | dbXrefs | |
| Description | retinoid isomerohydrolase RPE65 | ||||
| GTO ID | GTC3440 |
| Trial ID | NCT05858983 |
| Disease | Retinal Dystrophy |
| Altered gene | RPE65 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | FT-001 |
| Phase | Phase1|Phase2 |
| Recruitment status | Recruiting |
| Title | A Multi-center, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of FT-001 Administered Via Subretinal Injection in Subjects With Biallelic RPE65 Mutation-associated Retinal Dystrophy |
| Year | 2023 |
| Country | China |
| Company sponsor | Frontera Therapeutics |
| Other ID(s) | FT001-C101 |
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