| Gene symbol | ABCA4 | Synonyms | ABC10, ABCR, ARMD2, CORD3, FFM, RMP, RP19, STGD, STGD1 | Type of gene | protein-coding |
| Chromosome | 1 | Map location | 1p22.1 | dbXrefs | |
| Gene perturbation-related omics dataset | PerturbAtlas | PertOrg | ||||
| Description | ATP binding cassette subfamily A member 4 | ||||
| GTO ID | GTC3914 |
| Trial ID | NCT06467344 |
| Disease | Stargardt Disease | Cone Rod Dystrophy | Juvenile Macular Degeneration |
| Altered gene | ABCA4 |
| Therapeutic/Target gene | Therapeutic gene |
| Therapy | Gene transfer |
| Treatment | ACDN-01 |
| Phase | Phase1|Phase2 |
| Recruitment status | Recruiting |
| Title | ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy |
| Year | 2024 |
| Country | United States |
| Company sponsor | Ascidian Therapeutics, Inc |
| Other ID(s) | ACDN-01-001 |
| Vector information | |||||||||
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| Cohort 1 | |||||||
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