ACDN-01
Asset
RNA exon editing therapy to replace mutated ABCA4 exons.
- Stage
- Phase 1
- Modality
- Gene therapy
- Target
- ABCA4
- 4
- Indications
- 1
- Trial
- 1
- Developer
Indications
4Cone rod dystrophy
Phase 1, Recruiting, NCT06467344, clinicaltrials.gov, 8 Oct 2026
Juvenile macular degeneration
Phase 1, Recruiting, NCT06467344, clinicaltrials.gov, 8 Oct 2026
Stargardt disease
Phase 1, Recruiting, NCT06467344, clinicaltrials.gov, 8 Oct 2026
1 more
In the full profile
- Targets and what it contains1
- Owner and partners1
- Trials1
Every asset’s record: target, stage, trials and the evidence behind it.
Updated 8 Oct 2026, 1 source