Table 2 of
Molleti, Mol Vis 2025; 31:454-461. Table 2 of
Molleti, Mol Vis 2025; 31:454-461.
Table 2. Summary of achromatopsia gene therapy clinical trials.
| NCT Registration # | Phase | Gene | Vector | Sponsor | Status | Route | Year of Last Update |
|---|---|---|---|---|---|---|---|
| 2935517 | Phase I/II | CNGA3 | rAAV2tYF-PR1.7-hCNGA3 | Beacon Therapeutics | Active, not recruiting | Sub-retinal | 2022 |
| 2599922 | Phase I/II | CNGB3 | rAAV2tYF-PR1.7-hCNGB3 | Beacon Therapeutics | Active, not recruiting | Sub-retinal | 2022 |
| 3758404 | Phase I/II | CNGA3 | AAV2/8-hG1.7p.coCNGA3 | MeiraGTx UK II Ltd | Completed | Sub-retinal | 2022 |
| 3001310 | Phase I/II | CNGB3 | AAV2/8-hCARp.hCNGB3 | MeiraGTx UK II Ltd | Completed | Sub-retinal | 2023 |
| 2610582 | Phase I/II | CNGA3 | rAAV.hCNGA3 | STZ eyetrial | Active, not recruiting | Sub-retinal | 2024 |