TABLE 1.
Clinical trials of gene therapy using viral vectors for IRDs (ClinicalTrials.gov).
| Phase | Conditions | Target gene | Interventions | Result | Sponsors and collaborators | NCT number |
| 1/2 | Achromatopsia | CNGB3 | Subretinal rAAV2tYF-PR1.7-hCNGB3 | NA | Applied Genetic Technologies Corp | NCT02599922 |
| 1/2 | Achromatopsia | CNGA3 | Subretinal rAAV.hCNGA3 | NA | STZ eyetrial | NCT02610582 |
| 1/2 | Achromatopsia | CNGB3 | Subretinal AAV2/8 viral vector | NA | MeiraGTx UK II Ltd | NCT03001310 |
| 2 | Choroideremia | REP1 | Subretinal AAV-REP1 | NA | Targeted Genetics Corporation | NCT02407678 |
| 2 | Choroideremia | REP1 | Subretinal AAV2-REP1 | NA | Byron Lam | NCT02553135 |
| 2 | Choroideremia | REP1 | Subretinal rAAV2.REP1 | NA | STZ eyetrial | NCT02671539 |
| 1/2 | Choroideremia | REP1 | Subretinal rAAV2.REP1 | Improved visual acuity at 6 months | UK Department of Health and Wellcome Trust | NCT01461213 |
| 1/2 | Choroideremia | REP1 | Subretinal rAAV2.REP1 | NA | Ian M. MacDonald, Alberta Innovates Health Solutions | NCT02077361 |
| 1/2 | Choroideremia | CHM | Subretinal AAV2-hCHM | NA | Spark Therapeutics | NCT02341807 |
| 1/2 | LCA | RPE65 | Subretinal tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) | Moderate and temporary improvement of retinal sensitivity | National Institute for Health Research and others; Targeted Genetics Corporation | NCT00643747 |
| 1 | LCA | RPE65 | Subretinal rAAV2-CBSB-hRPE65 | Improved visual sensitivity at 3 and 12 months followed by gradual diminution over 6 years, no serious adverse event | University of Pennsylvania, National Eye Institute (NEI) | NCT00481546 |
| 1 | LCA | RPE65 | Subretinal AAV2-hRPE65v2 | Modest improvement in subjective vision up to 2 years. The greatest improvement was noted in children. 1 of 12 subjects had temporary macular hole, no other adverse events. Greatest improvement in young patients. | Spark Therapeutics, The Children’s Hospital of Philadelphia | NCT00516477 |
| 1 | LCA | RPE65 | Subretinal rAAV2-hRPE65 | NA | Hadassah Medical Organization | NCT00821340 |
| 1 | LCA | RPE65 | Subretinal AAV2/5 OPTIRPE65 | NA | MeiraGTx UK II Ltd. | NCT02781480 |
| 1/2 | LCA | RPE65 | Subretinal AAV2/5 OPTIRPE65 | NA | MeiraGTx UK II Ltd., Syne Qua Non-Limited | NCT02946879 |
| 1/2 | LCA | RPE65 | Subretinal AAV2-hRPE65v2 (contralateral eye treatment) | Contralateral eyes of 11 subjects from 12 subjects who were enrolled in the previous phase 1 trial. Improved mobility and light sensitivity from day 30 to year 3. One case of endophthalmitis | Spark Therapeutics, The Children’s Hospital of Philadelphia | NCT01208389 |
| 3 | LCA, RP | RPE65 | Subretinal AAV2-hRPE65v2 | Bilateral subretinal injection. Significant improvement in functional vision as measured by the change in mobility testing between baseline and one year | Spark Therapeutics | NCT00999609 |
| ½ | LCA | RPE65 | Subretinal rAAV2-CB-hRPE65 | NA | Applied Genetic Technologies Corp | NCT00749957 |
| ½ | LCA | RPE65 | Subretinal rAAV2/4.hRPE65 | NA | Nantes University Hospital | NCT01496040 |
| ½ | LHON | ND4 | Intravitreal rAAV2-ND4 | Improved visual acuity and enlarged visual field, No local or systemic adverse events | Bin Li | NCT01267422 |
| 3 | LHON | ND4 | Intravitreal GS010 (AAV2/2 ND4) | NA | GenSight Biologics | NCT02652767 |
| 3 | LHON | ND4 | Intravitreal GS010 (AAV2/2-ND4) | NA | GenSight Biologics | NCT02652780 |
| ½ | Retinoschisis | RS1 | Intravitreal AAV8 scRS/IRBPhRS | NA | National Eye Institute (NEI) | NCT02317887 |
| ½ | Retinoschisis | RS1 | Intravitreal rAAV2tYF-CB-hRS1 | NA | Applied Genetic Technologies Corp | NCT02416622 |
| 1 | RP | MERTK | Subretinal rAAV2-VMD2-hMERTK | NA | Fowzan Alkuraya | NCT01482195 |
| ½ | RP | Channelrhodopsin-2 | Intravitreal RST-001 | NA | RetroSense Therapeutics | NCT02556736 |
| 1/2 | RP, X-linked | RPGR | Subretinal AAV-RPGR | NA | NightstaRx Limited | NCT03116113 |
| ½ | Stargardt Disease | ABCA5 | Subretinal SAR422459, Lentiviral vector | NA | Sanofi | NCT01367444 |
| ½ | Stargardt Disease | ABCA4 | Subretinal SAR422459, Lentiviral vector | NA | Sanofi | NCT01736592 |