Table 1.
Current clinical trials in gene therapy for IRDs
| Target gene | Drug/Vector | Phase | Route | Sponsor | Trial ID |
|---|---|---|---|---|---|
| LCA | |||||
| RPE65 | tgAAG76 (rAAV2/2.hRPE65p. hRPE65) | I/II | SR | UCL | NCT00643747 |
| AAV OPTIRPE65 | I/II | SR | MeiraGTx UK II Ltd | NCT02946879 | |
| AAV2-hRPE65v2 (voretigene neparvovec-rzyl) | III | SR | Spark Therapeutics | NCT00999609 | |
| rAAV2-CBSB-hRPE65 | I | SR | Uni of Penn | NCT00481546 | |
| rAAV2-hRPE65 | I | SR | Hadassah Medical Organization | NCT00821340 | |
| voretigene neparvovec-rzyl | III | SR | Novartis Pharmaceuticals (Japan) | NCT04516369 | |
| AAV2-hRPE65v2 (voretigene neparvovec-rzyl | Long term (15 years) | SR | Spark Therapeutics | NCT03602820 | |
| CEP290 | Sepofarsen | II/III | IVT | ProQR Therapeutics | NCT04855045 |
| QR-110 | I/II | IVT | ProQR Therapeutics | NCT03913130 | |
| EDIT-101 | I/II | SR | Editas Medicine | NCT03872479 | |
| GUCY2D | SAR439483 | I/II | SR | Atsena Therapeutics | NCT03920007 |
| Choroideremia | |||||
| REP-1 | AAV2-REP1BIIB111 (GEMINI) | II | SR | NightstaRx Ltd | NCT03507686 |
| rAAV2.REP1 vector | I/II | SR | Ian M. MacDonald | NCT02077361 | |
| AAV2-REP1 | II | SR | Byron Lam | NCT02553135 | |
| rAAV2.REP1 | I/II | SR | University of Oxford | NCT01461213 | |
| AAV2.REP1 | II | SR | University of Oxford | NCT02407678 | |
| rAAV2.REP1 | II | SR | STZ eyetrial | NCT02671539 | |
| AAV2-hCHM | I/II | SR | Spark Therapeutics | NCT02341807 | |
| RP | |||||
| vMCO-010 Optogenetic Therapy | II | IVT | Nanoscope Therapeutics | NCT04945772 | |
| MERTK-associated RP | |||||
| MERTK | rAAV2-VMD2-hMERTK | I | SR | King Khaled Eye Specialist Hospital | NCT01482195 |
| Advanced RP | |||||
| AAV2-virally-carried Multi-Characteristic Opsin I (vMCO-I) | I/II | IVT | Nanoscope Therapeutics |
NCT04919473 | |
| RST-001 | I/II | IVT | Allergan | NCT02556736 | |
| XLRS | |||||
| RS1 | AAV-RS1 vector AAV8-scRS/IRBPhRS) | I/II | IVT | NEI | NCT02317887 |
| rAAV2tYF-CB-hRS1 | I/II | IVT | AGTC | NCT02416622 | |
| Nonsyndromic RP | |||||
| Gene therapy: GS030-DP & Medical device: GS030-MD | I/II | IVT | GenSight Biologics | NCT03326336 | |
| PDE6A-associated RP | |||||
| rAAV.hPDE6A | I/II | SR | STZ eye trial | NCT04611503 | |
| PDE6B-associated RP | |||||
| AAV2/5-hPDE6B | I/II | SR | Horama S.A. | NCT03328130 | |
| XLRP | |||||
| RPGR | AAV8-RPGR Biological BIIB112 (XIRIUS) |
I/II | SR | NightstaRx Ltd | NCT03116113 |
| AAV2/5-RPGR | I/II | SR | MeiraGTx UK II Ltd | NCT03252847 | |
| AAV5-RPGR | III | SR | MeiraGTx UK II Ltd | NCT04671433 | |
| 4D-125 | I/II | IVT | 4D Molecular Therapeutics | NCT04517149 | |
| rAAV2tYF-GRK1-hRPGRco | II/III | SR | AGTC | NCT04850118 | |
| RLBP1-associated RP | |||||
| RLBP1 | CPK850 | I/II | SR | Novartis Pharmaceuticals | NCT03374657 |
| Bietti’s crystalline dystrophy | |||||
| CYP4V2 | rAAV2/8-hCYP4V2 | Early Phase 1 | Beijing Tongren Hospital | NCT04722107 | |
| Usher syndrome | |||||
| USH1B | SAR421869 | I/II | SR | Sanofi | NCT01505062 |
| USH2A (Exon13) | QR-421a RNA antisense oligonucleotide | III | IVT | ProQR Therapeutics | NCT05158296 |
| Autosomal dominant RP | |||||
| RHO (P23mutation) | QR-1123 | I/II | IVT | ProQR Therapeutics | NCT04123626 |
| Achromatopsia | |||||
| CNGA3 | AAV- CNGA3 | I/II | SR | MeiraGTx UK II Ltd | NCT03758404 |
| CNGB3 | AAV - CNGB3 | I/II | SR | MeiraGTx UK II Ltd | NCT03001310 |
| CNGA3 | AGTC-402 | I/II | SR | AGTC | NCT02935517 |
| CNGB3 | AGTC-401 | I/II | SR | AGTC | NCT02599922 |
| CNGA3 | rAAV.hCNGA3 | I/II | SR | STZ eyetrial | NCT02610582 |
Source: www.clinicaltrials.org AGTC-Applied Genetic Technologies Corp., NEI-National Eye Institute, UCL-University College London, SR-subretinal, IVT-intravitreal, LCA-Leber Congenital Amaurosis, RP-Retinitis Pigmentosa, XLRS-X-linked Retinischisis