Table 4.
List of therapeutic molecules in clinical trials for the treatment RDs.
| Retinal dystrophy | Targeted gene | Therapeutic approach | Phase | Year | NCT ID |
|---|---|---|---|---|---|
| LCA | CEP290 Intron 26(IVS26) | subretinal EDIT-101 (CRISPR/Cas9) | I/II | 2019–24 | NCT03872479 |
| CEP290 p.Cys998X | Intravitreal QR-110(Antisense oligonucleotides) | I/II | 2017–19 | NCT03140969 | |
| I/II | 2019–21 | NCT03913130 | |||
| II/III | 2019–21 | NCT03913143 | |||
| RPE65 | Subretinal rAAV2-CBSB-hRPE65 | I | 2007–26 | NCT00481546 | |
| Subretinal tgAAG76(rAAV2/2.hRPE65p.hRPE65) | I/II | 2007–14 | NCT00643747 | ||
| rAAV2-CBSB-hRPE65 | I/II | 2009–17 | NCT00749957 | ||
| Subretinal AAV2-hRPE65v2 | I | 2007–18 | NCT00516477 | ||
| III | 2012–29 | NCT01208389 | |||
| I/II | 2010–26 | NCT00999609 | |||
| Subretinal rAAV2-hRPE65 | I | 2009–17 | NCT00821340 | ||
| Subretinal rAAV2-CBSB-hRPE65 Applied | I/II | 2009–17 | NCT00749957 | ||
| Subretinal rAAV-2/4.hRPE65 | I/II | 2011–14 | NCT01496040 | ||
| Subretinal AAV2/5-OPTIRPE65 | I/II | 2016–18 | NCT02781480 | ||
| I/II | 2016–23 | NCT02946879 | |||
| CHM | REP1 | Subretinal rAAV2.REP1 | I/II | 2015–25 | NCT02077361 |
| Subretinal rAAV2.REP1 | I/II | 2011–17 | NCT01461213 | ||
| Subretinal AAV2.REP1 | II | 2016–21 | NCT02407678 | ||
| Subretinal rAAV2.REP1 | II | 2016–18 | NCT02671539 | ||
| Subretinal rAAV2.REP1 | II | 2015–18 | NCT02553135 | ||
| Subretinal BIIB111(AAV2-REP1) | II | 2018–22 | NCT03507686 | ||
| Subretinal AAV2-REP1 | III | 2017–20 | NCT03496012 | ||
| CHM | Intravitreal 4D-100 | I | 2020–23 | NCT04483440 | |
| Subretinal AAV2-hCHM | I/II | 2015–22 | NCT02341807 | ||
| RP | PDE6B | Subretinal AAV2/5-hPDE6B | I/II | 2017–24 | NCT03328130 |
| RLBP1 | Subretinal CPK850 | I/II | 2018–26 | NCT03374657 | |
| PDE6A | Subretinal rAAV.hPDE6A | I/II | 2019–25 | NCT04611503 | |
| USH2A | Intravitreal QR-421a | I/II | 2019–22 | NCT03780257 | |
| Advanced RP | ChR2 | Intravitreal RST-001 | I/II | 2015–35 | NCT02556736 |
| adRP | RHO | unilateral IVT injection QR-1123 | I/II | 2019–21 | NCT04123626 |
| XLRS | RS1 | Intravitreal rAAV2tYF-CB-hRS1 | I/II | 2015–23 | NCT02416622 |
| Intravitreal AAV8-scRS/IRBPhRS | I/II | 2015–23 | NCT02317887 | ||
| XLRP | RPGR | sub-retinal BIIB112 | I/II | 2017–20 | NCT03116113 |
| Intravitrea 4D-125 | I/II | 2020–23 | NCT04517149 | ||
| Subretinal AAV2/5-RPGR | I/II | 2017–20 | NCT03252847 | ||
| Subretinal AGTC-501 (rAAV2tYF-GRK1-RPGR) | I/II | 2018–26 | NCT03316560 | ||
| Subretinal AAV5-RPRG | III | 2021–22 | NCT04671433 | ||
| Achromatopsia | CNGB3 | Subretinal rAAV2tYF-PR1.7-hCNGB3 | I/II | 2016–25 | NCT02599922 |
| Subretinal rAAV.hCNGA3 | I/II | 2015–27 | NCT02610582 | ||
| Leber Hereditary Optic Neuropathy |
ND4 | GS010; Sham intravitreal injection | III | 2016–19 | NCT02652767 |
| III | 2016–18 | NCT02652780 | |||
| Intravitreal scAAV2-P1ND4v2 | I | 2014–23 | NCT02161380 | ||
| Intravitreal rAAV2-ND4 | — | 2011–15 | NCT01267422 | ||
| USH1B | — | Subretinal SAR422459 | I/II | 2012–19 | NCT01505062 |
| — | Blood draw for the laboratory assessment | I/II | 2013–32 | NCT02065011 | |
| Stargardt's Macular Degeneration | — | Long term follows up in all patients who received SAR422459 in previous study TDU13583 | I/II | 2012–34 | NCT01736592 |
| — | SAR422459 | I/II | 2011–19 | NCT01367444 | |
| Neovascular AMD | — | Subretinal RetinoStat | I | 2011–15 | NCT01301443 |
| — | I | 2012–29 | NCT01678872 | ||
| — | Intravitreal AAV2-sFLT01 | I | 2010–18 | NCT01024998 | |
| — | Subretinal rAAV.sFlt-1; Control (ranibizumab alone) | I/II | 2011–17 | NCT01494805 |
Abbreviations: RPE65: Retinal pigmented epithelium-specific protein with molecular mass 65 kDa; CEP290: Centrosomal Protein 290; ChR2: Channelrhodopsin-2; PDE6B: Phosphodiesterase 6B; RLBP1: Retinaldehyde-binding protein 1; USH2A: Usherin; PDE6A: Phosphodiesterase 6A; RPGR: Retinitis pigmentosa GTPase regulator; REP1: Rab escort protein 1; RS1: Retinoschisin.