Table 2.
Disease Target | Product | Modality | Phase | Sponsor | NCT Number |
---|---|---|---|---|---|
Retinitis pigmentosa/LCA | OCU400 AAV.hNr2e3 | Gene therapy | 1/2 | Ocugen | NCT05203939 |
Rod-cone dystrophy | SPVN06/AAV-RdCVF-RdCVFL | Gene therapy | 1/2 | SparingVision | NCT05748873 |
Stargardt disease | OCU410ST/AAV5.hRORA | Gene therapy | 1/2 | Ocugen | NCT05956626 |
Non-neovascular AMD | VOY-101 | Gene therapy | 1/2a | Perceive Biotherapeutics | NCT06087458 |
AAVCAGsCD59 | Gene therapy | 2 | Janssen | NCT03144999 | |
OCU410ST/AAV5.hRORA | Gene therapy | 1/2 | Ocugen | NCT06018558 | |
Ionis-FB-LRx | Antisense oligonucleotide | 2 | Ionis | NCT03446144 | |
Ionis-FB-LRx | Antisense oligonucleotide | 2 | Ionis | NCT03815825 | |
GT005 | Gene therapy | 1/2 | Gyroscope | NCT03846193 | |
Neovascular AMD | RGX-314 | Gene therapy | 1/2 | REGENXBIO | NCT03066258 |
RGX-314 | Gene therapy | 2/3 | REGENXBIO/AbbVie | NCT04704921 | |
RGX-314 | Gene therapy | 3 | REGENXBIO/AbbVie | NCT05407636 | |
SKG0106 | Gene therapy | 1/2 | Skyline Therapeutics | NCT05986864 NCT06213038 | |
4D-150 | Gene therapy | 1/2 | 4DMT | NCT05197270 | |
ADVM-022 | Gene therapy | 2 | Adverum | NCT05536973 |
Note: Inclusion in this table does not necessarily indicate that a trial is active.