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. 2024 Dec 17;13(12):28. doi: 10.1167/tvst.13.12.28

Table 2.

Gene-Agnostic Gene and Genetic Therapy Clinical Trials for IRDs and AMD

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.