Table 1.
Summary of clinical trials for inherited retinal diseases (IRDs) using DNA therapies.
| Gene/Condition | Therapeutic Molecule | Clinical Trial Identifier | Status |
|---|---|---|---|
| Gene Augmentation | |||
| ABCA4 | SAR422459 | NCT01367444 | Recruiting |
| SAR422459 | NCT01736592 | Enrolling by invitation | |
| CHM | AAV2-hCHM | NCT02341807 | Active, not recruiting |
| AAV2-REP1 | NCT03496012 | Recruiting | |
| AAV2-REP1 | NCT03507686 | Recruiting | |
| AAV2-REP1 | NCT02407678 | Active, not recruiting | |
| rAAV2.REP1 | NCT02671539 | Active, not recruiting | |
| rAAV2.REP1 | NCT02077361 | Completed | |
| rAAV2.REP1 | NCT01461213 | Completed | |
| AAV2-REP1 | NCT03584165 | Enrolling by invitation | |
| AAV2-REP1 | NCT02553135 | Completed | |
| CNGA3 | AGTC-402 | NCT02935517 | Recruiting |
| AAV2/8-hG1.7p.coCNGA3 | NCT03758404 | Recruiting | |
| rAAV.hCNGA3 | NCT02610582 | Active, not recruiting | |
| CNGB3 | rAAV2tYF-PR1.7-hCNGB3 | NCT02599922 | Recruiting |
| AAV2/8-hCARp.hCNGB3 | NCT03001310 | Recruiting | |
| AAV-CNGB3 | NCT03278873 | Recruiting | |
| MERTK | rAAV2-VMD2-hMERTK | NCT01482195 | Recruiting |
| MYO7A | UshStat | NCT02065011 | Enrolling by invitation |
| PDE6B | AAV2/5-hPDE6B | NCT03328130 | Recruiting |
| RPE65 | AAV OPTIREP | NCT02946879 | Recruiting |
| rAAV2-CBSB-hRPE65 | NCT00481546 | Active, not recruiting | |
| AAV2-hRPE65v2- | NCT00516477 | Active, not recruiting | |
| AAV2-hRPE65v2 | NCT00999609 | Active, not recruiting | |
| AAV2-hRPE65v2 | NCT01208389 | Active, not recruiting | |
| AAV2-hRPE65v2 | NCT03602820 | Active, not recruiting | |
| tgAAG76 (rAAV 2/2.hRPE65p.hRPE65) | NCT00643747 | Completed | |
| rAAV2-CB-hRPE65 | NCT00749957 | Completed | |
| rAAV2-hRPE65 | NCT00821340 | Completed | |
| rAAV2/4.hRPE65 | NCT01496040 | Completed | |
| AAV RPE65 | NCT02781480 | Completed | |
| RLBP1 | CPK850 | NCT03374657 | Recruiting |
| RPGR | AAV8-RPGR | NCT03116113 | Recruiting |
| AAV2/5-hRKp.RPGR | NCT03252847 | Recruiting | |
| rAAV2tYF-GRK1-RPGR | NCT03316560 | Recruiting | |
| RS1 | AAV8-scRS/IRBPhRS | NCT02317887 | Recruiting |
| rAAV2tYF-CB-hRS1 | NCT02416622 | Active, not recruiting | |
| Genome editing | |||
| CEP290 | EDIT-101 (AGN-151587) | NCT03872479 | Recruiting |
| Optogenetics | |||
| Advanced RP | RST-001 | NCT02556736 | Recruiting |
| Non-syndromic RP Retinitis Pigmentosa | GS030-DP | NCT03326336 | Recruiting |
Black and gray are just used to indicate the different sectiond and can be considered as headers. Gene or disease condition (first column), therapeutic molecule (second column), clinical trial identifier (third column) and the current status of the trial (last column) are indicated. Completed trials are highlighted in bold. Data obtained from https://clinicaltrials.gov/. RP: retinitis pigmentosa.