Table 2.

Multiple Interventions Targeting the Same IRDs in Ongoing and Planned Clinical Trials

IRD	Intervention	Study Sponsor	CT.gov identifier
ABCA4-related retinopathy
Gene therapy Visual cycle modulators Complement factor inhibitor hES-Derived RPE transplantation	EIAV-CMVp-ABCA4 (SAR422459) Emixustat (RPE65 inhibitor) ALK-001 (deuterated vitamin A) Zimura (avacincaptad pegol) MA09-hRPE	Sanofi Acucela Alkeus Pharmaceuticals IVERIC Bio Astellas Pharma	NCT01367444 NCT03772665 NCT02402660 NCT03364153 NCT01345006
CEP290-related retinopathy
Splice-modulating oligonucleotide CRISPR-Cas9 gene editing	sepofarsen (QR-110) EDIT-101 (AGN-151587)	ProQR Therapeutics NV Editas Medicine/Allergan	NCT03913143 NCT03872479
REP1 choroideremia
Gene therapy	AAV2-hCHM AAV2-REP1 rAAV2.REP1 AAV-REP1 4D-110	Spark Therapeutics Nightstar Therapeutics/Biogen STZ eyetrial University of Oxford 4D Molecular	NCT02341807 NCT03496012 NCT02671539 NCT02407678 Not available
CNGA3 achromatopsia
Gene therapy	rAAV2tYF-PR1.7-hCNGA3 AAV2/8-hG1.7p.coCNGA3 rAAV.hCNGA3	Applied Genetic Technologies Corp MeiraGTx STZ eyetrial	NCT02935517 NCT03758404 NCT02610582
CNGB3 achromatopsia
Gene therapy	rAAV2tYF-PR1.7-hCNGB3 rAAV2/8-hCARp.hCNGB3	Applied Genetic Technologies Corp MeiraGTx	NCT02599922 NCT03001310
RPE65-related retinopathy
Gene therapy	rAAV2/5-OPTIRPE65 rAAV2-CBSB-hRPE65 rAAV2-CB-hRPE65 rAAV2/4.hRPE65 rAAV2-hRPE65 AAV2-hRPE65v2/Luxturna	MeiraGTx University of Pennsylvania Applied Genetic Technologies Corp Nantes University Hospital Hadassah Medical Organization Spark Therapeutics	NCT02946879 NCT00481546 NCT00749957 NCT01496040 NCT00821340 NCT00999609; Approved
RPGR-related retinopathy
Gene therapy	rAAV2tYF-GRK1-RPGR rAAV5-hRKp.RPGR rAAV8-RPGR (BIIB112)	Applied Genetic Technologies Corp MeiraGTx Nightstar Therapeutics/Biogen	NCT03316560 NCT03252847 NCT03116113
RS1 X-linked retinoschisis
Gene therapy	rAAV2tYF-CB-hRS1 AAV8-scRS/IRBPhRS	Applied Genetic Technologies Corp National Eye Institute (NIH)	NCT02416622 NCT02317887