Table 1.

Summary of ongoing clinical trials targeting LCA/EOSRD

Gene	Drug	Clinical trial ID (NCT #)	Study phase	Route of delivery	Status	Sponsor	Location	Number of participants
RPE65	rAAV 2/2.hRPE65p.hRPE65–OPTIRPE65	NCT00643747– NCT02781480	Phase I–II	Subretinal	Completed	MeiraGTx	UK	12–15
RPE65	rAAV2-CBSB–hRPE65	NCT00481546	Phase I	Subretinal	Active, not recruiting	University of Pennsylvania	USA	15
RPE65	voretigene neparvovec-rzyl (LuxturnaTM)	NCT00999609	Phase III	Subretinal	Active, not recruiting	Spark Therapeutics	USA	31
RPE65	rAAV2-CB–hRPE65	NCT00749957	Phase I-II	Subretinal	Completed	Applied Genetic Technologies Corp	USA	12
GUCY2D	SAR439483	NCT03920007	Phase I–II	Subretinal	Active, not recruiting	Sanofi	USA	15
CEP290	QR-110	NCT03140969	Phase I/II	Intravitreal	Completed	ProQR Therapeutics	USA and Belgium	11
CEP290	AGN-151587	NCT03872479	Phase I–II	Subretinal	Recruiting	Allergan	USA	18
RPE65 & LRAT	QLT091001	NCT01521793	Phase I	Oral	Completed	QLT Inc.	USA and Europe	27
RPE65 & LRAT	QLT091001	NCT01014052	Phase I	Oral	Completed	QLT Inc.	USA and Europe	32
–	Human RPE cells	NCT03566147	Phase I	Subretinal	Unknown	Eyecure Therapeutics Inc.	China	30
–	Argus II retinal prosthesis	NCT01490827	Observational	Subretinal	Terminated	Second Sight Medical Products	Europe	52
–	RST-001	NCT02556736	Phase I/II	Intravitreal	Active, not recruiting	Allergan	USA	12
–	GS030-DP and GS030-MD	NCT03326336	Phase I/II	Intravitreal	Recruiting	GenSight Biologics	USA and Europe	18

EOSRD, early-onset severe retinal degeneration; LCA, leber congenital amaurosis; RPE, retinal pigment epithelium.