Table 3.

Clinical trials of gene therapy for neovascular eye diseases

	Trial identifier	Disease	Phase	Target gene	Vector	Administration route	Actual/estimated enrolment	Trial country	Sponsor	Year	Status
Inhibition of the VEGF-signaling cascade	NCT03066258	Wet AMD	I	Soluble anti-VEGF protein	AAV8 (RGX-314)	Intravitreal	42	USA	Regenxbio	2017	Open
	NCT01024998	Wet AMD	I	sFLT01	AAV2.sFlt-1	Intravitreal	19	USA	Sanofi	2010	Open
	NCT01494805	Wet AMD	I/II	sFLT-1	rAAV-sFlt-1	Subretinal	40	Australia	Adverum Biotechnologies	2011	Completed
	NCT00395057	Wet AMD	II	siRNA against VEGFR-1	AGN211745 (Sirna-027)	Intravitreal	138	USA, Australia and Philippines	Allergan	2007	Terminated
	NCT00259753	Wet AMD	II	siRNA against VEGF	Bevasiranib (Cand5)	Intravitreal	120	USA	OPKO Health	2005	Completed
	NCT00722384	Wet AMD	I	siRNA against VEGF	Bevasiranib (Cand5)	Intravitreal	15	USA	OPKO Health	2004	Completed
	NCT00363714	Wet AMD	I/II	siRNA against VEGFR-1	AGN211745 (Sirna-027)	Intravitreal	26	USA	Allergan	2004	Completed
	NCT00306904	DME	II	siRNA against VEGF	Bevasiranib (Cand5)	Intravitreal	48	USA	OPKO Health	2006	Completed
Inhibition of alternative angiogenic pathways	NCT03144999	Wet AMD	I	Soluble CD59	AAVCAGsCD59	Intravitreal	17	USA	Hemera Biosciences	2017	Open
	NCT01678872	Wet AMD	I	Endostatin- Angiostatin	Lenti-EIAV (RetinoStat)	Subretinal	18	USA	Oxford BioMedica	2012	Active, not recruiting
	NCT01301443	Wet AMD	I	Endostatin-Angiostatin	Lenti-EIAV (RetinoStat)	Subretinal	21	USA	Oxford BioMedica	2011	Completed
	NCT00109499	Wet AMD	I	PEDF	AdGVPEDF.11D	Intravitreal	N/A	USA	GenVec	2005	Completed

N/A not available, AAV adeno-associated virus, DME diabetic macular edema, Lenti lentivirus