Molecular Vision: Zhao, Mol Vis 2025; 31:256-265. Table 1

Table 1. Gene therapy drugs and main clinical research results in BCD.

Characteristics	ZVS101e	NGGT001	VGR-R01
Basic Information
Trail number	NCT04722107	NCT06302608	NCT05399069
Vector	rAAV8	rAAV2	rAAV8
CYP4V2 Gene	Wild-type	Codon optimized	Codon optimized
Administration route	Sub-retinal injection	Sub-retinal injection	Sub-retinal injection
Study design
Number of patients	12	12	15
Dose groups	Single dose:	Two doses:	Three doses:
	7.5×10¹⁰ vg (n=12)	1.5×10¹¹ vg (n=6)	6.0×10¹⁰ vg (n=3)
		3.0×10¹¹ vg (n=6)	1.2×10¹¹ vg (n=6)
			2.0×10¹¹ vg (n=6)
Follow-up duration	6–12 months	9 months	6–9 months
Efficacy outcomes
Mean BCVA change¹	Day 180: 9.0±10.8	Day 270: 12.8±3.7	Day 180:
	Day 365: 11.0±10.6		Low dose: 13.7±2.2
			Medium dose: 21.1±7.5
			High dose: 27.4±16.7
			Day 270:
			Low dose: 13.3±1.9
			Medium dose: 27.9±16.5
BCVA improvement Rate²	Day 180: 77.8% (7/9)	46% (5/11) gained >15 letters	75% (9/12) gained >10 letters; 66.7% (8/12) gained >15 letters
	Day 365: 80% (4/5) gained letters;40% (2/5) gained >15 letters
Safety profile
Ocular adverse events³	Conjunctival edema	Conjunctival edema	Conjunctival edema
	Anterior chamber flare	Eyelid edema	Elevated IOP
	Posterior corneal deposits		Posterior corneal deposits

1. BCVA (best corrected visual acuity) changes are presented as mean ± standard deviation in ETDRS letters. 2. BCVA improvement rates are shown as percentages, with the actual number of patients shown in parentheses (n/N, where n=number of patients showing improvement, n=total number of patients evaluated). 3. Adverse events are listed in order of frequency of occurrence across all treatment groups. Abbreviations: BCD=Bietti crystalline dystrophy; rAAV=recombinant adeno-associated virus; vg=vector genome; IOP=intraocular pressure; ETDRS=early treatment diabetic retinopathy study.