Phase I/II Study of NGGT001 Gene Therapy for Bietti Crystalline Dystrophy.
Mid-dose gene therapy showed most consistent visual acuity gains in Bietti crystalline dystrophy
Phase I/II Study of NGGT001 Gene Therapy for Bietti Crystalline Dystrophy.
To evaluate the safety and preliminary functional outcomes of NGGT001 gene therapy across dose cohorts in adults with Bietti crystalline dystrophy (bietti crystalline dystrophy) with relatively preserved vision.
Twelve adults with genetically confirmed bietti crystalline dystrophy.
NGGT001 was well tolerated with no treatment-related serious adverse events and no evidence of intraocular inflammation or retinal toxicity.
Across treated eyes, modest improvements in functional outcomes were observed, with the most consistent changes in the mid-dose cohort (1.5 × 10 11 vg/eye).
Mean (standard deviation) microperimetry sensitivity changes from baseline were 0.86 (2.73) dB at month 6 and 0.73 (2.82) dB at month 12.
Untreated fellow eyes showed mean (standard deviation) BCVA changes of 6.9 (6.6) letters and microperimetry fluctuations of 0.60 (2.2) dB at month 12.
Subretinal NGGT001 demonstrated a favorable safety profile in this phase I/II study. The mid-dose cohort showed the most consistent functional improvements, although bilateral variability was observed.
These findings support further evaluation of NGGT001 in randomized controlled trials.