NGGT Shares Early Clinical Trial Results for BCD Treatment

In a recent publication in JAMA Ophthalmology, NGGT Inc., a biotechnology firm based in Walnut Creek, California, revealed promising results from a dose-escalation study examining the safety and potential efficacy of NGGT001, a gene therapy designed for Bietti Crystalline Dystrophy (BCD). BCD is an uncommon genetic eye condition stemming from mutations in the CYP4V2 gene, leading to progressive vision deterioration and often resulting in legal blindness. Presently, no approved treatments exist for BCD, offering little hope for patients afflicted by this condition.

The study, which took place across two locations, was an open-label trial involving 12 participants with confirmed genetic diagnoses of BCD. Each participant received a single subretinal injection of the gene therapy rAAV-hCYP4V2 (NGGT001), which delivers a functional version of the CYP4V2 gene. The injections contained either 1.5e11 or 3.0e11 total vector genomes. The primary aim of the study was to assess the safety of the treatment.

Throughout the 12-month follow-up period, the study recorded no significant safety issues. The majority of participants tolerated the gene therapy well, with only one individual experiencing mild intraocular inflammation that resolved promptly. Importantly, no severe adverse events or dose-limiting toxic effects were detected, providing an initial indication of the treatment's safety profile.

In terms of efficacy, although the trial's focus was on safety, some encouraging signs of vision improvement were noted. The treated eyes exhibited a mean enhancement of 13.9 letters on the Best Corrected Visual Acuity (BCVA) scale after 12 months, compared to an improvement of 6.3 letters in the untreated eyes. Notably, those participants who had residual autofluorescence (AF) in the fovea, which indicates remaining functional photoreceptors, experienced consistent visual gains during the study period.

The early results from this study suggest that NGGT001 may serve as a safe and potent therapeutic option for BCD. However, further investigations are essential to validate these findings and to explore the long-term impacts of the treatment.

Dr. Yiting Liu, Vice President of Translational Research at NGGT, expressed excitement about the trial outcomes, emphasizing the potential of NGGT001 to offer a safe treatment for BCD. Liu highlighted the observed visual improvements and the absence of severe adverse events over the year-long follow-up period. Dr. Lixin Jiang, CEO and Co-Founder of NGGT, echoed these sentiments, noting the impressive safety profile and sustained visual acuity improvements across participants as strong evidence supporting NGGT001's potential in treating BCD.

NGGT001 is founded on an AAV-based gene therapy approach aimed at supplying a functional CYP4V2 gene copy to restore proper fatty acid metabolism. BCD, an autosomal recessive disorder, involves a mutation in the CYP4V2 gene that disrupts metabolic enzyme function, resulting in lipid deposits forming in the retina and cornea. This leads to the degeneration of the retinal pigment epithelium, causing night blindness and decreasing vision, usually progressing to legal blindness between the second and third decades of life.

NGGT Inc. is a clinical-stage biotechnology company focused on developing innovative gene therapies for retinal, metabolic, and neurodegenerative diseases. The company is progressing with a robust therapeutic development pipeline, featuring multiple clinical and pre-clinical programs centered around several proprietary AAV products. These include NGGT001 for BCD, NGGT002 for PKU, NGGT006 for familial hypercholesterolemia, and NGGT007 for Wet-AMD.