Next Generation Gene Therapeutics (NGGT) Announces Publication of Early Clinical Trial Results for NGGT001 for the Treatment of Bietti's Crystalline Dystrophy (BCD)

Results from dose-escalation study show safety and preliminary efficacy signal of NGGT001 for the treatment of BCD

WALNUT CREEK, Calif., Jan. 14, 2025 /PRNewswire/ -- NGGT Inc., ("Next Generation Gene Therapeutics" or "NGGT" or the "Company"), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced the publication in the journal JAMA Ophthalmology of the results from a dose-escalation study in patients with BCD, entitled "Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy".  The peer-reviewed article can be accessed here: Safety and Vision Outcomes Following Gene Therapy for Bietti Crystalline Dystrophy

Bietti Crystalline Dystrophy (BCD) is a rare, inherited genetic eye disease that causes progressive vision loss, often leading to legal blindness. Caused by mutations in the CYP4V2 gene, BCD currently has no approved treatment, leaving patients with limited options and a bleak prognosis.

This open-label, dose-escalation trial, conducted at two sites, involved 12 participants with genetically confirmed BCD. Participants received a single subretinal injection of either 1.5 e11 or 3.0e11 total vector genomes of a gene therapy called rAAV-hCYP4V2 (NGGT001), which delivers a functional copy of the CYP4V2 gene.

Key findings of the trial:

Safety: The trial found no substantial safety concerns associated with the gene therapy over 12 months of follow-up. Only one participant experienced mild intraocular inflammation, which resolved quickly. No severe adverse events related to the treatment or dose-limiting toxic effects were observed.

Vision Improvement: While the trial was primarily designed to assess safety, encouraging improvements in vision were observed. At 12 months, the treated eye showed a mean improvement of 13.9 letters on the BCVA, compared with 6.3 letters in the untreated eye. Importantly, participants with residual autofluorescence (AF) in the fovea, a sign of remaining functional photoreceptors, showed sustained visual gains over the 12 months.

These early findings suggest that gene therapy with rAAV-hCYP4V2 has the potential to be a safe and effective treatment for BCD. However, further research is needed to confirm these findings and to determine the long-term effects of the therapy.

"We are thrilled to see the results from our dose-escalation trial, which indicate the promise of NGGT001 to serve as a safe treatment for BCD," said Yiting Liu, Ph.D., VP of Translational Research at NGGT. "Across the 12-month span of patient follow-up, we observed promising indications of visual improvement following treatment with NGGT001 and no severe adverse events. We look forward to further clinical trials to validate NGGT001's ability to provide a durable treatment for this debilitating inherited disease."

"Given the lack of effective treatments for BCD, we are extremely encouraged by these results," said Lixin Jiang, M.D., Ph.D., CEO and Co-Founder of NGGT. "The impressive early safety profile of NGGT001 and sustained improvement in visual acuity in the study across patients strongly supports its potential for the treatment of BCD."

NGGT001 is an AAV-based gene therapy candidate designed to provide a functional copy of the CYP4V2 gene to restore enzymatic fatty acid metabolism. BCD is a severe autosomal recessive genetic disorder caused by a mutation in the CYP4V2 gene that causes metabolic enzyme dysfunction wherein yellow-white crystalline lipid deposits form in the retina and cornea, causing the retinal pigment epithelium to degenerate. Resultant symptoms include night blindness and decreased vision in the second to third decade of life, progressing to legal blindness.

About NGGT Inc.
NGGT is a clinical-stage biotechnology company developing novel gene therapies for the treatment of retinal, metabolic and neurodegenerative diseases. The Company is currently advancing an extensive therapeutic development pipeline with multiple clinical and pre-clinical programs built around several proprietary AAV (Adeno-Associated Virus) products: NGGT001, a Phase I/II-ready gene therapy for Bietti's Crystalline Dystrophy; NGGT002, a Phase I/II-ready gene therapy for PKU; NGGT006, a gene therapy for familial hypercholesterolemia, currently undergoing Investigator-Initiated Trials; and NGGT007, a gene therapy for Wet-AMD. NGGT is headquartered in Walnut Creek, California. For more information, please visit www.nggtbio.com, contact [email protected] and follow us on X and LinkedIn. 

For more information:
NGGT, Inc.
2910 Camino Diablo, Suite 120
Walnut Creek, CA 94597
[email protected]

SOURCE NGGT Inc.