ArticleMolecular therapy. Methods & clinical development2024
Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration.
Article in Molecular therapy. Methods & clinical development, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.
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Who cites it
6 citing papers in PubMed.
- AAV-based gene therapies for neovascular AMD.Gene therapy · 2026Review
- Advances in Gene Therapy for Age-Related Macular Degeneration: A Narrative Review.Journal of clinical medicine · 2026Review
- AAV-mediated multiple gene therapy combining VEGFA-targeting miR-agshRNAs and PEDF for the suppression of choroidal neovascularization.Molecular therapy. Nucleic acids · 2026Article
- Gene Therapy for Wet Age-Related Macular Degeneration.Bioengineering (Basel, Switzerland) · 2025Review
- Angiopoietin-1 and Tie2-Based Dual Cell Therapy Enhances Antiangiogenic Barrier Function in a Retina-Mimetic Model for Neovascular Retinal Disease.Tissue engineering and regenerative medicine · 2025Article
- The ambition for a one-and-done vision-saving AAV vector.Molecular therapy. Methods & clinical development · 2025Article
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Authors and funding
8 authors.
Funding
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Abstract
Age-related macular degeneration (AMD) is a leading cause of vision loss in individuals over the age of 55. Approximately 10%-15% of AMD patients develop choroidal neovascularization (CNV), leading to wet AMD (wAMD), which accounts for nearly 90% of AMD-related blindness. Inhibition of vascular endothelial growth factor (VEGF) is the standard treatment for wAMD. However, the frequent administration of the current treatment imposes a significant burden on wAMD patients. Therefore, there is an unmet need for treatments that require less-frequent administration. Here, we present findings on the safety and efficacy of NG101, a recombinant adeno-associated virus (rAAV) vector encoding aflibercept, an anti-VEGF agent, for wAMD therapy. A single subretinal injection of NG101 effectively reduced CNV lesion leakage and size at doses as low as 1 × 10
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