NG101 gene therapy reduced supplemental anti-VEGF injections by 90% at 44 weeks in a Phase 1/2a wet AMD trial, with no serious safety signals reported at ASRS 2026.
Written By: Anshu Gupta, PharmD
Reviewed By: Pharmacally Editorial Team
Reyon Pharmaceutical and its development partner Elisigen have reported new Phase 1/2a (NCT05984927) clinical data for NG101, an investigational gene therapy for wet age-related macular degeneration (wAMD), showing sustained efficacy and a favorable safety profile through 44 weeks in the trial’s mid-dose cohort. The findings were presented at the American Society of Retina Specialists (ASRS) 2026 annual meeting, highlighting the therapy’s potential to reduce the long-term treatment burden associated with repeated anti-VEGF injections.
The newly reported 44-week findings from the mid-dose cohort build on the previously disclosed 52-week follow-up results from the low-dose cohort, which were presented in May. Together, the data strengthen evidence of a dose-dependent treatment response while supporting a favorable safety profile across the dose levels evaluated to date.
NG101 Targets Long-Term VEGF Suppression
Wet age-related macular degeneration is a leading cause of irreversible vision loss in older adults. Current treatment typically requires intravitreal anti-VEGF injections every few weeks to control abnormal blood vessel growth and retinal fluid accumulation, placing a significant burden on patients and healthcare systems.
NG101 is an adeno-associated virus (AAV)-based gene therapy intended to provide sustained intraocular production of an anti-VEGF therapeutic protein after a single administration. According to Elisigen, the therapy incorporates its proprietary CAT311 promoter technology, which enables high levels of gene expression despite using relatively low vector doses.
Phase 1/2a Data Show Durable Efficacy and Favorable Safety
The 44-week analysis of the mid-dose cohort (Cohort 2) demonstrated a 90% reduction in the frequency of supplemental anti-VEGF injections compared with patients’ historical treatment requirements. The findings suggest that many patients-maintained disease control with substantially fewer rescue injections following a single administration of NG101.
Investigators also reported improvements in visual function and retinal anatomical measures compared with the previously studied low-dose cohort, supporting a dose-dependent increase in therapeutic activity.
An important feature of the study was the low vector dose required to achieve these outcomes. According to the company, the dose administered in Cohort 2 represented approximately 2.3% to 10% of doses used by competing investigational gene therapies, reflecting the enhanced expression achieved through the CAT311 promoter platform.
Safety findings remained encouraging. No serious adverse events (SAEs) or dose-limiting toxicities (DLTs) related to treatment were reported in the mid-dose cohort through the 44-week evaluation period.
Manufacturing Strategy Advances Alongside Clinical Development
The clinical progress also supports Reyon Pharmaceutical’s manufacturing plans. Under a development agreement signed with Elisigen in 2020, Reyon holds exclusive global manufacturing rights for NG101 and plans to produce commercial supplies at its Good Manufacturing Practice (GMP)-certified bio-manufacturing facility in Chungju, South Korea.
Because NG101 achieves therapeutic activity at relatively low doses, commercial manufacturing could require less viral vector material, potentially improving production efficiency and scalability if the therapy reaches the market.
Next Milestone: High-Dose Cohort Results
According to Reyon Pharmaceutical, the next major clinical milestone will be the 24-week results from the high-dose cohort (Cohort 3), which are expected to be presented at the American Academy of Ophthalmology (AAO) meeting later this year.
Positive findings from that cohort could support advancement into a Phase 2b trial while strengthening opportunities for global licensing partnerships. The companies stated they will continue supporting ongoing clinical development alongside preparations for potential commercial manufacturing should NG101 progress successfully through later-stage development.
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About the Writer
Anshu Gupta (LinkedIn) is a PharmD professional and healthcare writer with interests in clinical research, pharmacovigilance, regulatory affairs, and medical writing. She has presented research at academic conferences and completed certifications in Good Clinical Practice (GCP), ICH-GCP, and drug safety. Passionate about clinical trials and evidence-based medicine, she is committed to translating scientific evidence into accurate, reliable, and accessible healthcare content.