Reyon Pharmaceutical and Neuracle Genetics announced that they have completed the dosing of all 20 subjects in the North American Phase 1/2a clinical trial for 'NG101', a co-developed gene therapy for wet age-related macular degeneration (wet AMD), on December 17 (local time). This clinical trial was conducted as an open-label, dose-escalation study. While the trial was originally scheduled to dose a total of 18 subjects—with 6 participants each in the low-, mid-, and high-dose cohorts—the total number expanded to 20 as two additional participants were included in the high-dose cohort.
An official explained, "We previously presented the initial clinical data for the low-dose cohort (Cohort 1) at the Retina Society 2025 meeting held in the United States on September 12. According to that presentation, the 6-month follow-up results demonstrated encouraging outcomes, showing a 91% reduction in the frequency of anti-VEGF rescue therapy, while visual acuity and anatomical metrics remained stable. Based on these findings, we highly anticipate even greater therapeutic efficacy in the high-dose cohort."
NG101 is an adeno-associated virus (AAV)-based gene therapy that carries the gene for the active ingredient of Eylea. It is designed to allow retinal cells to express the therapeutic protein over the long term through a single administration. Unlike conventional treatments that require repeated injections, NG101 has a strong advantage in dramatically reducing the injection burden on patients, drawing high interest from the global market. Based on this evaluation, NG101 is targeting the macular degeneration treatment market, which is projected to grow from approximately 10 trillion KRW in 2025 to approximately 23 trillion KRW by 2031.
Reyon Pharmaceutical holds exclusive global manufacturing and supply rights for NG101 and will be responsible for producing commercial quantities in the future through its biopharmaceutical manufacturing facility at the Chungju plant, which was constructed with an investment of approximately 300 billion KRW.
The Chungju plant houses Korea's only facility dedicated to E. coli fermentation-based pDNA production and has also completed the development of animal cell culture-based AAV process technology. Consequently, it is expected to become the only facility in Korea capable of providing one-stop production for both plasmid DNA (pDNA) and AAV. Reyon Pharmaceutical is already playing a pivotal role by manufacturing the AAV-use pDNA for the Phase 2 clinical trial of NG101. Securing superior pDNA productivity compared to foreign biotech firms is expected to provide a foundation for NG101 to gain a stable and cost-effective supply advantage in the global competitive landscape.
All subjects in this clinical trial will be followed up for up to 5 years to evaluate the long-term efficacy and safety of NG101. Considering the overall schedule, both companies project that the interim report will be secured by the third quarter of 2026. Based on this data, they plan to fully initiate discussions for global Phase 2b entry and technology licensing (licensing out).
An official from Reyon Pharmaceutical stated, "The completion of dosing for NG101 is a major milestone for Reyon Pharmaceutical, which plays a core role across the entire life cycle of clinical trials, manufacturing, and commercialization. Backed by our exclusive global manufacturing and supply rights, we are preparing to ensure seamless production of commercial volumes at the Chungju plant." The official added, "We will secure a meaningful competitive edge in future global technology licensing and partnering processes, thereby contributing to presenting a new paradigm for wet macular degeneration treatment."
