NG101 / Neuracle Sci, Reyon Pharma - LARVOL DELTA

Low-Dose AAV Gene Therapy NG101 for Wet AMD: Preclinical Results and Ongoing Phase 1/2a Trial Design (ARVO 2025) - P1/2 | "At 3×109 vg/eye, NG101 showed comparable efficacy to Eylea (aflibercept) in suppressing CNV formation in NHPs. By delivering instructions for the eye to produce therapeutic proteins on its own, we hope to reduce the need for repeated treatments. If successful, this approach could mean fewer doctor visits, less stress, and better long-term vision for people affected by wet AMD."

Gene therapy • P1/2 data • Preclinical • Age-related Macular Degeneration • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

NG101 AAV Gene Therapy in Subjects with Wet Age-Related Macular Degeneration (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Neuracle Genetics, Inc | Trial primary completion date: Jan 2025 ➔ Jan 2026

Trial primary completion date • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Lee Yeon Pharmaceutical, GMP production in full swing… Gene therapy 'NG101' clinical trials 1 and 2a underway [Google translation] (MSN News) - "Yeonghan Pharmaceutical announced on the 6th that the wet age-related macular degeneration gene therapy (NG101) that it is jointly developing with its partner company, Newracle Genetics, is currently undergoing phase 1/2a clinical trials at a total of four clinical trial institutions located in the United States and Canada....Neuracle Genetics plans to announce the results of the related clinical trial within the first half of the year."

P1/2 data • Trial status • Wet Age-related Macular Degeneration

Preclinical evaluation of NG101, a potential AAV gene therapy for wet age-related macular degeneration. (PubMed, Mol Ther Methods Clin Dev) - "Only mild and transient ocular inflammatory responses were observed. Overall, these findings suggest that NG101, with its efficacy at low doses and sustained expression, is a promising therapeutic candidate for wAMD."

Gene therapy • Journal • Preclinical • Age-related Macular Degeneration • Gene Therapies • Inflammation • Macular Degeneration • Ocular Inflammation • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

Preclinical Evaluation of NG101 for Treatment of Wet Age-Related Macular Degeneration (ASGCT 2024) - "Considering the excellent aflibercept expression ability of NG101, we assessed the efficacy of NG101 at doses of 3x109 and 6x109 vg/eye in the NHP CNV model and demonstrated that NG101 effectively inhibited the growth and leakage of new vessels, to the same extent as intravitreal injection of Eylea (2mg). In non-human primates, NG101 administration surpassed the required Aflibercept amount for wAMD treatment in ocular tissues. Evaluation in NHPs showed NG101's efficacy in inhibiting vessel growth and leakage at significant low doses, making it a promising candidate for wAMD treatment with potential reductions in AAV gene therapy-related toxicities."

Preclinical • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration

NG101 AAV Gene Therapy in Subjects With Wet Age-Related Macular Degeneration (clinicaltrials.gov) - P1/2 | N=18 | Recruiting | Sponsor: Neuracle Genetics, Inc | Not yet recruiting ➔ Recruiting

Enrollment open • Gene therapy • Age-related Macular Degeneration • Gene Therapies • Macular Degeneration • Ophthalmology • Retinal Disorders • Wet Age-related Macular Degeneration