VeonGen Therapeutics, a clinical-stage company based in Munich, Germany, announced preliminary data from its ongoing Phase I/II first-in-human trial of VG801 gene therapy in patients with ABCA4-associated retinal disease, including Stargardt disease. According to the company, nine patients have completed six-month follow-up, with some reaching twelve months. VeonGen said the data will be presented in an oral session at the 2026 Association for Research in Vision and Ophthalmology annual meeting in Denver this May.
The company reported that VG801 was well tolerated, with no dose-limiting toxicities or serious adverse events observed to date across both adult and pediatric patients who have been dosed. VeonGen stated that preliminary efficacy assessments showed "consistent functional improvements" in best-corrected visual acuity and a proprietary Virtual Reality Visual Test, sustained through six and twelve months of follow-up. However, the company did not disclose specific numerical values for either endpoint, nor did it report the total number of patients enrolled or dosed, the dose levels administered, or the magnitude of any observed changes. No control arm was described.
The Phase I/II trial is evaluating safety, tolerability, and preliminary efficacy of VG801 in patients with ABCA4-mutated retinal dystrophies. The study design does not appear to include randomization or a placebo comparator based on available disclosures. The VRVT endpoint has been accepted into the US FDA Rare Disease Endpoint Advancement pilot program, and VeonGen said it has conducted a separate validation study to support potential use of VRVT in a future pivotal trial. VG801 holds FDA Regenerative Medicine Advanced Therapy designation, Rare Pediatric Disease designation, and Orphan Drug designation, as well as European Medicines Agency clinical trial authorization and China CDE IND approval. Efficacy data in quantitative form were not reported, dosing details were not disclosed, and the data are interim and based on a small subset of patients who have completed follow-up.
VG801 is a dual-AAV vector therapy designed to deliver a full-length functional ABCA4 gene to photoreceptor cells. There are currently no approved therapies for Stargardt disease; the only approved retinal gene therapy, voretigene neparvovec (Luxturna), targets RPE65-associated retinal dystrophy rather than ABCA4 mutations. Cross-trial comparisons are limited by differences in study design, duration, and patient populations.
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