Munich, Germany — VeonGen Therapeutics, a clinical-stage genetic medicine company developing next-generation gene therapies, today announced clinical progress of VG801, with an oral presentation at the 2026 Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting, to be held May 3-7 in Denver, Colorado.
VG801, an investigational gene therapy for ABCA4-associated retinal disease, including Stargardt disease, is being evaluated in an ongoing Phase 1/2 first-in-human clinical trial assessing safety, tolerability, and preliminary efficacy (NCT07002398). Both adult and pediatric patients have been dosed. Of these, nine have completed six-month follow-up, and some have reached twelve months. VG801 has been well tolerated, with no dose-limiting or serious adverse events reported to date.
Preliminary efficacy data show consistent functional improvements in best-corrected visual acuity (BCVA) and a newly developed Virtual Reality Visual Test (VRVT), with improvements sustained through six- and twelve-month follow-up. VRVT has been accepted into the FDA Rare Disease Endpoint Advancement (RDEA) Pilot Program, supporting its development as a novel functional vision endpoint. VeonGen has conducted a dedicated VRVT validation study to support its potential use in a future pivotal trial toward a Biologics License Application (BLA) submission.
Clinical data from the ongoing study will be presented in an oral presentation at ARVO 2026 in the “Stem Cell and Gene Therapy” session, highlighting VG801’s scientific and clinical relevance.
VG801 has received key regulatory authorizations, including FDA IND clearance, EMA CTA approval, and China CDE IND approval, and has been granted FDA Regenerative Medicine Advanced Therapy (RMAT) designation, supporting enhanced regulatory interactions and a potential accelerated development pathway.
“We are encouraged by VG801’s clinical progress and its upcoming oral presentation at ARVO 2026,” said Dr. Caroline Man Xu, Co-founder and Chief Executive Officer of VeonGen Therapeutics. “We continue to engage with regulatory authorities, including RDEA and RMAT, to advance VG801 toward a pivotal trial and potential accelerated regulatory submission. Our focus remains on efficiently advancing clinical programs to bring novel therapies to patients with high unmet medical needs, while exploring strategic partnerships to leverage VG801 and our proprietary technology platforms across ophthalmology and other therapeutic areas.”
About VG801
VG801 is a dual-AAV gene therapy in development for the treatment of Stargardt disease and other ABCA4-associated retinal dystrophies. By delivering a full-length functional ABCA4 gene, VG801 has the potential to address the underlying genetic cause of all forms of ABCA4-mutated Stargardt disease. VG801 is enabled by VeonGen’s proprietary vgAAV capsid for efficient photoreceptor transduction and the vgRNA REVeRT large-gene delivery platform, which delivers the full-length functional ABCA4 gene. These differentiated technologies are designed to directly address the underlying Stargardt disease mechanism, with the potential to drive meaningful functional improvements. VG801 has been selected for the U.S. Food and Drug Administration (FDA) Rare Disease Endpoint Advancement (RDEA) pilot program and holds FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease (RPDD), and Orphan Drug Designations (ODD).
About Stargardt Disease
Stargardt disease is the most prevalent inherited retinal disorder, affecting approximately 1 in 8,000–10,000 individuals worldwide. Caused by mutations in the ABCA4 gene, Stargardt disease is characterized by progressive central vision loss, often beginning in childhood or adolescence, leading to a gradual decline in visual acuity and, in some cases, legal blindness. Currently, there are no approved treatments for Stargardt disease, highlighting a critical unmet medical need.
About VeonGen Therapeutics
Headquartered in Munich, Germany, VeonGen Therapeutics is a clinical-stage genetic medicine company developing next-generation gene therapies for patients with high unmet medical needs. The company’s ophthalmology clinical pipeline includes VG801, a dual-AAV gene therapy for Stargardt disease and ABCA4-related retinal disorders, and VG901, an intravitreally delivered AAV gene therapy for retinitis pigmentosa caused by CNGA1 mutations.
VeonGen’s three proprietary technology platforms (vgAAV, vgRNA REVeRT, and AAV Transactivation) form a robust toolbox designed to overcome key challenges in gene therapy by enabling efficient transduction, delivering large genes beyond traditional AAV limits, and regulating transcription of disease-relevant genes. These technology capabilities expand the potential of AAV-based therapies. VeonGen is also exploring applications and partnerships in ophthalmology, cardiovascular, central nervous system (CNS), and other therapeutic areas.
For more information, please visit www.veongen.com.
VeonGen Therapeutics Contact
Dr. Caroline Man Xu
Co-Founder and CEO
[email protected]
VeonGen Therapeutics Media Contact
MC Services AG
Shaun Brown / Julia von Hummel
phone: +49 (0)89 2102280
[email protected]