EQS-News: VeonGen Therapeutics
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VeonGen Announces FDA Alignment on Pivotal Phase 3 Development and Clinical Progress of VG801 for Stargardt Disease
Munich, Germany, November 5, 2026 -- VeonGen Therapeutics, a clinical-stage genetic medicine company developing next-generation gene therapies, today announced significant regulatory and clinical progress for VG801, its investigational dual-AAV gene therapy for Stargardt disease and other ABCA4-associated retinal dystrophies. VeonGen reached alignment with the U.S. Food and Drug Administration (FDA) at a Regenerative Medicine Advanced Therapy (RMAT) Type B meeting on key clinical and Chemistry, Manufacturing and Controls (CMC) aspects of the VG801 pivotal Phase 3 program. The FDA meeting minutes document alignment on the pivotal study design, including the primary endpoint, study duration, and control arm. Regarding CMC, FDA alignment includes the use of the existing clinical batch for the pivotal Phase 3 study and key aspects of the commercial manufacturing strategy. These outcomes provide important regulatory clarity and support preparations for global pivotal Phase 3 development. In parallel, VeonGen is advancing the Virtual Reality Visual Test (VRVT) through the FDA's Rare Disease Endpoint Advancement (RDEA) Pilot Program to support its development as a functional vision measure in Stargardt disease. VG801 has also received Phase 1/2 clinical trial authorizations in Europe and China, supporting its global development strategy. The ongoing first-in-human Phase 1/2 clinical study (NCT07002398) has dosed 15 patients across three dose cohorts, including adults and pediatric patients. VG801 has demonstrated consistent and sustained functional improvements through Month 12 across multiple assessments, including best-corrected visual acuity (BCVA), VRVT and microperimetry fixation. Among the 13 patients with measurable baseline BCVA, all demonstrated improvements from baseline to Month 6 or Month 12, with 11 achieving gains of at least 5 ETDRS letters, including 7 achieving at least 10 letters and 4 achieving at least 15 letters. These findings, together with stable retinal structures and favorable safety profile to date, support VG801's therapeutic potential. "FDA alignment on the pivotal Phase 3 clinical and CMC strategy represents an important de-risking milestone for VG801 and a significant step toward global pivotal development," said Dr. Caroline Man Xu, Co-founder and Chief Executive Officer of VeonGen Therapeutics. "The consistent and sustained functional improvements observed in both adult and pediatric patients reinforce our confidence in the program. Our differentiated technologies are designed to address the underlying genetic cause of Stargardt disease, with the potential to preserve or improve vision for affected patients. We remain focused on advancing VG801 toward global pivotal development and ultimately delivering this innovative therapy to patients worldwide." VG801 leverages VeonGen's proprietary gene therapy platforms, combining its novel vgAAV2 capsid with vgRNA REVeRT (Reconstitution Via mRNA Trans-splicing) to enable delivery and reconstitution of a full-length functional human ABCA4 gene in retinal photoreceptor cells. This differentiated dual-AAV approach is designed to address the underlying genetic cause of Stargardt disease across different ABCA4 mutations, offering the potential for a genotype-agnostic therapeutic approach and functional benefit to patients. About VG801 VG801 is an investigational dual-AAV gene therapy developed for Stargardt disease and other ABCA4-associated retinal dystrophies. It combines VeonGen's proprietary vgAAV capsid technology for efficient photoreceptor transduction and vgRNA REVeRT large-gene delivery technology to deliver and reconstitute a full-length functional human ABCA4 gene in retinal photoreceptor cells. VG801 has received FDA Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease (RPD), and Orphan Drug (ODD) designations, and has been selected for the FDA's Rare Disease Endpoint Advancement (RDEA) pilot program. Stargardt disease is one of the most common inherited macular dystrophies, 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. There are currently no approved treatments for Stargardt disease, highlighting a significant unmet medical need. Headquartered in Munich, Germany, VeonGen Therapeutics is a clinical-stage genetic medicine company developing innovative therapies that address the underlying causes of disease. Its ophthalmology clinical pipeline includes VG801 for Stargardt disease and ABCA4-associated retinal dystrophies, and VG901, an intravitreal AAV gene therapy for retinitis pigmentosa caused by CNGA1 mutations. VeonGen's three proprietary technology platforms (vgAAV, vgRNA REVeRT, and AAV Transactivation) address key challenges in gene therapy, including cellular transduction, large gene delivery beyond AAV size limits, and regulation of disease-relevant gene expression. 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.
05.11.2026 CET/CEST Dissemination of a Corporate News, transmitted by EQS News - a service of EQS Group. |
2429262 05.11.2026 CET/CEST
