ViGeneron Rebrands As VeonGen Therapeutics, Earns FDA Rare Pediatric Disease Designation For VG801 Gene Therapy In Stargardt Disease, And Reports Clinical Progress

VeonGen Therapeutics, previously known as ViGeneron, has announced its rebranding as the company transitions into a clinical-stage genetic medicine developer focused on advancing gene therapies for diseases with significant unmet medical needs. The company has moved two of its gene therapy programs into clinical development. VG801 is a novel dual AAV gene therapy aimed at treating Stargardt disease and other retinal disorders caused by ABCA4 mutations. 

VG901 is designed for retinitis pigmentosa linked to CNGA1 mutations and is delivered through intravitreal injection. Both candidates are based on VeonGen’s proprietary technology platforms, vgRNA REVeRT, which enables the delivery of large genes beyond the 4.7 kb AAV cargo limit using mRNA transsplicing, and vgAAV, a specially engineered capsid system that enhances the safety and efficiency of gene delivery.

VG801 has also received Rare Pediatric Disease Designation (RPDD) from the U.S. FDA for ABCA4 mutation-associated retinal dystrophy. The therapy is currently being tested in a first-in-human Phase 1/2 trial, with patient dosing already in progress. Alongside the trial, VeonGen is collaborating with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program to establish a relevant clinical endpoint for future studies. 

Dr. Caroline Man Xu, Co-founder & Chief Executive Officer of VeonGen Therapeutics, stated, “This rebranding reflects our journey – from a platform innovator to a clinical -stage company with two gene therapies in the clinic. With VG801 and VG901 progressing in clinical trials and our platforms demonstrating robust translational potential, we are well positioned to expand the frontier of genetic medicine in ophthalmology and beyond. The FDA Rare Pediatric Disease Designation for VG801 not only highlights the strength of our scientific approach but also reinforces our focus on accelerating the development and delivery of transformative therapies for patients in urgent need.”

Stargardt disease and similar ABCA4-related retinal dystrophies are the most common inherited retinal diseases globally, affecting around 1 in 8,000 to 10,000 individuals and often leading to vision loss in children and young adults. Beyond ophthalmology, VeonGen is also exploring the broader application of its gene therapy platforms in areas such as cardiovascular and central nervous system disorders and is actively seeking collaborations to expand its pipeline.