ViGeneron Rebrands as VeonGen, Earns FDA RPDD for Gene Therapy

VeonGen Therapeutics has officially announced its rebranding from ViGeneron, marking a strategic transformation into a clinical-stage genetic medicine company dedicated to developing innovative gene therapies for diseases with high unmet medical needs.

Transition to a Clinical-Stage Genetic Medicine Company

The rebranding reflects VeonGen’s evolution from a platform innovator to a company with active clinical programs. It has advanced two first-in-class gene therapy candidates into human trials:

       • VG801: A dual adeno-associated virus (AAV) gene therapy targeting Stargardt disease and other ABCA4-related retinal disorders.

       • VG901: An AAV-based therapy designed for intravitreal delivery to treat retinitis pigmentosa caused by CNGA1 mutations.

Proprietary Platforms Supporting Gene Therapy Development

Both therapies are supported by VeonGen’s proprietary platforms that address the limitations of conventional AAV-based gene delivery:

       • vgRNA REVeRT: Uses mRNA trans-splicing to enable delivery of large genes exceeding the AAV’s 4.7 kb cargo limit.

       • vgAAV: A capsid-engineering platform optimized for safe, efficient gene delivery via intravitreal or subretinal routes.

FDA Rare Pediatric Disease Designation for VG801

VG801, VeonGen’s lead investigational product, has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for the treatment of ABCA4 mutation-associated retinal dystrophy. This novel gene therapy is now being evaluated in a first-in-human Phase 1/2 clinical trial, with patient dosing already in progress.

Simultaneously, VeonGen is collaborating with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program to develop a functional clinical endpoint. VG801 is aimed at treating patients with biallelic ABCA4 mutations associated with Stargardt disease and related inherited retinal dystrophies.

Strategic Vision and Future Expansion

“This rebranding reflects our journey—from a platform innovator to a clinical-stage company with two gene therapies in the clinic,” said Dr. Caroline Man Xu, Co-founder & CEO of VeonGen Therapeutics. “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.”

Beyond ophthalmology, VeonGen is also exploring therapeutic applications and strategic partnerships in cardiovascular, central nervous system (CNS), and other high-impact disease areas.