• VeonGen is advancing two innovative, first-in-class gene therapies in clinical trials, utilizing its unique vgRNA REVeRT and vgAAV technology platforms.
• Its leading program, VG801, has been granted FDA Rare Pediatric Disease Designation and is currently in a Phase 1/2 clinical trial for Stargardt disease, with the development of functional endpoints ongoing through the FDA’s RDEA program.

MUNICH, June 05, 2025 — VeonGen Therapeutics, previously known as ViGeneron, announced today its rebranding to reflect its transformation into a clinical-stage genetic medicine company. The company is focused on creating innovative gene therapies for patients with significant unmet medical needs.

VeonGen has progressed two novel gene therapy programs to the clinical stage, representing a significant advancement in addressing diseases that lack approved treatment options. These programs include VG801, a novel dual AAV gene therapy for Stargardt disease and other ABCA4-related retinal disorders, and VG901, an AAV gene therapy delivered via intravitreal injection for retinitis pigmentosa caused by CNGA1 mutations. Both programs leverage VeonGen’s exclusive genetic medicine platforms, designed to overcome key gene delivery challenges in AAV-based therapies: vgRNA REVeRT, which employs mRNA trans-splicing to deliver large genes exceeding the AAV capacity of 4.7 kb; and vgAAV, an engineered capsid platform optimized for safe and efficient delivery through intravitreal or subretinal administration.

VeonGen also announced that VG801, its primary program, has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA) for treating ABCA4 mutation-associated retinal dystrophy. The investigational therapy is currently being assessed in an initial Phase 1/2 clinical trial in humans, with patient dosing underway. Simultaneously, VeonGen is developing a functional endpoint in collaboration with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program. VG801 is intended to treat patients with biallelic ABCA4 mutations associated with Stargardt disease and related retinal dystrophies – the most prevalent inherited retinal disease, affecting approximately 1 in 8,000 to 10,000 individuals globally and a primary cause of macular degeneration in children and young adults.

“This rebranding showcases our evolution from a platform innovator to a clinical-stage company with two gene therapies undergoing clinical evaluation. With VG801 and VG901 advancing in clinical trials and our platforms demonstrating strong translational potential, we are well-positioned to push the boundaries of genetic medicine in ophthalmology and beyond,” stated Dr. Caroline Man Xu, Co-founder & Chief Executive Officer of VeonGen Therapeutics. “The FDA Rare Pediatric Disease Designation for VG801 underscores the strength of our scientific approach and reinforces our commitment to accelerating the development and delivery of transformative therapies for patients with urgent needs.”

VeonGen’s exclusive gene therapy platforms are designed for broad therapeutic applications. In addition to ophthalmology, the company is exploring expansion and partnerships in cardiovascular, central nervous system (CNS), and other disease areas.

About VeonGen Therapeutics

VeonGen Therapeutics is a clinical-stage genetic medicine company focused on developing next-generation gene therapies for patients facing significant unmet medical needs. The company’s proprietary ophthalmology pipeline includes two first-in-class clinical programs: VG801, a dual AAV gene therapy that utilizes mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the full-length ABCA4 gene for Stargardt disease and related retinal disorders; and VG901, an intravitreally administered AAV gene therapy based on the vgAAV platform targeting retinitis pigmentosa caused by CNGA1 mutations.

VeonGen’s three distinct technology platforms provide a comprehensive toolkit designed to address critical gene delivery challenges and unmet needs related to AAV-based therapies. The vgAAV platform features engineered AAV capsids with improved transduction efficiency and the ability to overcome biological barriers, enabling less invasive delivery methods such as intravitreal and systemic administration. The vgRNA REVeRT (Reconstitution via mRNA Trans-splicing) platform facilitates the delivery and reconstitution of large genes—exceeding the 4.7 kb AAV cargo limit—at the mRNA level in tissues targeted by a selected AAV capsid. The third platform, AAV Transactivation, uses CRISPR/Cas-mediated transcriptional regulation to enable in vivo activation or repression of disease-relevant genes—expanding the potential of AAV-based therapies beyond gene supplementation and enabling new applications in genetic diagnostics.

With significant potential for applications beyond ophthalmology, VeonGen is also pursuing expansion and partnerships in cardiovascular, central nervous system (CNS), and other disease areas. The company was established in 2017 by a team with extensive expertise in AAV vector engineering, gene therapy development, and clinical translation. VeonGen is headquartered in Munich, Germany.

For more information, please visit

“`