ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease

• VeonGen advances two proprietary, first-in-class gene therapies in the clinic, enabled by its novel vgRNA REVeRT and vgAAV technology platforms
• Lead program VG801 receives FDA Rare Pediatric Disease Designation and is progressing in a first-in-human Phase 1/2 trial for Stargardt disease, with functional endpoint development underway through the FDA’s RDEA program

MUNICH, June 05, 2025 (GLOBE NEWSWIRE) -- VeonGen Therapeutics, formerly known as ViGeneron, today announced its rebranding to reflect its evolution into a clinical-stage genetic medicine company focused on developing transformative gene therapies for patients with high unmet medical needs.

VeonGen has advanced two first-in-class gene therapy programs into clinical development, marking a significant step toward addressing diseases with no 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 intravitreally delivered AAV gene therapy for retinitis pigmentosa caused by CNGA1 mutations. Both programs are enabled by VeonGen’s proprietary genetic medicine platforms, designed to address critical gene delivery challenges in AAV-based therapies: vgRNA REVeRT, which utilizes mRNA trans-splicing to deliver large genes exceeding the AAV cargo limit of 4.7 kb; and vgAAV, an engineered capsid platform optimized for safe and efficient delivery via intravitreal or subretinal administration.

VeonGen also announced that its lead program VG801 has received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) for the treatment of ABCA4 mutation-associated retinal dystrophy. The investigational therapy is currently being evaluated in a first-in-human Phase 1/2 clinical trial, with patient dosing underway. Concurrently, VeonGen is developing a functional endpoint in collaboration with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program. VG801 aims to treat patients with biallelic ABCA4 mutations linked to Stargardt disease and related retinal dystrophies - the most common inherited retinal disease, affecting about 1 in 8,000 to 10,000 people worldwide, and a leading cause of macular degeneration in children and young adults.

“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,” said Dr. Caroline Man Xu, Co-founder & Chief Executive Officer 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.”

VeonGen’s proprietary gene therapy platforms are designed for broad therapeutic applicability. 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 developing next-generation gene therapies for patients with high unmet medical needs. The company’s proprietary ophthalmology pipeline features two first-in-class clinical programs: VG801, a dual AAV gene therapy that leverages 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 delivered AAV gene therapy based on the vgAAV platform targeting retinitis pigmentosa caused by CNGA1 mutations.

VeonGen’s three differentiated technology platforms serve as a robust toolbox designed to address critical gene delivery challenges and unmet needs associated with AAV-based therapies. The vgAAV platform features engineered AAV capsids with enhanced transduction efficiency and the ability to overcome biological barriers, enabling less invasive delivery routes such as intravitreal and systemic administration. The vgRNA REVeRT (Reconstitution via mRNA Trans-splicing) platform enables 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, leverages CRISPR/Cas-mediated transcriptional regulation to enable in vivo activation or repression of disease-relevant genes—extending the potential of AAV-based therapies beyond gene supplementation and enabling novel applications in genetic diagnostics.

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

For more information, please visit www.veongen.com

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