ViGeneron Rebrands as VeonGen Therapeutics; Announces FDA Rare Pediatric Disease Designation for Lead Gene Therapy
VeonGen Therapeutics announced its rebranding from ViGeneron 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. 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, which are designed to address critical gene delivery challenges in AAV-based therapies: vgRNA REVeRT. The platforms utilize 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 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.
“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,” Dr. Caroline Man Xu, Co-founder & CEO of VeonGen Therapeutics, said in a company news release. “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.