ViGeneron rebrands as VeonGen Therapeutics, announces rare pediatric disease designation for VG801

ViGeneron has rebranded to VeonGen Therapeutics to reflect its evolution into a “clinical-stage genetic medicine company focused on developing transformative gene therapies for patients with high unmet medical needs.”

In addition to the rebranding, VeonGen also announced the US Food and Drug Administration (FDA) has given Rare Pediatric Disease Designation (RPDD) to its lead program, VG801, for the treatment of ABCA4 mutation-associated retinal dystrophy.

VG801 is 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.

VG801 is currently being evaluated in a first-in-human phase 1/2 clinical trial, with patient dosing ongoing. The company is also developing a functional endpoint in collaboration with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program.

Earlier this year, VeonGen’s VG901 to treat CNGA1-associated retinitis pigmentosa (RP) received RPDD.

Caroline Man Xu, PhD, co-founder and CEO of VeonGen Therapeutics, commented on the rebranding in a press release from the company.

“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 Man Xu. “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.”

In addition to VG801, VeonGen is developing VG901, an intravitreally delivered AAV gene therapy for retinitis pigmentosa caused by CNGA1 mutations. VG901 also received RPDD status at the beginning of 2025.

References:

1. ViGeneron rebrands as VeonGen Therapeutics and announces FDA Rare Pediatric Disease Designation and clinical progress for lead gene therapy VG801 in Stargardt Disease. Published June 5, 2025. Accessed June 5, 2025. https://www.globenewswire.com/news-release/2025/06/05/3094322/0/en/ViGeneron-Rebrands-as-VeonGen-Therapeutics-and-Announces-FDA-Rare-Pediatric-Disease-Designation-and-Clinical-Progress-for-Lead-Gene-Therapy-VG801-in-Stargardt-Disease.html