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

MUNICH, June 05, 2025 (GLOBE NEWSWIRE)   ViGeneron has officially rebranded to VeonGen Therapeutics, marking its evolution into a clinical-stage genetic medicine company. This new identity reflects its sharpened focus on developing transformative gene therapies, leveraging adeno-associated virus (AAV) technology, for patients with high unmet medical needs.

In a significant development, VeonGen Therapeutics announced that its lead program, VG801, has been granted Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration (FDA). VG801 is an innovative dual AAV gene therapy, designed to treat ABCA4 mutation-associated retinal dystrophy, encompassing conditions like Stargardt disease. This advanced AAV gene therapy employs mRNA trans-splicing via the vgRNA REVeRT and vgAAV platforms to deliver the crucial full-length ABCA4 gene. Patient dosing is currently underway in a first-in-human phase 1/2 clinical trial for this AAV gene therapy. VeonGen is also working closely with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program to establish a robust functional endpoint for this AAV-based treatment.

Earlier this year, VG901, another promising AAV gene therapy from VeonGen, also received RPDD. VG901 is an intravitreally delivered AAV gene therapy aimed at treating retinitis pigmentosa caused by CNGA1 mutations.

Caroline Man Xu, PhD, co-founder and CEO of VeonGen Therapeutics, highlighted that the rebranding underscores the company’s journey from a platform innovator to a clinical-stage entity with two AAV gene therapies actively progressing in clinical trials. The FDA’s RPDD for VG801 further validates their scientific approach and commitment to accelerating the development and delivery of these transformative AAV therapies for patients in urgent need. The robust translational potential of their AAV platforms positions VeonGen well to expand the frontier of genetic medicine in ophthalmology and beyond, demonstrating the versatility of AAV in gene therapy.