VeonGen’s AAV Gene Therapy for Stargardt Disease Receives FDA RMAT Designation

MUNICH, Germany, August 21, 2025 – VeonGen Therapeutics, a clinical-stage genetic medicine company, announced today that the U.S. Food and Drug Administration (FDA) has granted a Regenerative Medicine Advanced Therapy (RMAT) designation to its lead investigational gene therapy, VG801, for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies.

The RMAT designation is a key milestone that will accelerate the development and review of VG801, a therapy designed to address the underlying genetic cause of all ABCA4 mutations by delivering a full-length, functional ABCA4 gene.

“Receiving RMAT designation is strong recognition of VG801’s therapeutic potential for Stargardt disease, the most common inherited retinal disorder with no approved therapies,” said Dr. Caroline Man Xu, Co-founder & Chief Executive Officer of VeonGen Therapeutics. “This recognition highlights the promise of our novel platforms and provides an opportunity to accelerate VG801’s development and deliver a much-needed therapy to patients as quickly and efficiently as possible.”

VG801 is a dual-AAV gene therapy that utilizes VeonGen’s proprietary vgRNA REVeRT and vgAAV platforms to restore full-length ABCA4 expression. The therapy is currently being evaluated in a Phase I/II clinical trial, with patient dosing already underway.

In addition to the RMAT designation, VG801 has also received Rare Pediatric Disease and Orphan Drug designations. VeonGen has also been selected to collaborate with the FDA under the Rare Disease Endpoint Advancement (RDEA) pilot program.

Stargardt disease, which affects approximately 1 in 8,000 to 10,000 individuals worldwide, is caused by mutations in the ABCA4 gene and leads to progressive central vision loss, often resulting in legal blindness. The lack of approved treatments for this condition underscores the critical unmet medical need that VG801 aims to address.