FDA Grants RMAT Designation to VeonGen’s Gene Therapy for Stargardt Disease

VeonGen Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to VG801, the company’s lead investigational therapy targeting Stargardt disease and other ABCA4 mutation–associated retinal dystrophies.

VG801: Gene Therapy Candidate for ABCA4-Related Retinal Dystrophies

VG801 is a dual-AAV gene therapy designed to deliver a full-length, functional ABCA4 gene, directly addressing the underlying genetic cause of Stargardt disease across all known ABCA4 mutations. The investigational candidate is currently being evaluated in a phase 1/2 clinical trial, with patient dosing underway.

The therapy leverages VeonGen’s proprietary vgRNA REVeRT platform in combination with engineered vgAAV capsids, developed for targeted delivery and high efficiency in photoreceptor cells.

RMAT Designation Granted for VG801

The RMAT designation is granted to regenerative medicine therapies, including gene therapies, that demonstrate preliminary clinical evidence of potentially addressing serious or life-threatening diseases with unmet medical needs. RMAT status offers several regulatory advantages, including:

       • Enhanced interaction with the FDA during development

       • Streamlined clinical and manufacturing guidance

       • Eligibility for priority review of a Biologics License Application (BLA)

       • Accelerated regulatory review pathways

With the RMAT designation, VG801 now holds three FDA designations including RMAT, Rare Pediatric Disease and Orphan Drug.

Broader Regulatory Collaboration

VeonGen also announced its collaboration with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program. The partnership aims to support the development of a novel functional endpoint specifically for Stargardt disease, further aligning regulatory pathways with patient-centered clinical goals.

Expert Commentary

“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 Caroline Man Xu, PhD, Co-founder & CEO of VeonGen Therapeutics. “This recognition highlights the promise of our novel vgRNA REVeRT and vgAAV platforms and provides an opportunity to accelerate VG801’s development and deliver a much-needed therapy to patients as quickly and efficiently as possible.”