FDA Grants RMAT Designation to VeonGen’s VG801 for Stargardt Disease
VeonGen Therapeutics announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to VG801, its lead investigational therapy for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies.
VG801 is designed to deliver a full-length, functional ABCA4 gene, targeting the root genetic cause of Stargardt disease across all ABCA4 mutations. The investigational therapy is currently under evaluation in a phase 1/2 clinical trial, with patient dosing underway.
RMAT designation is reserved for regenerative medicine therapies, including gene therapies, that show preliminary clinical evidence of addressing serious or life-threatening diseases with unmet needs. Benefits of RMAT status include:
Increased engagement with the FDA during development
Streamlined clinical and manufacturing guidance
Opportunities for priority review of a biologics license application (BLA)
Accelerated pathways for regulatory review
With this milestone, VG801 now holds RMAT, Rare Pediatric Disease, and Orphan Drug designations. In addition, VeonGen (formerly ViGeneron) said it is collaborating with the FDA under the Rare Disease Endpoint Advancement (RDEA) pilot program, working toward the development of a novel functional endpoint for Stargardt disease.
“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 & Chief Executive Officer 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.”
VG801 is a dual-AAV gene therapy designed to restore full-length ABCA4 expression through VeonGen’s proprietary vgRNA REVeRT platform, coupled with engineered vgAAV capsids for efficient delivery to photoreceptor cells. By targeting the full ABCA4 gene, the therapy has the potential to address all forms of ABCA4-mutated Stargardt disease.