VeonGen Therapeutics announced that its investigational gene therapy, VG801, has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA). The therapy is being developed to treat Stargardt disease and other retinal dystrophies caused by mutations in the ABCA4 gene.

VG801 delivers a full-length, functional ABCA4 gene through a dual-AAV approach designed to restore photoreceptor cell function. The therapy is now in a phase 1/2 clinical trial, with patient dosing underway.

The RMAT designation allows for closer collaboration with the FDA, including opportunities for accelerated clinical and regulatory review. The designation joins Rare Pediatric Disease and Orphan Drug status already granted to VG801. VeonGen has also been chosen to participate in the FDA’s Rare Disease Endpoint Advancement program, aimed at developing new clinical trial measures for Stargardt disease. RP