VG801

VG801 is a novel gene therapy designed to treat Stargardt disease and other retinal dystrophies associated with biallelic mutations in the ABCA4 gene. It employs a dual adeno-associated virus (AAV) vector system that leverages mRNA trans-splicing technology (REVeRT platform) to deliver and reconstitute the full-length human native ABCA4 gene, which is too large for single AAV packaging. Each AAV vector carries half of the ABCA4 coding sequence; after delivery, these are trans-spliced at the mRNA level within retinal cells to produce functional full-length ABCA4 protein. The therapy uses VeonGen’s proprietary vgAAV capsid technology for efficient and widespread retinal transduction. VG801 is currently being evaluated in a first-in-human phase 1/2 clinical trial, with ongoing patient dosing, and has received FDA Rare Pediatric Disease Designation for its lead indication[2][3][4][5][6].