The FDA cleared the investigational new drug application for a phase 1/2 study investigating VG801, a treatment for Stargardt disease and other retinal diseases associated with mutations in the ABCA4 gene.
According to a press release from ViGeneron GmbH, this is the first FDA IND clearance for an mRNA trans-splicing gene therapy. VG801 utilizes two gene therapy delivery technology platforms: REVeRT, designed to reconstruct ABCA4 messenger RNA through transduction with dual AAV vectors, and a vgAAV capsid designed to spur widespread retinal transduction.
The phase 1/2 multicenter, open-label,