AAV8 ELOVL2 gene therapy

AAV8 ELOVL2 gene therapy is an investigational gene therapy designed to deliver the human ELOVL2 gene to retinal cells using an adeno-associated virus serotype 8 (AAV8) vector. The primary mechanism involves transference and expression of the ELOVL2 gene, which encodes an enzyme critical for the biosynthesis of very long-chain polyunsaturated fatty acids (VLC-PUFAs) in the retina. Increasing retinal levels of VLC-PUFAs is believed to support photoreceptor function and may slow or prevent degeneration associated with dry age-related macular degeneration (dry AMD). The therapy is being developed by Visgenx, a biotechnology company focused on treatments for retinal diseases. As of early 2025, this program remains in preclinical development for dry AMD[1][6][8][2].