rAAV2-OPA1

rAAV2-OPA1 is an investigational gene therapy designed to treat Dominant Optic Atrophy (DOA), a hereditary mitochondrial disease primarily caused by mutations in the OPA1 gene. The therapy uses a recombinant adeno-associated virus serotype 2 (rAAV2) vector to deliver a functional copy of the human OPA1 gene directly into retinal ganglion cells (RGCs) via intravitreal injection. By restoring OPA1 protein expression, the treatment aims to rescue mitochondrial function, prevent RGC degeneration, and slow or halt vision loss associated with DOA. Preclinical studies have demonstrated that AAV-mediated delivery of OPA1 can protect visual function and improve mitochondrial bioenergetics in relevant animal models and patient-derived cells[1][3][5]. Clinical development is ongoing for autosomal dominant optic atrophy due to OPA1 mutation[3].