ABO-505

ABO-505 is an investigational gene therapy designed to treat autosomal dominant optic atrophy (ADOA), a hereditary form of vision loss primarily caused by mutations in the Opa1 gene. The therapy uses an adeno-associated virus (AAV204) vector to deliver a functional copy of the human Opa1 gene directly into the retina via para-retinal injection. This approach targets retinal ganglion cells and the optic nerve, which are most affected in ADOA. Preclinical studies have demonstrated that ABO-505 leads to robust expression of Opa1 at both RNA and protein levels in cell culture and animal models, resulting in improved retinal signaling and visual acuity in treated mutant mice. Developed by Abeona Therapeutics.