ABO-504

ABO-504 is an investigational gene therapy developed by Abeona Therapeutics for the treatment of Stargardt disease, a form of juvenile macular degeneration. The therapy uses a dual adeno-associated virus (AAV) vector strategy with the Cre-LoxP recombination system to efficiently reconstitute the full-length ABCA4 gene in retinal cells. Mutations in ABCA4 are responsible for Stargardt disease, leading to toxic compound accumulation and progressive vision loss. Preclinical studies have demonstrated that ABO-504 can produce full-length ABCA4 protein in cell culture and express both ABCA4 mRNA and protein in the retina following subretinal administration[1][2][3][4][5][6].