ABO-503

ABO-503 is an investigational gene therapy developed for the treatment of X-linked retinoschisis (XLRS), a rare, monogenic retinal disease that leads to irreversible loss of photoreceptor cells and severe visual impairment. XLRS is caused by mutations in the RS1 protein, which mediates cell-cell adhesion in the retina. ABO-503 consists of a functional human RS1 gene packaged within Abeona Therapeutics' proprietary AIM capsid AAV204. Preclinical studies have demonstrated that delivery of ABO-503 to the retina results in robust RS1 expression, improved cone photoreceptor density, increased thickness of the photoreceptor outer nuclear layer, and significant improvement in cone photoreceptor function as measured by electroretinogram analysis[1][2][5][7].