LX103

LX103 is an investigational recombinant adeno-associated virus (rAAV) gene therapy developed for the treatment of X-linked retinoschisis (XLRS). XLRS is a rare genetic retinal disease caused by mutations in the RS1 gene, which encodes the retinoschisin protein necessary for maintaining the structural integrity of the retina. The absence or dysfunction of this protein leads to the splitting of retinal layers (schisis) and progressive vision loss. LX103 is designed to deliver a functional copy of the human RS1 gene directly to the retinal cells via a single unilateral intravitreal (IVT) injection. By restoring the expression of retinoschisin, the therapy aims to repair the retinal structure and improve visual outcomes for patients.