SKG0402

SKG0402 is an investigational adeno-associated virus (AAV) vector-based gene therapy being developed by Skyline Therapeutics for the treatment of Fabry disease. The therapy utilizes Skyline's proprietary capDRIVE® AAV technology platform, which integrates advanced capsid discovery, transgene design, and vector engineering to optimize delivery and expression. SKG0402 is designed to deliver a functional copy of the GLA gene to target tissues, aiming to restore the activity of the enzyme alpha-galactosidase A. This restoration is intended to facilitate the breakdown of globotriaosylceramide (Gb3) and related glycosphingolipids, which systemically accumulate in patients with Fabry disease. Preclinical data presented at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting in 2023 demonstrated the therapy's efficacy in reducing substrate levels in Fabry disease mouse models.