SKG0301

SKG0301 is an adeno-associated virus (AAV) vector-based gene therapy developed by Skyline Therapeutics for the treatment of Pompe disease, a rare metabolic disorder. The therapy utilizes Skyline's proprietary capDRIVE AAV technology platform, which integrates optimized capsid discovery, transgene design, and vector engineering to enhance therapeutic efficacy and safety. SKG0301 is designed to deliver a functional version of the gene encoding acid alpha-glucosidase (GAA), the enzyme deficient in Pompe disease, to restore metabolic function and address the underlying genetic cause of the disorder. Preclinical studies presented at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting demonstrated therapeutic potential in mouse models of Pompe disease.