AAV-FGGY

AAV-FGGY is an adeno-associated virus (AAV) vector-based gene therapy designed to overexpress the FGGY carbohydrate kinase. FGGY is a key enzyme in the glycosylation pathway of alpha-dystroglycan, specifically responsible for converting ribitol to ribitol-5-phosphate (Rbo5P). This metabolite is a critical precursor for the synthesis of CDP-ribitol, which is utilized by fukutin and fukutin-related protein (FKRP) to build the matriglycan chains necessary for muscle cell membrane integrity. In dystroglycanopathies, such as those caused by FKRP mutations (e.g., LGMD2I/R9), alpha-dystroglycan glycosylation is reduced or lost. AAV-FGGY aims to restore this glycosylation by bypassing or supplementing the metabolic pathway, thereby improving muscle pathology and function.