GYS1 ASO#2

GYS1 ASO#2 is an experimental antisense oligonucleotide (ASO) designed for the treatment of Pompe disease through substrate reduction therapy. It specifically targets the mRNA of glycogen synthase 1 (GYS1), the enzyme responsible for glycogen synthesis in skeletal muscle, to induce its degradation via an RNase H-mediated mechanism. By reducing GYS1 expression, the drug aims to curb the aberrant accumulation of glycogen that characterizes Pompe disease, particularly in skeletal muscles where standard enzyme replacement therapy (ERT) often shows limited efficacy. Preclinical studies in Gaa-/- mouse models have demonstrated that GYS1 ASO#2 can significantly reduce muscle glycogen content, eliminate autophagic buildup, and improve motor function, especially when administered in combination with recombinant human acid alpha-glucosidase (GAA).

Other names

Gys1 ASOGys-1 ASOGys 1 ASO