ABXC-73

ABXC-73 is a novel CD71 Centyrin:Gys1 siRNA conjugate designed to treat Pompe disease. It functions by utilizing a Centyrin protein to target CD71 (transferrin receptor type 1, TfR1) for cellular internalization. Once inside the cell, the conjugated Gys1 siRNA inhibits the expression and enzymatic activity of glycogen synthase 1 (GYS1), a key enzyme in glycogen synthesis. This mechanism aims to restore glycogen balance by reducing glycogen levels in muscle, as demonstrated in a mouse model of Pompe disease where it also improved exercise performance. Unlike existing enzyme replacement therapies (ERTs) that degrade aberrant glycogen, ABXC-73 focuses on inhibiting glycogen synthesis.