JR-162

**JR-162** is a investigational enzyme replacement therapy (ERT) developed by JCR Pharmaceuticals for **Pompe disease**, a lysosomal storage disorder caused by acid alpha-glucosidase (GAA) deficiency leading to glycogen accumulation in muscles and central nervous system (CNS) tissues. It is a fusion protein of an anti-human transferrin receptor antibody (J-Brain Cargo® technology) and recombinant human GAA (rhGAA), enabling enhanced delivery to muscle tissues via mannose-6-phosphate (M6P) and transferrin receptors (TfR), as well as CNS penetration across the blood-brain barrier (BBB) through receptor-mediated transcytosis. Preclinical studies in GAA knockout mice demonstrated significant glycogen reduction in brain, superior efficacy in type II dominant muscles (quadriceps, tibialis anterior, diaphragm) compared to standard rhGAA, suppression of CNS histopathological changes, and potential benefits for both myogenic and neurogenic myopathies.[1][2][4][5][10]