JR-013

JR-013 is a blood-brain barrier (BBB)-penetrating recombinant human acid alpha-glucosidase (GAA) developed by JCR Pharmaceuticals for the treatment of Pompe disease (glycogen storage disease type II). The drug utilizes JCR's proprietary J-Brain Cargo technology, which involves fusing the GAA enzyme to an antibody fragment that targets the transferrin receptor (TfR). This mechanism facilitates receptor-mediated transcytosis across the BBB, allowing the enzyme to reach the central nervous system (CNS) in addition to peripheral tissues. By addressing the lysosomal accumulation of glycogen in the brain and spinal cord, JR-013 aims to treat the neurological and motor symptoms of Pompe disease that are often refractory to standard enzyme replacement therapies, which cannot cross the BBB.