JR-479

JR-479 is an investigational recombinant fusion protein designed as a blood-brain barrier (BBB)-penetrating enzyme replacement therapy (ERT) for the treatment of GM2 gangliosidoses, including Tay-Sachs and Sandhoff diseases. It consists of β-Hexosaminidase A (rDNA origin) fused with JCR Pharmaceuticals' proprietary J-Brain Cargo technology, which targets the transferrin receptor to enable efficient delivery across the BBB into the central nervous system. The drug aims to supplement deficient HexA enzyme activity in affected individuals, thereby reducing pathological accumulation of GM2 ganglioside glycolipids in neuronal tissues. Preclinical studies have demonstrated that intravenous administration of JR-479 results in brain distribution, reduction of substrate concentrations in both CNS and peripheral tissues, suppression of neuronal vacuolation, and significant extension of lifespan in animal models with GM2 gangliosidosis[3][4][6][8].