Robust preclinical [37,58,59,60,61] and clinical [62,63,64,65] evidence shows that it exhibits unequivocal dual efficacy against peripheral/somatic and CNS manifestations in patients with genetic IDS deficiency by delivering the enzyme therapeutic via TfR-mediated transcytosis across the BBB (a proprietary technology named J-Brain Cargo®). Here, TFRC is linked to mucopolysaccharidosis type 2.