JR-443

JR-443 is a preclinical, brain-penetrant enzyme replacement therapy candidate being developed by JCR Pharmaceuticals for mucopolysaccharidosis (MPS), a group of lysosomal storage disorders caused by deficiencies in specific lysosomal enzymes leading to accumulation of glycosaminoglycans and progressive multi-organ dysfunction.[6][7][11][16] Using JCR’s blood–brain barrier (BBB)-penetrating technology, JR-443 is designed for intravenous administration to deliver a recombinant lysosomal enzyme (a glucuronidase-replacement type therapy) into both peripheral tissues and the central nervous system, aiming to address neurological manifestations that are poorly controlled by conventional ERTs.[6][7][11][16] JR-443 has shown brain delivery and reduction of pathological substrates in animal models after IV dosing, and is currently at a preclinical research and development stage without reported human trials or regulatory approvals.[6][7][16]