K2B246

K2B246 is a preclinical recombinant enzyme replacement therapy (ERT) being developed for the treatment of Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB; MPS IIIB). Developed by Key2Brain in collaboration with Chiesi Group, the drug utilizes the proprietary BrainBlood platform, which consists of a blood-brain barrier (BBB) shuttle peptide fused to the therapeutic enzyme alpha-N-acetylglucosaminidase (NAGLU). MPS IIIB is a lysosomal storage disorder caused by a deficiency in NAGLU, leading to the toxic accumulation of heparan sulfate in the central nervous system and progressive neurodegeneration. K2B246 is designed to cross the BBB after systemic administration, addressing the significant unmet need for treating the neurological manifestations of the disease that standard ERTs cannot reach.