K2B261

K2B261 is a preclinical-stage recombinant enzyme replacement therapy (ERT) being developed by Key2Brain in collaboration with Chiesi Group for the treatment of Mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome. The therapy leverages Key2Brain's proprietary BrainBlood platform, which utilizes a peptide shuttle to facilitate the transport of the enzyme alpha-L-iduronidase (IDUA) across the blood-brain barrier (BBB) via receptor-mediated transcytosis. This approach is specifically designed to address the central nervous system (CNS) manifestations of MPS I, such as cognitive decline and neurological impairment, which are not effectively managed by standard systemic ERTs that cannot cross the BBB. The partnership between Chiesi and Key2Brain aims to advance K2B261 as a potential solution for the significant unmet medical need in lysosomal storage disorders with neurological involvement.

Other names

IDUA-BBB shuttlerecombinant human alpha-L-iduronidase fusion protein