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ANG-IDUA is a preclinical-stage peptide-enzyme conjugate being developed by Angiochem for the treatment of Mucopolysaccharidosis type I (MPS I), also known as Hurler syndrome. The therapeutic candidate consists of the enzyme alpha-L-iduronidase (IDUA) conjugated to a proprietary peptide from Angiochem's EPiC platform. This platform is designed to leverage receptor-mediated transcytosis, specifically targeting the Low-density lipoprotein receptor-related protein 1 (LRP1), to facilitate the transport of the enzyme across the blood-brain barrier (BBB). By enabling the enzyme to reach the central nervous system, ANG-IDUA aims to address the cognitive and neurological decline associated with MPS I, which is a significant limitation of current standard-of-care enzyme replacement therapies that do not effectively penetrate the brain.
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