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PBAS499 is a preclinical-stage small molecule being developed by Pluvia Biotech for the treatment of Phenylketonuria (PKU) and Hyperphenylalaninemia (HPA). The drug acts by targeting Phenylalanine hydroxylase (PAH), the enzyme responsible for converting phenylalanine to tyrosine. Mutations in the PAH gene lead to the accumulation of phenylalanine, which is toxic to the brain. PBAS499 is designed to stabilize or enhance the activity of the PAH enzyme, thereby reducing blood phenylalanine levels and preventing the neurological complications associated with PKU.
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