
JNT-517 is a small-molecule ингибитор of the phenylalanine transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The FDA has granted JNT-517 Rare Pediatric Disease Designation.
JNT-517 is a small-molecule inhibitor of the phenylalanine transporter SLC6A19 that has the potential to be a first-in-class oral therapy used to treat any person with PKU, regardless of age or genotype. JNT-517 acts at a novel, cryptic allosteric site to block kidney reabsorption of Phe and offers a promising new approach to reduce blood Phe levels. The FDA has granted JNT-517 Rare Pediatric Disease Designation.
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