GS144

GS144 (also known as GenSci144) is an oral small-molecule inhibitor of the epithelial neutral amino acid transporter B0AT1, encoded by the *SLC6A19* gene. Developed by Changchun GeneScience Pharmaceuticals, GS144 is designed to inhibit the reabsorption of neutral amino acids in both the renal tubules and the intestine. By blocking SLC6A19, GS144 increases the urinary excretion of neutral amino acids, including phenylalanine, thereby reducing plasma phenylalanine levels in patients with phenylketonuria (PKU) independently of residual phenylalanine hydroxylase (PAH) activity. Additionally, in chronic kidney disease (CKD), GS144 alleviates the metabolic and reabsorptive burden on proximal tubules, mitigating renal inflammation, fibrosis, and tubular necrosis. GS144 has received IND approvals in China for both PKU and CKD and is currently in Phase I clinical development.