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OX-NLA is a small molecule drug candidate being developed for the treatment of primary hyperoxaluria. It is designed as a potent inhibitor of glycolate oxidase (GO), an enzyme in the liver responsible for the production of glyoxylate, which is subsequently converted to oxalate. By inhibiting glycolate oxidase, OX-NLA aims to reduce the overproduction of oxalate, thereby preventing the formation of calcium oxalate crystals in the kidneys and urinary tract, which leads to progressive kidney damage and systemic oxalosis. The drug is intended to provide a therapeutic alternative to existing RNA interference (RNAi) therapies by offering a different modality for substrate reduction therapy in patients with Type 1 primary hyperoxaluria (PH1).
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