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SYNT-203 is an orally administered, gastrointestinal-stable enzyme replacement therapy being developed for the treatment of maple syrup urine disease (MSUD). The drug consists of a leucine decarboxylase enzyme engineered for stability and performance in the digestive tract. Its mechanism involves breaking down and eliminating excess leucine in the gut to control circulating leucine levels, thereby addressing the metabolic defect underlying MSUD. The therapy is designed to be gut-restricted, minimizing systemic exposure and potential side effects. Developed using Syntis Bio’s DIGEST platform technology, SYNT-203 aims to extend intestinal activity and enable continuous leucine elimination with simplified dosing regimens. Originally developed by Codexis as CDX-62103, it was acquired by Syntis Bio in April 2024[1][2][3][4][8].
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