Development of Once-daily Oral Treatment for Maple Syrup Urine Disease

Syntis Bio Inc, Boston MA

Investigators

Abstract

Project Abstract Syntis Bio is advancing SYNT-213, a potentially transformative therapeutic approach for Maple Syrup Urine Disease (MSUD). This once-daily oral therapy combines the innovative gastrointestinal synthetic epithelial lining (SYNT™) with a protease-stable leucine decarboxylase enzyme (CDX-6210) to potentially extend enzyme activity in the small intestine. By targeting both dietary and recirculated leucine, SYNT-213 may offer a paradigm shift in gut-restricted oral enzyme replacement therapies. This project aims to optimize SYNT-213 for robust gastrointestinal adhesion and enzyme stability, with the potential to significantly reduce leucine levels in preclinical MSUD models. The goal is to develop a solid dosage form that may simplify administration, offering a long-term, disease-modifying treatment for MSUD patients and potentially improving clinical outcomes through enhanced dietary flexibility.