Federal grant · project grant (b)
Development of Once-daily Oral Treatment for Maple Syrup Urine Disease - 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.
Committed
$343,635
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