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

Award Number: R44DK143766
ORGANIZATION: NATIONAL INSTITUTE OF DIABETES & DIGESTIVE & KIDNEY DISEASES
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)
PERIOD OF PERFORMANCE START DATE: 09/10/2025
PERIOD OF PERFORMANCE END DATE: 08/31/2026

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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.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2025 ( Subtotal = $343,635 )
2025	2025	SYNTIS BIO INC	135 MORRISSEY BLVD	BOSTON	MA	02125	SUFFOLK	USA	Diabetes, Digestive, and Kidney Diseases Extramural Research	000	1	9/8/2025	NEW	$343,635
														Subtotal = $343,635

Grand Total All Awards = $343,635

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

Award Number: R44DK143766

ORGANIZATION: NATIONAL INSTITUTE OF DIABETES & DIGESTIVE & KIDNEY DISEASES

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: SCIENTIFIC/HEALTH RESEARCH (INCLUDES SURVEYS)

PERIOD OF PERFORMANCE START DATE: 09/10/2025

PERIOD OF PERFORMANCE END DATE: 08/31/2026

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