MEDICAL NEED

Dysregulation of certain metabolic pathways due to various gene mutations can lead to either shortages of necessary end products, or buildup of toxic constituents, either of which may lead to debilitating disease in an underserved patient population.

OUR PRODUCT

CNSA-001 is an oral formulation of synthetic sepiapterin, a precursor to the BH4 enzyme, which is critical in the synthesis of numerous metabolic products.

DEVELOPMENT

CNSA-001 is in development as a possible treatment for various conditions characterized by deficiencies in the tetrahydrobiopterin pathway, including phenylketonuria and primary BH4 deficiency.

 

 

Developing Medicines to Improve the Lives of Patients with Debilitating Diseases

 

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