MEDICAL NEED

Dysregulation of 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 diseases.

OUR PRODUCT

CNSA-001 is an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products.

DEVELOPMENT

CNSA-001 is being developed as a possible treatment for orphan metabolic diseases associated with defects in the tetrahydrobiopterin biochemical pathways including phenylketonuria and primary BH4 deficiency.

 

 

Developing Medicines to Improve the Lives of Patients with Debilitating Diseases

 

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