Arginine Reducing Therapies

Arginine Reducing Therapies – Target Pipeline List 04/2019

Targets: Arginine

This Target Pipeline List provides an overview of therapeutic proteins in development for treatment of arginine-dependent tumors or for enzyme replacement therapy.

Amino acid deprivation is a validated approach for controlling tumor growth. Arginine is an essential amino acid to many tumor types, however, many of these malignancies lack the relevant metabolic enzymes, such as argininosuccinate synthetase (ASS) and/or ornithine transcarbamylase (OTC), to produce arginine. These OTC and/or ASS deficient tumors are therefore completely dependent upon external sources of arginine, making arginine deprivation therapy an attractive strategy for tumor treatment.

Arginase 1 Deficiency is a rare genetic disorder caused by a mutation in the Arginase 1 gene that leads to the inability to degrade arginine. This results in excessively high levels of arginine in the blood, referred to as hyperargininemia. Replacement of the Arginase 1 missing in patients and reduce elevated blood arginine levels to the normal physiological range. Normalization of arginine levels is anticipated to slow or halt the progression of disease in these patients.

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