AYX-401

Red cell encapsulated arginase for oncology and arginase deficiency

Blood cells flowing through artery

Red Cell Encapsulated Arginase for Oncology and Arginase Deficiency

Red cell encapsulated arginase for oncology and arginase deficiency

  • Arginase deficiency (argininemia) is a rare, autosomal recessive urea cycle disorder caused by biallelic pathogenic variants in the ARG1 gene. It results in a complete or partial lack of the arginase-1 enzyme, primarily in the liver cytosol and red blood cells.
  • Loargys® is approved in the EU and currently under FDA review. However, it is an injection administered either intravenously or subcutaneously once a week. AYX-401 will allow for less frequent administration with a potentially superior pharmacokinetic and pharmacodynamic profile.