AX-0422 is an investigational RNA editing oligonucleotide being developed as a potential therapy for MPS I Hurler syndrome (MPS IH, or Hurler syndrome). It is designed to correct a disease-causing mutation in the IDUA gene.
The IDUA gene provides instructions to make the alpha-L-iduronidase enzyme. This enzyme breaks down complex sugar molecules called glycoaminoglycans (GAGs) in cells. In people living with Hurler syndrome, mutations in the IDUA gene mean that little or no working alpha-L-iduronidase enzyme is produced. Without enough of this enzyme, GAGs cannot be broken down and buildup over time. This causes damage to organs and tissues throughout the body and brain.
AX-0422 is designed to edit the W402X mutation of the RNA copy of the IDUA gene with the aim of helping the body produce a working alpha-L-iduronidase enzyme. By restoring the enzyme’s function, AX-0422 aims to reduce the harmful buildup of GAGs and address the symptoms affecting the body and the brain in people with Hurler syndrome.