ATX-01

ATX-01 is a first-in-class antimiR oligonucleotide (antisense oligonucleotide) designed to target microRNA 23b (miR-23b). It is a synthetic single-stranded oligonucleotide of 16 nucleotides in length, linked to a hydrophobic conjugate for enhanced tissue delivery. The drug’s dual mechanism of action involves inhibiting miR-23b to both reduce toxic DMPK mRNA foci and increase muscleblind-like (MBNL) protein levels. This approach addresses the molecular underpinnings of myotonic dystrophy type 1 (DM1), a rare neuromuscular disorder characterized by muscle weakness and multisystem complications. By targeting miR-23b, ATX-01 aims to correct mis-splicing events caused by MBNL loss-of-function and reduce the toxic RNA burden in affected tissues such as muscle, heart, and nervous system. It is administered intravenously as an infusion solution. Developed by ARTHEx Biotech for DM1, it has received orphan drug designation from US and European authorities[1][2][3][4][5][6][7].

Other names

antimiR-23bantimiR23bantimiR 23bantagomiR-23b-v1antagomiR23b-v1antagomiR 23b-v1antagomiR-23b-v2antagomiR23b-v2antagomiR 23b-v2