AX-2404

AX-2404 is an investigational RNA editing therapy being developed by ProQR Therapeutics for the treatment of Rett syndrome, a severe neurodevelopmental disorder. The drug utilizes ProQR's proprietary Axiomer RNA editing technology to specifically target the MECP2 R255X nonsense mutation. By employing a synthetic editing oligonucleotide (EON), AX-2404 recruits endogenous ADAR (Adenosine Deaminase Acting on RNA) enzymes to the target Methyl CpG binding protein 2 (MECP2) mRNA. This process facilitates a site-specific Adenosine-to-Inosine (A-to-I) conversion, which corrects the premature stop codon (UAG to UIG, read as UGG/Tryptophan) and restores the expression of full-length, functional MeCP2 protein. AX-2404 is currently in the discovery stage of development.