AX-2403

AX-2403 is an investigational RNA editing therapy being developed by ProQR Therapeutics for the treatment of Rett syndrome, specifically targeting patients with the MECP2 R168X mutation. The therapy utilizes ProQR's proprietary Axiomer RNA editing platform, which involves the use of editing oligonucleotides (EONs) to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to a specific target site. By mediating a single nucleotide change (adenosine-to-inosine editing), AX-2403 aims to correct the R168X nonsense mutation in the MECP2 mRNA, thereby restoring the production of functional MeCP2 protein. This program is currently in the discovery phase and is supported by the Rett Syndrome Research Trust (RSRT).