AX-2402

AX-2402 is an investigational RNA-editing therapeutic being developed by ProQR Therapeutics for the treatment of Rett syndrome, specifically targeting individuals with the R270X mutation in the MECP2 gene. The drug utilizes ProQR’s proprietary Axiomer RNA editing platform, which enables targeted correction of disease-causing mutations at the RNA level. By modulating expression and function of methyl-CpG-binding protein 2 (MECP2), a key protein implicated in Rett syndrome pathogenesis, AX-2402 aims to restore normal gene function and address underlying genetic defects. The therapy is currently advancing toward clinical trials with support from the Rett Syndrome Research Trust[1][6][7][9].