AX-2406

AX-2406 is an investigational RNA editing therapeutic being developed by ProQR Therapeutics for the treatment of Rett Syndrome, specifically targeting the MECP2 R133H mutation. Utilizing ProQR's proprietary Axiomer RNA editing platform, AX-2406 employs editing oligonucleotides (EONs) to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to the target mRNA. This recruitment enables a precise adenosine-to-inosine (A-to-I) conversion, correcting the point mutation at the RNA level and restoring the production of functional Methyl-CpG Binding Protein 2 (MeCP2). Rett Syndrome is a severe, progressive neurodevelopmental disorder that primarily affects females and is caused by mutations in the MECP2 gene. AX-2406 is currently in the discovery stage of development.