AX-2405

AX-2405 is an RNA editing therapeutic candidate developed by ProQR Therapeutics for the treatment of Rett Syndrome. It specifically targets the MECP2 R294X mutation, a nonsense mutation that results in a premature stop codon and subsequent loss of functional Methyl-CpG Binding Protein 2 (MECP2). Utilizing ProQR's proprietary Axiomer platform, AX-2405 consists of an editing oligonucleotide (EON) that recruits endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to the target mRNA. This recruitment facilitates a site-specific adenosine-to-inosine (A-to-I) conversion, which the cellular machinery reads as guanosine, thereby correcting the stop codon and allowing for the translation of full-length, functional MECP2 protein. The program is currently in the discovery phase and aims to address a mutation found in approximately 6% of individuals with typical Rett Syndrome.