AX-3875

AX-3875 is an investigational RNA editing therapy being developed by ProQR Therapeutics for the treatment of rare metabolic and central nervous system (CNS) disorders. The drug is part of ProQR's proprietary Axiomer platform, which utilizes synthetic RNA strands called editing oligonucleotides (EONs) to recruit the body's endogenous Adenosine Deaminase Acting on RNA (ADAR) enzyme. Once recruited, ADAR performs a precise adenosine-to-inosine (A-to-I) conversion at a specific site on the target RNA. Because the cell's translation machinery interprets inosine as guanosine, this process can effectively correct G-to-A mutations or otherwise modulate protein expression and function without the permanent genomic changes associated with DNA editing. As of early 2024, AX-3875 is in the discovery stage, and its specific molecular target and primary disease indication have not yet been publicly disclosed.