AX-4070

AX-4070 is an investigational RNA editing therapy being developed by ProQR Therapeutics for the treatment of a rare central nervous system (CNS) disorder. The drug is based on ProQR's proprietary Axiomer technology, which utilizes synthetic 'editing oligonucleotides' (E-oligos) to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to a specific target RNA sequence. This recruitment enables the precise conversion of adenosine to inosine (A-to-I), which is translated by the cellular machinery as guanosine. This mechanism can be used to correct pathogenic G-to-A mutations or to modify protein function at the transcript level without permanently altering the genome. As of early 2024, AX-4070 is in the discovery phase, and its specific molecular target and the exact CNS indication have not been publicly disclosed.