AX-0601

AX-0601 is an investigational RNA editing therapy being developed by ProQR Therapeutics for the treatment of metabolic diseases, specifically obesity and type 2 diabetes. The program utilizes the proprietary Axiomer platform, which employs synthetic editing oligonucleotides (EONs) to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to specific RNA transcripts. These enzymes catalyze the conversion of adenosine to inosine (A-to-I), which the cellular translation machinery subsequently reads as guanosine. This precise editing capability allows for the modulation of protein function or expression to address underlying disease drivers without permanently altering the genome. AX-0601 is currently in the discovery phase of development.