AX-1005

AX-1005 is an investigational RNA editing therapy being developed by ProQR Therapeutics for the treatment of cardiovascular diseases. It utilizes the proprietary Axiomer technology platform, which employs editing oligonucleotides (EONs) to recruit endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to a specific target RNA sequence. These enzymes mediate a site-specific adenosine-to-inosine (A-to-I) conversion, which the cellular translation machinery interprets as a guanosine (G). This precise nucleotide change can be used to correct disease-causing mutations, modulate protein expression, or alter protein function. AX-1005 is currently in the discovery stage, and while its specific biological target has not been publicly disclosed, it represents a key component of ProQR's expansion into common cardiovascular conditions.