AX-0811

AX-0811 is a next-generation RNA-editing oligonucleotide (EON) developed by ProQR Therapeutics for the treatment of cholestatic liver diseases. Utilizing the proprietary Axiomer platform, AX-0811 is designed to target the Sodium/taurocholate cotransporting polypeptide (NTCP), a key transporter responsible for the uptake of bile acids from the bloodstream into the liver. The therapy employs endogenous Adenosine Deaminase Acting on RNA (ADAR) enzymes to perform site-specific A-to-I (Adenosine-to-Inosine) base editing on the target RNA. This modification is intended to modulate NTCP function and reduce the accumulation of toxic bile acids within hepatocytes, thereby preventing liver damage. AX-0811 was generated using an AI-enabled discovery engine to optimize editing performance, demonstrating multifold higher editing efficiency in vivo at lower dose levels compared to earlier candidates. As of April 2026, the program is in the IND-enabling phase, with a Clinical Trial Application (CTA) filing planned for mid-2026.