AX-0810 + AX-0811

AX-0810 and AX-0811 are investigational ADAR-mediated RNA editing oligonucleotides (EONs) developed by ProQR Therapeutics. Both compounds are built on ProQR's proprietary Axiomer RNA editing technology platform, which recruits endogenous ADAR (adenosine deaminase acting on RNA) enzymes to make targeted single-nucleotide edits in RNA. Specifically, they target Na-taurocholate cotransporting polypeptide (NTCP, encoded by the SLC10A1 gene), a transport protein on hepatocytes that mediates the uptake of bile acids from the blood into the liver. By editing NTCP RNA to introduce a loss-of-function variant (NTCP Q68R), these therapies aim to reduce toxic bile acid reuptake and accumulation in hepatocytes, thereby mitigating inflammation, fibrosis, and progression to liver failure in cholestatic liver diseases such as biliary atresia and primary sclerosing cholangitis. AX-0810 is the first-generation lead candidate and has demonstrated positive target engagement and safety in a Phase 1 trial in healthy volunteers. AX-0811 is a next-generation candidate designed using ProQR's AI-enabled discovery engine, offering higher potency and longer durability, with a Phase 1 trial initiated in 2026.

Other names

AX-0810 and AX-0811AX0810 and AX-0811AX 0810 and AX-0811AX-0810/AX-0811