AX-2911 (ProQR Therapeutics)

AX-2911 (formerly known as PNPLA3-targeting editing oligonucleotide) is a single-stranded, GalNAc-conjugated RNA editing oligonucleotide (EON) developed by ProQR Therapeutics using its proprietary Axiomer technology platform. It is designed to treat metabolic dysfunction-associated steatohepatitis (MASH) by targeting the PNPLA3 I148M (rs738409) genetic variant, which is a major driver of hepatic steatosis and disease progression. AX-2911 recruits endogenous ADAR (Adenosine Deaminase Acting on RNA) enzymes to perform a site-specific adenosine-to-inosine (A-to-I) base edit on the mutant PNPLA3 mRNA, converting the pathogenic methionine codon (ATG) to a valine codon (GTG). This results in the expression of a functional, wildtype-like PNPLA3 I148V variant, restoring normal lipid homeostasis and reducing hepatic fat accumulation without silencing the PNPLA3 protein.

Other names

Axiomer mediated RNA editing PNPLA3PNPLA3-targeting editing oligonucleotidePNPLA-3-targeting editing oligonucleotidePNPLA 3-targeting editing oligonucleotide