AZD2693

AZD2693 is an investigational antisense oligonucleotide (ASO) therapeutic designed to selectively lower the expression of patatin-like phospholipase domain-containing 3 (PNPLA3) mRNA in the liver. It specifically targets individuals who are homozygous for the PNPLA3 rs738409 148M risk allele, a genetic variant strongly associated with increased risk and progression of metabolic dysfunction-associated steatohepatitis (MASH), formerly known as non-alcoholic steatohepatitis (NASH). By reducing hepatic PNPLA3 levels, AZD2693 aims to address a key driver of disease pathogenesis in this genetically defined population. Preclinical and early clinical studies have shown that AZD2693 reduces liver fat content and fibrosis markers with an acceptable safety profile. The drug is administered subcutaneously, typically once per month[1][5][6][9].

Other names

AZD2693 sodiumAZD-2693 sodiumAZD 2693 sodium