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IONIS-DGAT2Rx is an antisense oligonucleotide drug that inhibits diacylglycerol O-acyltransferase 2 (DGAT2), a key enzyme involved in the final step of triglyceride synthesis in the liver. By reducing DGAT2 expression through RNA interference, it decreases hepatic triglyceride production and accumulation. The drug is being developed primarily for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH), also known as metabolic dysfunction-associated steatohepatitis (MASH). Clinical studies have shown that treatment with IONIS-DGAT2Rx leads to significant reductions in liver fat content without increasing blood lipids or causing major safety concerns. The drug uses Ionis Pharmaceuticals' proprietary Ligand Conjugated Antisense (LICA) technology to enhance potency and reduce dosing frequency. It has reached Phase 2 clinical trials for NASH/MASH and type 2 diabetes[1][3][4][5][6].
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