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ATR-1072 is an antibody oligonucleotide conjugate (AOC) therapeutic developed by Atrium Therapeutics for the treatment of PRKAG2 syndrome, a rare genetic cardiomyopathy. The drug consists of a monoclonal antibody conjugated to a small interfering RNA (siRNA) payload, utilizing the AOC platform to achieve tissue-selective delivery directly to the heart. ATR-1072 works through a sequence-specific gene silencing mechanism to reduce the expression of PRKAG2 mRNA. By lowering PRKAG2 levels, the therapeutic aims to normalize AMP-activated protein kinase (AMPK) activity and reduce the pathogenic accumulation of glycogen in cardiac tissue, with the ultimate goal of improving heart function. ATR-1072 is currently in IND-enabling studies, with an investigational new drug (IND) filing anticipated in the second half of 2026.
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