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Anti-miR-33 is an antisense oligonucleotide therapeutic designed to inhibit microRNA-33 (miR-33a and miR-33b). miR-33 is an intronic microRNA located within the SREBP genes that suppresses the expression of the cholesterol transporter ABCA1, thereby lowering HDL cholesterol levels and impairing reverse cholesterol transport. Developed by Regulus Therapeutics, anti-miR-33 utilizes chemically modified oligonucleotides, specifically 2'-fluoro-methoxyethyl-phosphorothioate (2'F/MOE) modified oligonucleotides, to antagonize miR-33. Preclinical studies in mice and non-human primates (African Green monkeys) demonstrated its ability to increase circulating HDL, promote atherosclerotic plaque regression, and reduce inflammation. Despite promising preclinical data for cardiovascular disease and potential applications in kidney fibrosis, the program did not transition into clinical development and was ultimately not pursued further.
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