Drug intelligence / Profile preview

anti-miR-33

Development stage
Discontinued
Lead developer
Regulus Therapeutics
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Subcutaneous, Intravenous
01

Overview

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.

Other names
miR-33 inhibitormiR33 inhibitormiR 33 inhibitoranti-microRNA-33anti-microRNA33anti-microRNA 33antagomir-33antagomir33antagomir 33
02

Targets

miR-33b (microRNA-33b)

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