Drug intelligence / Profile preview

anti-miR-21

Development stage
Unknown
Lead developer
Regulus Therapeutics
Modality
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
Administration
Subcutaneous, Intravenous
01

Overview

**anti-miR-21** refers to synthetic oligonucleotides (including antagomirs and antisense oligonucleotides) designed to specifically bind and inhibit microRNA-21 (miR-21). miR-21 is frequently upregulated in numerous human diseases, especially cancers (such as multiple myeloma, colorectal cancer, and non-small cell lung cancer), fibrotic kidney disease (Alport syndrome), and heart failure. Mechanistically, anti-miR-21 blocks the function of miR-21 by complementary base pairing, leading to derepression of tumor suppressor genes (PTEN, BTG2, Rho-B, PDCD4, TIMP3, TPM1, maspin, RECK), and thereby downregulates pro-survival signaling such as AKT and ERK pathways[1][4][5][7][15]. This inhibition may result in reduced cell proliferation, reduced fibrosis, increased apoptosis, reversed angiogenesis, and improved organ function, depending on the disease context[1][3][6][4][15]. LNA (locked nucleic acid)-modified and phosphorothioate backbone antisense oligonucleotide agents are commonly used modalities for anti-miR-21[3][4][5]. Clinical candidates using anti-miR-21 mechanisms include RG-012 and Lademirsen, with investigation in Alport syndrome and other fibrotic disorders[5][6][7][14]. No anti-miR-21 agent is currently approved for any indication, but several candidates (not always simply named "anti-miR-21") have reached clinical trials in kidney disease and cancer[5][6][9][14][15].

Other names
anti-microRNA-21anti-microRNA21anti-microRNA 21antimiR-21antimiR21antimiR 21anti–miR-21anti-miR21anti-miR-21anti-miR 21anti-miR21 antagomiranti-miR-21 antagomiranti-miR 21 antagomir
02

Targets

miR-21 (MicroRNA-21)

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