Drug intelligence / Profile preview

anti-miR-503

Development stage
Preclinical
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
Intravenous
01

Overview

**anti-miR-503** is an antisense oligonucleotide therapeutic designed to inhibit the function of microRNA-503 (miR-503), a small non-coding RNA that regulates gene expression post-transcriptionally. By binding to miR-503, anti-miR-503 prevents this microRNA from repressing its target mRNAs, thereby upregulating those target genes. The inhibition of miR-503 has demonstrated **anticancer efficacy in preclinical studies**, particularly in malignant mesothelioma, where it induces apoptosis and reduces tumor growth. Anti-miR-503 was delivered in vivo using lipid nanoparticle (LNP) systems via intravenous injection, demonstrating an ability to suppress tumor progression through apoptotic mechanisms, independently of tumor cell proliferation. Identified targets of miR-503 relevant to cancer include genes associated with cell proliferation (BTG1, CCNG1, EDG1, TIMP2) and apoptosis. While miR-503 is typically described as a tumor suppressor in some tissues, in mesothelioma it may have an oncogenic role, and its inhibition may reduce tumor properties[2][6].

Other names
anti-miR-503anti-miR503anti-miR 503

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