Drug intelligence / Profile preview

agomiR-323a

Development stage
Preclinical
Lead developer
RiboBio
Modality
MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intratumoral, Intravenous
01

Overview

agomiR-323a is a synthetic, chemically modified double-stranded microRNA mimic of miR-323a, designed for enhanced stability and cellular uptake in vivo. It typically incorporates modifications such as 2'-O-methyl (2'-OMe) ribose substitutions and cholesterol conjugation to facilitate delivery without the need for transfection reagents. In pancreatic ductal adenocarcinoma (PDAC), agomiR-323a acts as a tumor suppressor by directly targeting the 3' untranslated region (UTR) of Hexokinase 2 (HK-2) mRNA. By downregulating HK-2 expression, agomiR-323a inhibits the glycolytic pathway (Warburg effect), leading to reduced glucose uptake, decreased lactate and ATP production, and the suppression of cancer cell proliferation and tumor growth. Preclinical studies in xenograft mouse models have demonstrated its potential to significantly reduce tumor volume and metabolic activity.

Other names
miR-323a mimicmiR323a mimicmiR 323a mimicmicroRNA-323a mimicmicroRNA323a mimicmicroRNA 323a mimichsa-miR-323a mimichsa-miR323a mimichsa-miR 323a mimic
02

Targets

HK2 (Hexokinase Type II)

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