Drug intelligence / Profile preview

folate-conjugated microRNA-34a

Development stage
Preclinical
Lead developer
Purdue University
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
Intravenous
01

Overview

Folate-conjugated microRNA-34a is an experimental **ligand-conjugated oligonucleotide therapeutic** in which a synthetic miR-34a mimic is directly conjugated to folate to enable receptor-mediated delivery into **folate receptor alpha expressing tumor cells**. The agent was developed in academic research, including work by Purdue University investigators, as a tumor-targeted miRNA replacement strategy intended to restore the tumor-suppressive activity of miR-34a, downregulate oncogenic miR-34a target genes, and inhibit tumor growth. Preclinical studies reported selective activity in FOLR1-high cancers including breast and lung cancer, with additional in vitro inhibitory effects in ovarian and cervical cancer models, while prostate cancer activity appeared limited by low target receptor expression. No marketed brand name, clinical approval, or commercial manufacturer has been identified.

Other names
folate + microRNA-34afolate-miR-34afolate-miR34afolate-miR 34aFM-miR-34aFM-miR34aFM-miR 34aFolamiRfolate-conjugated miR-34a
02

Targets

CD44 (CD44 antigen)MYC (MYC proto-oncogene protein)FOLR1 (FRα)CCND1 (Cyclin D1)BCL-2 (BCL-2 family)

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