Drug intelligence / Profile preview

5-fluorouracil modified miRNA mimics

Development stage
Preclinical
Lead developer
Stony Brook University
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous, Intratumoral
01

Overview

5-fluorouracil modified miRNA mimics are a class of experimental, RNA-based therapeutic compounds designed to combine the gene-silencing capabilities of tumor-suppressive microRNAs with the cytotoxic effects of the chemotherapy agent 5-fluorouracil (5-FU). In these mimics, uracil residues within the miRNA guide strand are substituted with 5-FU. This chemical modification allows the molecule to function as a traditional miRNA—targeting oncogenic mRNAs such as Bcl-2 and HMGB1—while simultaneously delivering 5-FU directly to cancer cells. A significant advantage of this platform is its reported ability to enter cells without the need for specialized transfection vehicles. Research conducted primarily at Stony Brook University has demonstrated their potential in treating colorectal cancer, pancreatic cancer, and non-small cell lung cancer, showing efficacy in inhibiting tumor growth and overcoming chemoresistance.

Other names
5-FU modified tumor suppressor miRNAs5-FU-miRNA mimics
02

Targets

BCL-2 (BCL-2 family)HMGB1 (High mobility group protein B1)

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