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miR-129 mimic
- Development stage
- Preclinical
- Lead developer
- Curamir Therapeutics
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, 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
- Administration
- Intravenous
01
Overview
miR-129 mimic is a synthetic double-stranded microRNA-129 replacement construct, often chemically modified by substituting uracil with 5‑fluorouracil (5‑FU), being developed as an experimental anticancer RNA therapeutic. 5‑FU–modified miR‑129 mimics (notably the candidate termed Mimic‑1) retain the native miR‑129 seed sequence and target spectrum while gaining increased stability, vehicle‑free cellular uptake, and additional cytotoxicity from intracellular release of 5‑FU metabolites that inhibit thymidylate synthase. These mimics downregulate multiple oncogenic and survival pathways by directly targeting transcripts such as BCL2, HMGB1, and E2F3, leading to G1 cell‑cycle arrest, apoptosis, and inhibition of proliferation in colorectal and non‑small cell lung cancer models, including chemotherapy‑ and tyrosine kinase inhibitor‑resistant cancer stem cell populations in vitro and in vivo.[1][3][4][6]
- Other names
- 5-FU-miR-129Mimic-1Mimic1Mimic 15-FU modified miR-129 mimic
02
Targets
BCL-2 (BCL-2 family)Bcl-2-like protein 2 mRNAE2F3 (Transcription factor E2F3)HMGB1 (High mobility group protein B1)TS (Thymidylate synthase)
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