Drug intelligence / Profile preview

miristen

Development stage
Phase 1
Lead developer
City of Hope Comprehensive Cancer Center
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

**Miristen** is an experimental anti-miR-126 oligonucleotide therapeutic. It is a synthetic oligodeoxynucleotide designed to antagonize **microRNA-126 (miR-126)**, which is highly expressed in leukemia stem cells (LSCs) and the endothelial cells of the bone marrow microenvironment. By depleting miR-126, Miristen disrupts the maintenance and self-renewal capacity of LSCs, alters the bone marrow environment, and reduces resources available to sustain leukemia. This approach targets both the malignant cells and their supportive niche, offering a strategy to eradicate leukemia at its root and prevent relapse. Preclinical studies show Miristen improves outcomes in acute myeloid leukemia (AML), myelodysplastic syndromes (MDS), and chronic myelogenous leukemia (CML), particularly when combined with standard treatments such as tyrosine kinase inhibitors (TKIs) or venetoclax. Miristen is developed at City of Hope and, as of early 2025, is in late preclinical development with preparations underway for first-in-human clinical trials[1][2][3][4][5][7][8][9].

Other names
miRisten

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