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microRNA-34a
- Development stage
- Unknown
- Lead developer
- Synlogic
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous, Intratumoral
01
Overview
microRNA-34a is a naturally occurring tumor-suppressive microRNA that is frequently downregulated in various cancers, including glioblastoma, medulloblastoma, and hepatocellular carcinoma. It functions by post-transcriptionally repressing multiple oncogenic targets involved in cell cycle progression, apoptosis resistance, and self-renewal, such as MET, NOTCH1/2, CDK6, BCL2, and MYC. As a therapeutic, synthetic mimics of miR-34a (most notably MRX34) have been developed for delivery via lipid nanoparticles (LNPs). While miR-34a showed potent anti-tumor activity in preclinical models, clinical development was halted during Phase 1 trials due to severe immune-related adverse events, highlighting the challenges of systemic microRNA delivery and off-target immune activation.
- Other names
- microRNA 34amicroRNA34amicroRNA-34ahsa-miR-34ahsa-miR34ahsa-miR 34a
02
Targets
NOTCH (Neurogenic locus notch homolog protein 3)BCL-2 (BCL-2 family)CDK6 (Cyclin-dependent kinase 6)MET (Mesenchymal-epithelial transition factor receptor)MYC (MYC proto-oncogene protein)NOTCH2 (Neurogenic locus notch homolog protein 2)
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