Drug intelligence / Profile preview

miR34a

Development stage
Discontinued
Lead developer
Synlogic
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

miR34a (microRNA-34a) is a naturally occurring tumor-suppressive microRNA that acts as a master regulator of multiple oncogenic pathways. It is transcriptionally induced by the p53 tumor suppressor and is frequently downregulated or lost in various human cancers, including hepatocellular carcinoma, lung cancer, and hematological malignancies. miR34a functions by binding to the 3' untranslated region (UTR) of target mRNAs, leading to their degradation or translational inhibition. Key direct targets of miR34a include oncogenes and cell cycle regulators such as BCL2, CDK4, CDK6, MET, MYC, NOTCH1, and PD-L1. In clinical development, a synthetic double-stranded miR-34a mimic encapsulated in a liposomal nanoparticle, known as MRX34, was developed by Mirna Therapeutics for the treatment of advanced solid tumors and hematological malignancies. However, the Phase I clinical trial of MRX34 was prematurely terminated due to severe immune-mediated adverse events, including cytokine release syndrome.

Other names
hsa-mir-34ahsa-mir34ahsa-mir 34amiR-34amiR34amiR 34amiR-34a mimicmiR34a mimicmiR 34a mimicmiR-Rx34miR-Rx-34miR-Rx 34microRNA 34amicroRNA34amicroRNA-34a
02

Targets

CD44 (CD44 antigen)MET (Mesenchymal-epithelial transition factor receptor)NOTCH (Neurogenic locus notch homolog protein 3)BCL-2 (BCL-2 family)SIRT1 (NAD-dependent protein deacetylase sirtuin-1)CD274 (Programmed cell death protein 1 ligand 1)MYC (MYC proto-oncogene protein)CDK6 (Cyclin-dependent kinase 6)

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