Drug intelligence / Profile preview

miR-34a dumbbell RNA

Development stage
Preclinical
Lead developer
Purdue University
Modality
RNA Therapeutics → Nucleic Acid Therapeutics
Administration
Intravenous
01

Overview

miR-34a dumbbell RNA (db34a) is an experimental RNA therapeutic consisting of a circular, dumbbell-shaped RNA molecule that encodes both miR-34a-3p and miR-34a-5p. Developed by researchers at the University of Illinois, it is produced using the permuted intron-exon (PIE) method in a bacterial expression system, which provides a stable and potentially safer alternative to viral or plasmid-based delivery systems. The therapeutic acts as a microRNA mimic, targeting and downregulating the expression of vascular endothelial growth factors A and B (VEGFA and VEGFB). This mechanism suppresses pathological angiogenesis and has been primarily investigated for the treatment of pancreatic cancer, where it also demonstrates the ability to activate macrophages.

Other names
miR-34a dbRNAmiR34a dbRNAmiR 34a dbRNAcircular dumbbell RNA expressing miR-34a
02

Targets

CD44 (CD44 antigen)MET (Mesenchymal-epithelial transition factor receptor)NOTCH (Neurogenic locus notch homolog protein 3)VEGFB (Vascular endothelial growth factor B)AXL (AXL receptor tyrosine kinase)BCL-2 (BCL-2 family)SIRT1 (NAD-dependent protein deacetylase sirtuin-1)VEGFA (Vascular endothelial growth factor A)

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