Drug intelligence / Profile preview

novel_miRNA_2 mimic

Development stage
Preclinical
Lead developer
Henry Ford Health
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

novel_miRNA_2 mimic is a synthetic microRNA mimic of a previously unannotated human microRNA, novel_miRNA_2 (sequence: UCCCUGUUCGGGCGCCACU), which acts as an endogenous suppressor of pathological subchondral bone remodeling during early-stage osteoarthritis (OA). Developed by researchers at Henry Ford Health, Michigan State University, and the University Health Network, the mimic functions as a canonical microRNA that is loaded into Argonaute 2 (AGO2)-containing RNA-induced silencing complexes (RISCs) to downregulate target genes involved in osteogenesis and mineralization, including RUNX2, ALPL, and IL11. In preclinical models, systemic delivery of the novel_miRNA_2 mimic during early-stage knee OA significantly reduces cartilage damage and osteophyte burden, demonstrating potential as a disease-modifying therapeutic.

Other names
novel_miRNA_2
02

Targets

ALPL (Tissue-nonspecific alkaline phosphatase)IL11 (Interleukin-11)RUNX2

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