Drug intelligence / Profile preview

FM-FolamiR-34a

Development stage
Preclinical
Lead developer
Purdue University
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

FM-FolamiR-34a is a first-in-class, vehicle-free, folate-conjugated fully modified microRNA-34a (miR-34a) therapeutic being developed by LigamiR Therapeutics (a spinout from Purdue University) for the treatment of solid tumors, including triple-negative breast cancer (TNBC) and non-small cell lung cancer (NSCLC). It consists of a fully chemically modified miR-34a mimic (FM-miR-34a) directly conjugated to a folate ligand. The folate ligand targets the high-affinity folate receptor alpha (FRα), which is overexpressed on various cancer cells, enabling receptor-mediated endocytosis and vehicle-free delivery. Once inside the cell, the fully modified miR-34a mimic acts as a tumor suppressor, downregulating multiple oncogenic targets simultaneously, including MET, AXL, CD44, MYC, and BCL-2, thereby inhibiting tumor growth, proliferation, and metastasis.

Other names
FM-miR-34a conjugateFM-miR34a conjugateFM-miR 34a conjugatefolate-conjugated fully modified miR-34afully modified FolamiR-34a
02

Targets

CD44 (CD44 antigen)MET (Mesenchymal-epithelial transition factor receptor)AXL (AXL receptor tyrosine kinase)BCL-2 (BCL-2 family)FOLR1 (FRα)MYC (MYC proto-oncogene protein)

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