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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.
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