December 3, 2025
RNA is well established in life sciences and biotech as both a therapeutic modality and a drug target. mRNA based vaccines and siRNA therapeutics have been successfully commercialized, while other types of RNA therapeutic products are still in pre-clinical and clinical development. However, long non-coding RNAs (lncRNAs) are still mysterious and their potential as a drug target remains unknown.
RNA is usually divided into coding RNA and non-coding RNA. Coding RNAs, like mRNA contain a 5’ cap, UTRs, a poly(A) tail, and a coding region for a protein. Non-coding RNA is a bit more elusive. Our current understanding is that non-coding RNA is involved in regulating gene expression and cell proliferation. For example, although miRNAs do not code for proteins, they bind to mRNA to alter its function.
Then there are long non-coding RNAs (lncRNAs), which are strands of RNA that are greater than 200 nucleotides. They also lack a protein coding region. Our current understanding is that lncRNA is involved in regulating gene expression and cell proliferation. Yet, their exact biological mechanisms are still being investigated. Researchers have documented distinct expression levels of lncRNA in patients with various diseases. However, how these expression levels translate as biomarkers, diagnostic agents, or even drug targets are not well understood.
We currently know that lncRNA can be upregulated or down regulated in various diseases, but this does not mean that lncRNAs will serve as a potential drug target. We still do not know whether lncRNAs have specificity. If lncRNAs lack specificity for their natural biological target (cross-talk), then targeting lncRNAs could have adverse effects. As researchers continue to engage in basic research in the lncRNA space, their potential as a drug target may be revealed.
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