Advances in RNA-based cancer therapeutics: pre-clinical and clinical implications

Oct 10, 2025Molecular cancer

Progress in RNA-based cancer treatments and their early and clinical impacts

AI simplified

mRNA Technology on OpenScience ↗PubMed ↗DOI ↗OA ↗

Abstract

RNA-based therapeutics are showing promising early-phase clinical outcomes in treating solid tumors and hematologic malignancies.

mRNA therapeutics are being developed for cancer immunotherapy and protein replacement.
Small interfering RNAs (siRNAs) and antisense oligonucleotides (ASOs) provide targeted gene silencing and splice correction.
MicroRNA therapies have potential for controlling various oncogenic pathways but face delivery and specificity challenges.
RNA aptamers are gaining attention for their role as tumor-targeting agents in drug delivery systems.
Advancements in lipid nanoparticles and tissue-specific delivery techniques have enhanced the stability and efficacy of RNA-based agents.
Ongoing challenges include scalability, immune activation, and penetration into deep tumors.

AI simplified

Cancer therapy has been revolutionised by the emergence of RNA-based therapeutics, providing several strategies and mechanisms to regulate gene expression via messenger RNA (mRNA), small interfering RNA (siRNA), microRNAs (miRNA), antisense oligonucleotides (ASOs), and RNA aptamers. The present review highlights the recent advances in the preclinical development and clinical applications of RNA-based therapeutics, focusing on the delivery strategies, biological targets, and pharmacological optimisation, together with key clinical data. mRNA therapeutics, especially those adapted from vaccine platforms are being developed for the cancer immunotherapy and protein replacement, while siRNAs and ASOs enable highly specific gene silencing and splice correction. miRNA therapies show potential for diverse oncogenic pathway control, despite ongoing challenges in the delivery and specificity. RNA aptamers are obtaining attention as tumor-targeting agents in the drug delivery systems. Progress in lipid nanoparticles, chemical modifications, and tissue-specific delivery has improved the stability and efficacy of these agents. Early-phase clinical trials report encouraging outcomes in both solid tumours and haematologic malignancies, particularly in overcoming resistance and modulating the tumor microenvironment (TME). Although challenges remain in scalability, immune activation, and deep-tumour penetration, RNA-based strategies are advancing towards integration into clinical oncology. Continued refinement of delivery technologies and targeted trial designs will be critical for translating these therapies into effective, personalized cancer treatments.

Full Text

We can’t show the full text here under this license. Use the link below to read it at the source.

View full text (PDF) ↗View full text (HTML) ↗

Advances in RNA-based cancer therapeutics: pre-clinical and clinical implications

Abstract

Full Text

You found one interesting study. We’ll send the next 7.

what lands in your inbox each week:

Recent issues from the mrna technology brief

Abstract

Full Text

Related papers

You found one interesting study. We’ll send the next 7.

what lands in your inbox each week:

Recent issues from the mrna technology brief