Beyond mRNA: non-coding RNAs open new frontiers for innovative therapies (thursday 3)

Discussion highlights the potential of non-coding RNAs and emerging RNA platforms to transform therapeutic development and precision medicine.

The session “A Future Beyond Messengers: Non-Coding RNAs as Targets for New Therapies”, held during the 10th International Symposium on Immunobiologicals (ISI), brought together researchers and experts to discuss scientific advances and challenges in applying non-coding RNAs and other RNA-based technologies to the development of new therapies.

Chaired by Patricia Neves (Fiocruz/Bio-Manguinhos), the session featured John Mattick (UNSW), Paulo de Paiva Amaral (Insper), Lucas Rocha (Mirscience), and Danielle Cunha (Fiocruz/Bio-Manguinhos).

Prior to the main presentations, two poster oral sessions provided relevant contributions. Renata Maria dos Santos discussed the analysis of aggregates in naked mRNA and mRNA encapsulated in lipid nanoparticles, highlighting implications for the quality and stability of mRNA vaccines. Daniele Ramos Rocha presented advances in consolidating mRNA-LNP production at Bio-Manguinhos, including the evaluation of critical quality attributes at pilot scale.

Opening the session, John Mattick explored the role of long non-coding RNAs (lncRNAs) and their potential to transform biomedicine. He emphasized that these elements, once considered non-functional, are now recognized as key regulators in gene expression and in the understanding of complex diseases.

Paulo de Paiva Amaral then addressed the challenges of translating scientific knowledge on non-coding RNAs into therapeutic applications. According to him, progress in this field represents a strategic opportunity for Brazil to expand its role in cutting-edge technologies related to precision medicine.

Lucas Rocha, from the startup Mirscience, presented the development of synthetic RNAs for the treatment of genetic muscle diseases. He highlighted the potential of new therapeutic platforms to overcome limitations of traditional approaches, particularly for rare diseases.

Closing the session, Danielle Cunha presented research on the use of RNA interference (RNAi) for the treatment of triple-negative breast cancer. She noted that results so far are promising, although challenges remain in advancing these therapies through clinical development and regulatory pathways.

Throughout the discussion, key bottlenecks in translating scientific discoveries into therapeutic products were highlighted, including funding constraints, regulatory challenges, and the need for robust infrastructure to support preclinical and clinical development.

The session made clear that, although still evolving, RNA-based technologies — particularly non-coding RNAs — represent one of the most promising frontiers in health innovation, with the potential to reshape therapeutic approaches and expand the reach of personalized medicine.

Text: Marcela Dobarro
Image: Monara Barreto