How it works?
Emerging targets
Personalized vaccines
Pharma activity
Barriers and challenges
Conclusion
Messenger Ribonucleic Acid (mRNA) vaccines gained global recognition during the Coronavirus Disease 2019 (COVID-19) pandemic, proving their value through rapid development, high efficacy, and broad distribution.
Unlike traditional vaccines that use weakened pathogens, mRNA vaccines instruct cells to produce antigens, triggering an immune response without the risk of infection. Their success in combating Severe Acute Respiratory Syndrome Coronavirus 2 (SARS-CoV-2) demonstrated the power of this platform, especially when paired with lipid nanoparticle (LNP) delivery systems.
This breakthrough has now catalyzed interest in using mRNA technology beyond COVID-19. Researchers are exploring its application in various infectious diseases, such as influenza, Zika, and tuberculosis, as well as in cancer immunotherapy, genetic disorders, and regenerative medicine.
The speed, adaptability, and scalability of mRNA vaccines offer a new frontier in precision medicine. However, improving delivery mechanisms and stability remains essential for maximizing their full potential in future clinical applications.1
This article explores how mRNA vaccine platforms are expanding into new disease areas, from flu to cancer, and what this means for biotech innovation.
Image Credit: QINQIE99/Shutterstock.com
How it works?
mRNA vaccines work by using synthetic mRNA sequences that instruct the body’s cells to produce specific antigens, typically proteins from a virus. Once injected, these mRNA molecules enter the cytoplasm of host cells, where ribosomes translate them into antigenic proteins. These proteins are then displayed on the cell surface, triggering both innate and adaptive immune responses.
Specifically, cytotoxic T cells target infected cells, while helper T cells and B cells drive antibody production. A critical component of this process is the delivery system- most commonly LNPs- which encapsulates the mRNA to protect it from enzymatic degradation and facilitate its entry into cells.1,2
LNPs improve stability and bioavailability and enable mRNA to reach immune cells efficiently. The modularity of mRNA vaccines allows for rapid reprogramming by simply altering the encoded sequence, making them highly adaptable to different pathogens or diseases.
This flexibility, coupled with a swift and scalable production process, positions mRNA technology as a promising platform for future vaccines targeting infectious diseases, cancers, and more.1,2
mRNA vaccines, explained
Emerging targets
mRNA vaccines are rapidly expanding beyond COVID-19, targeting a wide array of emerging diseases. For respiratory conditions like influenza and Respiratory Syncytial Virus (RSV), mRNA vaccines offer faster adaptability to viral mutations, ensuring more timely and effective protection.
In the case of Human Immunodeficiency Virus (HIV), where traditional vaccines have failed, mRNA technologies enable the generation of T cell and antibody responses that better mimic natural infection control.
Cancer is another major frontier. mRNA cancer vaccines are being designed to encode tumor-specific antigens, prompting the immune system to recognize and eliminate cancer cells. They have shown early promise in treating melanoma, prostate cancer, and non-small-cell lung cancer.2
Beyond common diseases, mRNA vaccines are also entering the realm of rare and orphan diseases by offering rapid design and production customized to genetic or antigenic profiles. Their scalability, precision, and ease of modification make them ideal for rare conditions that lack commercial incentives for traditional vaccine development.
Overall, mRNA technology represents a paradigm shift in immunization, offering unmatched speed, flexibility, and potential for personalization across a wide disease spectrum- from seasonal viruses to chronic, life-threatening, and genetically rare disorders.2
Personalized vaccines
Personalized vaccines in cancer immunotherapy represent a transformative approach that targets tumor-specific neoantigens (unique proteins resulting from genetic mutations in cancer cells).
Unlike traditional tumor-associated antigens, neoantigens are absent in normal tissues, making them ideal for precise immune targeting with minimal off-target effects. mRNA vaccines enable rapid, flexible, and scalable production of individualized therapies.
Using next-generation sequencing, a patient’s tumor-specific mutations are identified, and computational tools predict which neoantigens can most effectively stimulate a T cell response.3
The corresponding mRNA sequences are then synthesized and delivered via lipid nanoparticles to instruct the patient’s cells to produce these neoantigens, triggering the immune system to attack the cancer. Clinical trials have shown encouraging results, with mRNA vaccines such as Moderna’s mRNA-4157 and BioNTech’s BNT122 demonstrating strong immune responses and tumor regression.
These vaccines can be integrated with immune checkpoint inhibitors to boost efficacy further. Personalized mRNA cancer vaccines hold immense promise for treating melanoma, lung, colorectal, and other cancers.
However, challenges remain in optimizing delivery, reducing manufacturing costs, and ensuring rapid turnaround from biopsy to treatment. Still, this approach signals a new era of precision oncology, where therapies are not just disease-specific but patient-specific.3
Insight into Personalized Vaccines
Pharma activity
Moderna and BioNTech have emerged as global frontrunners in mRNA vaccine innovation, particularly after their rapid deployment of COVID-19 vaccines. Both companies leveraged decades of mRNA research and LNP delivery systems to bring safe and highly effective vaccines to market quickly.
BioNTech, in collaboration with Pfizer, developed BNT162b2, while Moderna introduced mRNA-1273, each demonstrating over 90% efficacy in clinical trials. These successes catalyzed broader biotech interest and funding into mRNA platforms.1,4
Both companies have significantly expanded their pipelines beyond COVID-19. BioNTech is advancing mRNA therapies for influenza, tuberculosis, and various cancers while also exploring circular RNA and self-amplifying RNA (saRNA) technologies to boost durability and reduce doses. Similarly, Moderna is developing mRNA vaccines for RSV, cytomegalovirus (CMV), seasonal influenza, and rare diseases. It is also applying mRNA to cancer immunotherapy and personalized vaccine approaches.1,4
Strategic partnerships have further accelerated innovation. BioNTech collaborates with Genentech, Fosun Pharma, and Regeneron, while Moderna partners with Merck for cancer therapies and Vertex for cystic fibrosis.
These alliances, combined with proprietary LNP technologies and scalable manufacturing, position both companies to lead the next wave of mRNA-based therapeutics and prophylactics.1,4
Why Pharmacovigilance Is More Critical Than Ever
Barriers and challenges
Despite their transformative potential, mRNA vaccines still face key challenges that limit their widespread adoption in personalized medicine and RNA therapeutics.
A major barrier is cold chain logistics. Most mRNA vaccines, including those developed by Moderna, require storage at sub-zero temperatures to maintain stability, complicating distribution in low-resource settings. This restricts access to potentially life-saving treatments, especially in rural or underserved regions.1,5
Another concern is the risk of immune-related side effects. While mRNA vaccines are designed to trigger precise immune responses, unmodified RNA or impurities can overstimulate the innate immune system, causing inflammation or adverse reactions.
Although techniques like chemical modification and LNP encapsulation help reduce this risk, they add complexity and cost to vaccine development.1,5
Scalability remains a hurdle, as manufacturing mRNA vaccines demands high-purity raw materials, specialized infrastructure, and tightly controlled processes. As a result, transitioning mRNA technology from pandemic response tools to routine RNA therapeutics for cancer, rare diseases, or individualized treatments remains an ongoing challenge.
Addressing these barriers is essential for realizing the full promise of mRNA vaccines in personalized medicine and advancing the next generation of oncology vaccine platforms.1,5
Conclusion
The future of the mRNA vaccine industry looks promising, with ongoing innovations poised to reshape global healthcare. Following the success of COVID-19 vaccines, the industry is shifting toward next-generation platforms that offer greater efficacy, stability, and broader applications.
Companies are exploring self-amplifying and circular mRNA structures, which can increase protein expression and require lower doses, reducing cost and side effects.
Novel delivery systems, including ionizable lipids, dendrimer-based carriers, and biologically derived exosomes, are being developed to enhance precision and reduce toxicity.
These advances aim to overcome current challenges like thermal instability, immune overactivation, and the need for cold chain logistics. mRNA technology is also expanding into oncology, personalized medicine, and treatments for genetic and rare diseases.
Organ-specific targeting and new administration routes like inhalation and oral delivery could make mRNA therapies more accessible and user-friendly. As a result, mRNA platforms are expected to lead the next era of scalable, rapid, and adaptable therapeutics worldwide.
References
- Al Fayez, N., Nassar, M.S., Alshehri, A.A., Alnefaie, M.K., Almughem, F.A., Alshehri, B.Y., Alawad, A.O. and Tawfik, E.A., 2023. Recent advancement in mRNA vaccine development and applications. Pharmaceutics, 15(7), p.1972. (2023). Recent advancement in mRNA vaccine development and applications. Pharmaceutics, 15(7), 1972.
- Chandra, S., Wilson, J. C., Good, D., & Wei, M. Q. (2024). mRNA vaccines: a new era in vaccine development. Oncology Research, 32(10), 1543.
- Vishweshwaraiah, Y. L., & Dokholyan, N. V. (2022). mRNA vaccines for cancer immunotherapy. Frontiers in immunology, 13, 1029069.
- Szabó, G. T., Mahiny, A. J., & Vlatkovic, I. (2022). COVID-19 mRNA vaccines: Platforms and current developments. Molecular Therapy, 30(5), 1850-1868.
- Rosa, S. S., Prazeres, D. M., Azevedo, A. M., & Marques, M. P. (2021). mRNA vaccines manufacturing: Challenges and bottlenecks. Vaccine, 39(16), 2190-2200.https://doi.org/10.1016/j.vaccine.2021.03.038