mRNA Beyond Vaccines: Expanding Applications in Pharma

When most of us think of mRNA technology, COVID-19 vaccines instantly come to mind. But mRNA is not just a vaccine superstar, it’s an emerging powerhouse in pharmaceutical innovation. From personalized cancer treatments to rare genetic disorders and cutting-edge immunotherapies, mRNA is reshaping the way medicine is practiced.

Let’s explore how this flexible technology is going beyond vaccines to deliver life-changing treatments.

Understanding mRNA in Medicine

mRNA serves as a molecular code that tells cells how to build certain proteins. In medicine, synthetic mRNA molecules can instruct the body’s cells to create target proteins, whether a harmless viral fragment to provoke immunity or a missing protein in a genetic condition. Unlike conventional biologic drugs, mRNA therapies can be created and modified in a short time, making them highly adaptable for personalized treatments.

Cancer Immunotherapy and Personalized Vaccines

One of the most exciting frontiers for mRNA is in cancer treatment, specifically personalized cancer vaccines. These are therapeutic vaccines tailored to an individual’s tumor, encoding tumor-specific antigens (such as neoantigens unique to that patient’s cancer) that train the immune system to attack the tumor. For instance, in a recent melanoma trial, an individualized mRNA vaccine called mRNA-4157 (V940) was given alongside the immunotherapy drug pembrolizumab. The combination reduced the risk of the cancer returning or causing death by 44% compared to immunotherapy alone.¹ This marked improvement (only 22% of patients had their cancer return with the vaccine+immunotherapy combination, versus 40% with immunotherapy alone) highlights the power of tailoring a vaccine to a patient’s unique tumor mutations. Thanks to mRNA’s flexibility, such vaccines can be custom-designed within weeks based on a patient’s tumor genetics, a true breakthrough in personalized oncology.

Expanding to Other Therapeutic Uses

mRNA’s potential extends well beyond vaccines and oncology. Researchers are exploring its use in treating rare genetic disorders by providing a code for the functional proteins that patients lack.² For example, one experimental therapy uses mRNA to replace the defective enzyme in methylmalonic acidemia, a rare metabolic disorder that causes a toxic buildup of acids. By supplying a genetic code, lab-synthesized mRNA directs cells to generate methylmalonyl-CoA mutase, the enzyme absent in these patients.³

In fact, multiple clinical trials are underway using mRNA for inherited metabolic conditions like propionic acidemia, essentially using mRNA as a form of protein replacement therapy for missing enzymes.⁴

Regenerative medicine is another area where mRNA shows remarkable potential. In one clinical study, researchers injected an mRNA encoding VEGF-A (a protein that promotes blood vessel growth) directly into patients’ heart muscle to repair damage after a heart attack. The idea is that the mRNA will spur new blood vessel formation and help regenerate heart tissue, an approach that early trials suggest is feasible.⁵

Another emerging application is in autoimmune diseases. Instead of triggering an immune attack, specially engineered mRNA can be used to tolerize the immune system (essentially 're-educating' the immune system not to attack certain targets). A landmark mouse study demonstrated that an mRNA vaccine encoding a self-antigen (a normal body protein) delivered in a non-inflammatory nanoparticle could suppress an MS-like autoimmune disease by inducing antigen-specific immune tolerance.

The mRNA prompted the production of regulatory T cells that calmed the autoimmune reaction. This proof of concept suggests mRNA might eventually treat autoimmune disorders (or severe allergies) by teaching the body to tolerate its own proteins, offering a targeted alternative to general immune-suppressing drugs.

The Power of Delivery Systems

A key challenge for any mRNA therapy is delivery, ensuring that the fragile mRNA reaches the right cells intact. If you injected naked mRNA into the body, it would be quickly degraded by enzymes and would struggle to enter cells. Scientists solved this problem with lipid nanoparticles (LNPs), which are tiny fat-based capsules that package the mRNA.⁷ The LNP acts like a protective bubble (a molecular delivery truck), shielding the mRNA cargo from destruction and shuttling it into cells.

 When an mRNA–LNP formulation is injected, the nanoparticle carries the mRNA through the bloodstream and merges with target cells, releasing the mRNA inside so the cell can start producing the encoded protein.⁸

This delivery technology continues to advance. Researchers are designing new LNP formulations to improve targeting to specific tissues and cell types. For instance, to treat a liver disorder, an mRNA’s LNP can be chemically tuned to be taken up primarily by liver cells. Similarly, LNP “vehicles” can be equipped with targeting molecules (like antibodies or sugars) to direct them to organs such as the spleen, lungs, or even to tumor sites. These innovations are paving the way for mRNA therapies in fields like gene therapy and precision oncology, where getting the genetic payload to the right place is crucial.

Building Momentum: Clinical Trials and Market Potential

The momentum behind mRNA is undeniable. Over 120 clinical trials are underway in oncology alone, spanning cancers like melanoma, pancreatic, and brain tumors.⁹ The ability to rapidly design, test, and scale mRNA treatments positions this technology as a future staple in personalized medicine and public health.

Meanwhile, mRNA vaccine research for infectious diseases is also accelerating, and an mRNA vaccine for RSV was approved in 2024, and candidates for influenza, HIV, and other viruses are in development.

A major appeal of mRNA therapeutics is their speed and adaptability. Designing a new mRNA drug for a different target is as simple as changing the nucleotide sequence in the lab, a far quicker process than traditional drug development. This raises the prospect of true personalized medicine: if each patient’s disease has a unique genetic signature, an mRNA therapy could be rapidly crafted to match that individual profile.¹⁰ It is no surprise that pharmaceutical companies are making significant investments in mRNA technology. Experts even predict that mRNA will become a staple platform in medicine, powering everything from tailored cancer immunotherapies to rapid-response vaccines in future pandemics.

Summary

mRNA has broken free from its vaccine fame to emerge as a transformative force in pharma. Whether in personalized cancer therapy, treating rare disorders, or revolutionizing immune modulation, mRNA is changing the game. With ongoing clinical trials and delivery technologies like LNPs fueling its advance, this versatile platform promises to redefine what it means to provide tailored, effective medical care.

Frequently Asked Questions (FAQs)

1. What are some real-world examples of mRNA therapies beyond vaccines?
 Examples include mRNA-4157 (a cancer vaccine reducing melanoma recurrence) and personalized vaccines targeting tumor neoantigens.

2. Why is delivery a challenge, and how is it solved?
 mRNA is fragile and easily degraded in the body. Lipid nanoparticles (LNPs) protect mRNA and help transport it safely into cells.

3. What makes mRNA an ideal platform for personalized medicine?
 mRNA can be tailored to encode specific proteins or antigens unique to patients or diseases, enabling rapid development and highly individualized treatments.

References

Adding New Vaccine Type to Leading Immunotherapy Dramatically Reduced Melanoma Recurrence. NYU Langone News. https://nyulangone.org/news/adding-new-vaccine-type-leading-immunotherapy-dramatically-reduced-melanoma-recurrence

Saxena S, Mandrah V, Tariq W, Das P, Sambhav K, Devi SH. The Future of mRNA Vaccines: Potential Beyond COVID-19. Cureus. Published online May 21, 2025. doi:https://doi.org/10.7759/cureus.84529

Valsecchi MC. Rare diseases the next target for mRNA therapies. Nature Italy. Published online May 9, 2021. doi:https://doi.org/10.1038/d43978-021-00058-x

TL Rare Disease Day Feb 28th, 2023—Six mRNA Therapeutics in Development. Trilinkbiotech.com. Published February 6, 2023. Accessed September 10, 2025. https://www.trilinkbiotech.com/blog/rare-disease-day-feb-28th-2023six-mrna-therapeutics-in-development-/

in. AZD8601 EPICCURE Phase II trial demonstrated safety and tolerability in patients with heart failure. Astrazeneca.com. Published November 15, 2021. Accessed September 10, 2025. https://www.astrazeneca.com/media-centre/press-releases/2021/azd8601-epiccure-phase-ii-trial-demonstrated-safety-and-tolerability-in-patients-with-heart-failure.html

Mariona Estapé Senti, Valle del, Schiffelers RM. mRNA delivery systems for cancer immunotherapy: Lipid nanoparticles and beyond. Advanced Drug Delivery Reviews. 2024;206:115190-115190. doi:https://doi.org/10.1016/j.addr.2024.115190

Lu RM, Hsu HE, Lynon J, et al. Current landscape of mRNA technologies and delivery systems for new modality therapeutics. Journal of Biomedical Science. 2024;31(1). doi:https://doi.org/10.1186/s12929-024-01080-z

Fujimori S. Inside the delivery vehicles critical to the success of mRNA - mRNA part 3 pfizer. www.pfizer.com. Published June 2022. https://www.pfizer.com/news/behind-the-science/inside-delivery-vehicles-critical-success

Yaremenko AV, Khan MM, Zhen X, Tang Y, Tao W. Clinical advances of mRNA vaccines for cancer immunotherapy. Med. 2025;6(1):100562. doi:https://doi.org/10.1016/j.medj.2024.11.015

Lu RM, Hsu HE, Lynon J, et al. Current landscape of mRNA technologies and delivery systems for new modality therapeutics. Journal of Biomedical Science. 2024;31(1). doi:https://doi.org/10.1186/s12929-024-01080-z