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This article provides an in-depth analysis of the scientific principles, delivery systems, and unique advantages of mRNA technology. It comprehensively reviews major applications and clinical breakthroughs across three key areas—infectious disease prevention, cancer therapy, and protein replacement—and unveils the critical bioanalytical methods underpinning its development.
mRNA Technology
mRNA technology is a biomedical approach that involves designing and introducing specific messenger RNA (mRNA) sequences into the body. These sequences instruct cells to produce target proteins or antigens, thereby triggering humoral or cellular immune responses in the host. mRNA technology has demonstrated immense potential and advantages in drug discovery and disease treatment, making it a major focus of global medical research.
The 2023 Nobel Prize in Physiology or Medicine was awarded to two pioneers of mRNA technology, Katalin Karikó and Drew Weissman. They were recognized for their discovery of nucleoside base modifications, which reduce the immunogenicity of mRNA and address its instability, thereby enabling the broad medical application of mRNA technology.
The mechanism of action for mRNA drugs involves delivering antigen-encoding mRNA into cells via specific delivery systems. The mRNA is then translated into protein within the body, stimulating an adaptive immune response to confer immune protection or therapeutic effect. This process can be broken down into four steps:
1.Delivery and Cellular Uptake: Chemically modified mRNA is encapsulated within a delivery system, typically lipid nanoparticles (LNPs). The LNPs protect the mRNA from degradation during transport, fuse with the cell membrane, and efficiently deliver the mRNA into the cytoplasm.
2.Translation and Expression: Once in the cytoplasm, the mRNA is released from the LNPs and binds to ribosomes. The ribosomes read the mRNA's coding sequence and use the cell's amino acid building blocks to synthesize the corresponding antigen (protein).
3.Immune Recognition and Response: The newly synthesized protein is processed and presented on the cell surface or secreted extracellularly. The immune system recognizes these antigens as "foreign," initiating both humoral and cellular immune responses.
4.mRNA Clearance: After the target antigen is produced, the mRNA is rapidly and naturally degraded by cellular enzymes and does not integrate into the human genome.
mRNA technology offers significant strengths for drug development:
Design Flexibility and Speed: mRNA drug design is straightforward, rapid, and adaptable. Once the genetic sequence of an effective antigen is known, design and development can commence. AI, big data, and networking technologies have further streamlined mRNA vaccine design, enabling the transition from sequence publication to effective candidate design in a matter of days.
Streamlined Manufacturing: The synthesis and production processes for mRNA drugs are relatively simple and cost-effective. They do not rely on cell culture or pathogen cultivation, allowing for rapid design, construction, and large-scale manufacturing.
Potent and Durable Immunity: mRNA vaccines can effectively stimulate both cellular and humoral immunity. Once in the cytoplasm, they enable sustained antigen protein expression until degradation, leading to longer-lasting adaptive immune responses and immunological memory.
Safety Profile: mRNA vaccines do not introduce the pathogen itself or its components into the body, eliminating the risk of infection from the vaccine. mRNA has a short half-life, degrades quickly, and does not interfere with the normal physiological functions or metabolism of host cells.
mRNA Drugs
Drugs developed using mRNA technology fall into three main categories: prophylactic vaccines, therapeutic vaccines, and therapeutic drugs. Statistics show over 120 mRNA drug candidates in clinical pipelines globally, predominantly in vaccines. Prophylactic vaccines account for 65%, therapeutic vaccines for 25%, and therapeutic drugs for approximately 10%, with most in Phase I clinical trials.
The first successful application of mRNA technology was in COVID-19 vaccines. Following the global outbreak in 2020, two mRNA vaccines received emergency authorization: Pfizer/BioNTech's BNT162b2 (Comirnaty) and Moderna's mRNA-1273 (Spikevax). Both vaccines use mRNA encoding the full-length spike protein to stimulate protective antibodies, effectively curbing the spread of COVID-19.
Another successfully launched mRNA prophylactic vaccine targets Respiratory Syncytial Virus (RSV): Pfizer's Abrysvo (approved September 2023) and Moderna's mRESVIA (approved May 2024). These RSV mRNA vaccines deliver mRNA encoding a stabilized conformation of the RSV F protein as an immunogen, showing good efficacy in preventing RSV-associated lower respiratory tract infections and pneumonia.
Currently, mRNA vaccine pipelines targeting varicella-zoster virus, influenza, Zika virus, tuberculosis, and others are under development or in clinical trials worldwide.
mRNA cancer therapeutic vaccines work by injecting mRNA encoding tumor antigens into patients. Host cells then produce these antigens, which are recognized by immune cells, activating a targeted immune response against cancer cells, establishing lasting anti-tumor memory, and killing tumor cells. Unlike traditional cancer vaccines, mRNA vaccines can present multiple antigens simultaneously, cover numerous tumor epitopes, and encode full-length tumor antigens, allowing antigen-presenting cells to present multiple epitopes concurrently or cross-present them, offering design flexibility and precision.
Recent years have seen remarkable achievements in mRNA cancer vaccine development:
In pancreatic cancer, a February 2025 Nature study reported that a personalized mRNA vaccine co-developed by BioNTech and Genentech induced specific T-cell responses in 50% of patients and reduced postoperative recurrence risk by 86%, improving survival rates for this challenging cancer.
At the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, 3-year follow-up data for Moderna and Merck's mRNA-4157 vaccine combined with pembrolizumab showed a 49% reduction in recurrence or death risk and a 62% reduction in distant metastasis risk for high-risk Stage III/IV melanoma patients. The 2.5-year recurrence-free survival rate was 74.8% in the combination group, far exceeding the 55.6% with monotherapy.
Over 20 mRNA cancer vaccines developed in China have entered clinical stages, covering major cancers like liver, lung, gastrointestinal, and pancreatic cancers. For instance, LK101 from Life's Carnival, the first Chinese mRNA cancer vaccine to receive FDA clearance for clinical trials, achieved a 100% 3-year survival rate in a Phase I trial for hepatocellular carcinoma patients when combined with ablation therapy. The first Chinese patient with a solid tumor was dosed in Boao, Hainan in April 2025. Everest Medicines' EVM16 completed its first patient injection in March 2025; its AI-driven neoantigen algorithm significantly improves customization efficiency, and combining it with PD-1 inhibitors shows promising efficacy enhancement.
mRNA technology can temporarily supplement missing or dysfunctional proteins in patients by delivering mRNA encoding functional proteins, offering potential treatments for genetic disorders, rare diseases, or autoimmune conditions. Examples include Moderna's mRNA-3704 (for methylmalonic acidemia) and mRNA-3927 (for propionic acidemia) in Phase I trials; Arcturus Therapeutics' LUNAR-1 (for ornithine transcarbamylase deficiency) receiving FDA Fast Track designation; and BioNTech's BNT211 (targeting myelin basic protein for multiple sclerosis) in preclinical stages.
Bioanalysis for mRNA Drugs
Bioanalysis for mRNA drugs primarily encompasses:
mRNA Quantification and Biodistribution: Commonly analyzed using Reverse Transcription Quantitative PCR (RT-qPCR).
LNP Distribution and Stability Analysis: Liquid Chromatography-Mass Spectrometry (LC-MS) is a standard quantitative method for assessing the lipid nanoparticle delivery system.
Expression Protein Detection: Since mRNA drugs must be translated into protein to function, confirming protein expression levels and distribution is crucial. Immunoassays like MSD Electrochemiluminescence (ECL) and ELISA are used to evaluate translation efficiency.
Immunogenicity Analysis for mRNA Vaccines: Assessing both cellular and humoral immune response efficacy.
Summary and Outlook
mRNA technology represents a major revolution and breakthrough in biomedicine. From infectious disease prevention to cancer treatment, and from genetic disease correction to tissue repair, mRNA drugs are opening new frontiers in therapy. However, challenges remain in their development and application, including technical complexity, production costs, delivery system optimization, and stringent regulatory requirements. In summary, mRNA holds vast promise for preventing infectious diseases and treating challenging conditions like cancer and various rare diseases. With advancements in personalized medicine and precision therapy, mRNA-based therapies are poised to become a cornerstone of the pharmaceutical industry, offering new hope for human health.
About SDM's Laboratories (Beijing Shouyan & Hangzhou Amador)
SDM 's affiliated laboratories (Beijing Shouyan and Hangzhou Amador) are committed to providing comprehensive bioanalytical testing services for a wide range of drug candidates, building extensive expertise in the field. For mRNA drug clinical trials, we have developed specialized bioanalytical platforms for Pharmacokinetics (PK), Pharmacodynamics (PD), and Immunogenicity. Our services include mRNA metabolism and distribution analysis via RT-qPCR, and assessment of cellular and humoral immune response levels through our vaccine immunogenicity testing platform. We develop tailored analytical strategies to support different mRNA therapies, offering high-quality laboratory technical support to advance the clinical development and application of mRNA drugs.
References
1. Polack F.P., Thomas S.J., Kitchin N., et al. Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine. N Engl J Med. 2020;383(27):2603-2615.
2. Yu Jing, Yang Zhenjun. Application of mRNA Vaccines in Cancer Immunotherapy. Journal of China Pharmaceutical University. 2025;56(4):444-452.
3. Pardi N., Hogan M.J., Weissman D. Recent advances in mRNA vaccine technology. Curr Opin Immunol. 2020;65:14-20.
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Authored by the SDM Bioanalysis Team, this article delves into the principles, delivery systems and advantages of mRNA technology, provides a comprehensive overview of its applications and clinical breakthroughs in three major fields—infectious disease prevention, cancer therapy and protein replacement therapy—and elaborates on the key bioanalytical methodologies.
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