Messenger RNA (mRNA) technology has evolved from a niche research concept to a transformative force in global healthcare. While the COVID-19 pandemic accelerated its visibility, the scope of mRNA extends far beyond vaccines. From oncology to rare genetic diseases, mRNA-based therapeutics are reshaping the way diseases are treated, driving both clinical innovation and regulatory recalibration.
For pharmaceutical companies, this rapid evolution creates a dual challenge: navigating complex approval frameworks while staying ahead in a highly competitive innovation race. Understanding the current regulatory landscape and how it is adapting to novel modalities like mRNA is critical for ensuring market readiness and long-term success.
Current Status of mRNA Technology
The success of COVID-19 vaccines from Moderna and Pfizer-BioNTech demonstrated the scalability and speed of mRNA deployment. A major milestone followed in May 2024, when the U.S. FDA approved Moderna’s RSV vaccine (mRESVIA) for adults aged 60+. In September 2025, the FDA further expanded its indication to include adults 18–59 at higher risk of RSV infection. These approvals highlight growing regulatory confidence in the adaptability and safety of mRNA beyond COVID-19.
mRNA technology leverages the body’s own cellular machinery to produce therapeutic proteins in vivo, rather than delivering proteins directly. This enables a versatile platform across infectious diseases, oncology, rare genetic conditions, and regenerative medicine.
The global mRNA therapeutics market is projected to reach USD 42.6 billion by 2034 at a CAGR of ~8.3% (BioSpace, 2025), underscoring its expanding role in modern medicine.
How Translational Biology Is Fast-Tracking Tomorrow’s Therapies
Applications of mRNA Technology in Healthcare
Cancer Immunotherapies
mRNA cancer vaccines are designed around a patient’s tumour-specific neoantigens, instructing immune cells to recognize and destroy cancer cells.
BioNTech and Moderna are advancing mRNA vaccines in clinical trials for melanoma, colorectal cancer, and non-small cell lung cancer, often in combination with checkpoint inhibitors.
Early-phase results indicate strong potential for highly personalized oncology treatments.
Genetic Disorders
mRNA can be engineered to produce proteins missing due to genetic mutations.
Sanofi’s Translate Bio program has explored mRNA for cystic fibrosis, aiming to instruct lung cells to generate functional CFTR proteins.
This demonstrates how mRNA could one day address diseases caused by defective protein synthesis.
Rare Metabolic Diseases
Inherited enzyme deficiencies, such as propionic acidemia and methylmalonic acidemia, are candidates for mRNA therapies that encode functional enzymes.
Correction: The FDA-approved drug patisiran (for transthyretin-mediated amyloidosis) is an RNA interference (siRNA) therapy, not mRNA. No mRNA therapies for metabolic disorders are yet approved, although several are in preclinical development.
Autoimmune Disorders
mRNA is being tested for immune tolerance induction by encoding autoantigens in a controlled way, with the goal of reducing inflammation and preventing immune attack on healthy tissues. These programs are currently in the research stages.
Regenerative Medicine & Cardiovascular Health
mRNA encoding growth factors can accelerate tissue repair after injury or ischemia.
Moderna’s preclinical program using VEGF-A mRNA aims to stimulate blood vessel growth in ischemic heart disease.
Collaborations, such as AstraZeneca with Moderna, are also exploring mRNA’s role in cardiovascular regeneration.
Key Benefits of mRNA Technology
- Speed: mRNA constructs can be rapidly designed and manufactured, enabling fast responses to pandemics and personalized therapies.
- Versatility: A single platform can encode diverse proteins for applications across infectious disease, oncology, and regenerative medicine.
- Non-Integrating Nature: Unlike DNA-based therapies, mRNA does not integrate into the host genome, minimizing risk of insertional mutagenesis.
- One Health Applications: mRNA vaccines are being developed for livestock, such as cattle and poultry, to combat diseases including bovine respiratory disease and avian flu, reducing antibiotic use and strengthening food security.
Challenges in mRNA Development
- Targeted Delivery: Lipid nanoparticles (LNPs) are effective but can trigger immune reactions and lack precision tissue targeting.
- Stability: mRNA molecules are inherently unstable, requiring cold-chain infrastructure and chemical modifications for preservation.
- Repeat Dosing: Repeated administration may trigger unwanted immune responses, limiting long-term use.
- Manufacturing Cost: Scaling production with stringent purity requirements is costly, though AI-driven optimization and advanced platforms are creating efficiencies.
Regulatory Landscape for mRNA Therapeutics
U.S. FDA (CBER)
- IND Application – required before initiating clinical trials.
- BLA Submission – required for full approval, including preclinical, clinical, and manufacturing data.
- EUA – deployed during public health emergencies.
- Post-Market Surveillance – ongoing monitoring via systems such as VAERS.
EMA (Europe)
The EMA treats mRNA as biological medicinal products, with evaluations focused on safety, efficacy, and quality under advanced therapy and vaccine frameworks.
WHO Guidelines
The 2021 WHO ECBS document on regulatory considerations for mRNA vaccines provides a global framework that emphasizes:
- Quality control and manufacturing consistency.
- Recognition of mRNA as a platform technology, allowing learnings from one product to inform the evaluation of others.
National Regulatory Authorities (NRAs) in Asia and the Middle East are aligning their frameworks with FDA, EMA, and WHO guidance to prepare for wider adoption.
Conclusion
mRNA technology is driving a new era of medical innovation, spanning personalized oncology, regenerative therapies, infectious disease prevention, and animal health. Advances in delivery systems, stability, and manufacturing are steadily expanding its clinical utility. As regulatory frameworks continue to evolve in parallel, the coming decade is positioned to see mRNA emerge as a cornerstone of therapeutic development.
How can DDReg help?
At DDReg, we specialize in navigating the regulatory and pharmacovigilance complexities of advanced therapies like mRNA. By combining scientific expertise with regulatory foresight, DDReg helps innovators accelerate approvals and bring next-generation mRNA therapies safely to patients worldwide.
Read more from DDReg experts here: How Translational Biology Is Fast-Tracking Tomorrow’s Therapies