5 Ways mRNA Is Revolutionizing Drug Development Beyond Vaccines

Messenger RNA or mRNA strand 3D rendering illustration with copy space. Genetics, science, medical research, genome replication concepts | Image Credit: © Matthieu - stock.adobe.com

COVID-19 vaccines from Pfizer-BioNTech and Moderna showed that mRNA can be deployed at scale and speed. In May 2024, the FDA approved Moderna’s RSV vaccine (mRESVIA) for adults over 60—signaling growing confidence in mRNA’s safety and versatility beyond COVID. That success unlocked massive investment into mRNA as a modular therapeutic platform. Companies likeBioNTech, CureVac, eTheRNA, and Ethris are leading innovation in both immunogenic and non-immunogenic RNA therapies. Major players are forming high-stakes partnerships, including:

• BioNTech + Bristol Myers Squibb: $1.5B deal for next-gen bispecifics
• CureVac + GSK, CRISPR Therapeutics, MD Anderson for cancer vaccines and CRISPR-based therapeutics in select disease areas.
• eTheRNA + Dropshot Therapeutics: $950M deal for cardiac/renal mRNA drugs

With an expected CAGR of 1.2%, the mRNA therapeutics market is forecasted to hit $71.7 billion by 2034.From cancer immunotherapies to CRISPR-based gene editing, here’s how mRNA is driving the next generation of medicine:

1. Emerging mRNA formats

Beyond non-replicating mRNA, newer constructs like self-amplifying mRNAs, or saRNAs, and circular RNAs, or circRNAs, are showing increased durability, reduced immunogenicity, and improved expression. Companies and institutions working on saRNAs include MiNA Therapeutics, Nature’s Toolbox, Virogin Biotech Limited, with Orna Therapeutics, Shanghai CirCode Biomedicine, and multiple university groups among those working on circRNAs.

2. Cancer immunotherapy gets a boost

Companies like BioNTech, CureVac, and eTheRNA are developing multivalent, personalized mRNA cancer vaccines. Some trials combine these with checkpoint inhibitors like PD-1 blockers to enhance tumor-specific T-cell activation.

3. Gene editing powered by mRNA

Intellia Therapeutics’ NTLA-2001 uses an mRNA-LNP system to deliver CRISPR-Cas9 in vivo, demonstrating that mRNA can enable direct, systemic gene editing.

4. Next-gen delivery systems

Lipid nanoparticles were just the start. Innovations now include lipoplexes,polyplexes, andSNaPL (lipidoid nanoparticle for inhalation). These are improving tissue targeting, reducing degradation, and expanding delivery options like nasal sprays and inhalers.

5. Expanding non-immunogenic applications

mRNA is being used for protein replacement therapy, regenerative medicine, and rare disease treatment, such as for hereditary transthyretin amyloidosis.

Bottom line

mRNA has moved far beyond the hype. For those working in biotech R&D, drug discovery, or manufacturing, it's time to think of mRNA not as a product—but as a platform that’s rewriting the rules of medicine.

*The above is a summary of the article “Looking Beyond mRNA-based COVID-19 Vaccines to Innovative Therapeutics,” available in full on our sister website PharmTech.com!