Next-Generation RNA Therapeutics Market: Transforming Drug Discovery and Precision Medicine

Opportunities Ahead

• Circular RNA (circRNA): Emerging as a stable and versatile therapeutic with potential in gene regulation.

• AI in Drug Discovery: Artificial intelligence is accelerating RNA sequence design and therapeutic target discovery.

• Personalized Vaccines: Advancements in mRNA are paving the way for customized cancer vaccines tailored to individual patients.

• Manufacturing Innovations: Investment in RNA manufacturing technologies is reducing production costs and improving scalability.

Leading Companies of the Next-Generation RNA Therapeutics Market

• Moderna, Inc.

• BioNTech SE

• Alnylam Pharmaceuticals

• Ionis Pharmaceuticals

• Arrowhead Pharmaceuticals

• Arcturus Therapeutics

• CureVac AG

These companies are heavily investing in RNA platforms, clinical trials, and global collaborations to solidify their market positions.

Recent Development

• In May 2025, AbbVie and ADARx Pharmaceuticals announced a collaboration and license option agreement to develop small interfering RNA (siRNA) therapeutics targeting neuroscience, immunology, and oncology. siRNA molecules regulate gene expression at the mRNA level, preventing the production of disease-causing proteins. This partnership underscores AbbVie’s strategy to expand its RNA therapeutics portfolio and ADARx’s expertise in late clinical-stage RNA technologies.
• In May 2027, Biogen Inc. entered a strategic collaboration with City Therapeutics, a privately held biopharma advancing RNA interference (RNAi)-based medicines. The partnership will focus on developing novel RNAi therapies for central nervous system (CNS) diseases, combining City’s RNAi trigger engineering with Biogen’s proprietary drug delivery technologies. Biogen will lead IND-enabling studies, global clinical development, and commercialization.
• In July 2025, Inverna Therapeutics, a Denmark-based biotech co-founded by the University of Southern Denmark and Argobio, officially launched as a specialist in next-generation RNA therapeutics. The company’s lead program focuses on Huntington’s disease, supported by its core platform in sequence-based splice modulation.
• In January 2025, Argobio announced the formation of Inverna Therapeutics in collaboration with the University of Southern Denmark. The company will concentrate on severe genetic diseases, beginning with Huntington’s disease. Inverna’s proprietary platform, developed from the research of Professor Brage Storstein Andresen, focuses on splice modulation technology. The approach is expected to deliver safer, more targeted RNA-based therapies compared with conventional antisense and RNA drugs, creating new opportunities for rare disease treatment.

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 Future Outlook

The Next-Generation RNA Therapeutics Market is poised to redefine modern medicine. By enabling disease-modifying treatments and precision-based therapies, RNA-based innovations are expected to shift healthcare from symptom management to genetic-level cures.

As AI-driven drug discovery, advanced manufacturing, and global collaborations converge, the next decade promises groundbreaking solutions that could transform healthcare accessibility worldwide.

Market Segmentation & Comparison

Below is a comparison table highlighting different segments of the RNA therapeutics market:

Next-Generation RNA Therapeutics Market Segmentation Insights:

The Next-Generation RNA Therapeutics Market is segmented across multiple dimensions, reflecting the diversity of therapeutic approaches, applications, and delivery mechanisms:

By Therapy Type

• mRNA (Messenger RNA): Dominates due to vaccine success and rising applications in oncology.

• siRNA (Small Interfering RNA): Strong adoption for genetic disorders and rare diseases.

• Antisense Oligonucleotides (ASO): Used for precision treatment in neurology and cardiovascular disorders.

• Circular RNA (circRNA): Gaining momentum as a next-gen, stable therapeutic with high durability.

• RNA Editing & Others: Emerging fields expected to reshape genetic-level treatment.

By Application

• Oncology: Largest share, driven by RNA-based cancer vaccines and immunotherapies.

• Infectious Diseases: mRNA vaccines for flu, COVID variants, and other viral infections.

• Cardiovascular Disorders: RNA-based therapies addressing genetic drivers of heart disease.

• Neurology: Rapidly expanding in treatments for Alzheimer’s, Parkinson’s, and rare neurological conditions.

• Rare Genetic Disorders: High growth potential with precision-targeted RNA drugs.

By Delivery Mechanism

• Lipid Nanoparticles (LNPs): Widely used for mRNA vaccine delivery and systemic administration.

• Polymeric Carriers: Under research for controlled and sustained RNA release.

• Viral Vectors: Applied in certain RNA editing and gene modulation therapies.

• Other Nanocarriers: Exploring biocompatible and next-gen delivery solutions.

By End User

• Biopharmaceutical & Biotechnology Companies – Lead in commercialization and product launches.

• Hospitals & Specialty Clinics – Major consumers for advanced therapies.

• Research & Academic Institutes – Key drivers in clinical trial pipelines.