RNA-based Therapies Market to Reach USD 213.54 Billion by 2033, Driven by Rare Diseases, Oncology, and Advances in RNA Technologies | DataM Intelligence

AUSTIN, Texas and TOKYO, Sept. 22, 2025 /PRNewswire/ -- According to DataM Intelligence, "The RNA-Based Therapies Market Size, valued at USD 90.56 billion in 2024, is projected to reach USD 213.54 billion by 2033, expanding at a CAGR of 10.0% during the forecast period".

The growth of the RNA-based therapeutics market is propelled by technological advancements in RNA delivery, rising prevalence of rare genetic disorders, cancers, and infectious diseases, as well as the momentum created by the commercial success of mRNA vaccines. Increased investments, partnerships between pharmaceutical and biotech firms, and pipeline innovation are accelerating clinical adoption and broadening applications of RNA modalities.

However, the industry faces challenges such as high development costs, scalability hurdles, stringent regulatory pathways, and safety concerns. Despite these restraints, opportunities are expanding in oncology, cardiometabolic disorders, immunology, and rare diseases, driven by innovations such as self-amplifying RNA and small activating RNA platforms.

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Market Dynamics

• Driver – Rising Prevalence of Genetic and Rare Diseases:
  With over 7,000 rare diseases affecting nearly 300 million people worldwide, RNA-based therapeutics are increasingly viewed as a transformative treatment option. Modalities such as antisense oligonucleotides (ASOs), siRNAs, and mRNAs are particularly suited to target well-defined genetic mutations and orphan diseases.
• Restraint – Complex Regulatory Pathways and Safety Concerns:
  Stringent regulatory requirements and the need for long-term safety data remain key challenges, often extending development timelines and raising costs for developers.

Market Segmentation Insights

By Type

mRNA Vaccines dominated the market in 2024, accounting for over US$ 36 billion in revenues. Pfizer, Moderna, and BioNTech continue to lead this space, expanding indications beyond infectious diseases into oncology and rare diseases.

Antisense Oligonucleotides (ASOs) generated approximately US$ 20 billion in 2024, with therapies from Sarepta and Biogen addressing neuromuscular and genetic disorders.

RNA Interference (RNAi) therapies, valued at US$ 15 billion, are growing rapidly, led by Alnylam Pharmaceuticals' expanding portfolio for metabolic and rare diseases.

CRISPR/Cas-based RNA editing systems, though still emerging, represented US$ 6.5 billion in 2024, with increasing clinical trial activity focused on rare genetic and hematologic disorders.

RNA Aptamers and Others contributed US$ 13 billion, primarily in oncology and cardiovascular applications.

By Indication

Oncology dominated the indication segment in 2024, generating revenues of US$ 28 billion. RNA therapeutics are increasingly used in precision oncology through mRNA vaccines and RNAi-based silencing of oncogenic pathways.

Rare Genetic Diseases contributed US$ 22 billion, fueled by high-impact therapies for Duchenne muscular dystrophy, spinal muscular atrophy, and transthyretin amyloidosis.

Infectious Diseases remained significant, valued at US$ 19 billion in 2024, as mRNA vaccine platforms expand into influenza, RSV, and emerging pathogens.

Neuromuscular Disorders, driven by ASO-based therapies, contributed US$ 12 billion, while Other indications such as metabolic and ophthalmic diseases accounted for US$ 9 billion.

By End User

Hospitals & Clinics generated over US$ 45 billion in 2024, driven by oncology and rare disease treatments. Pharmaceutical & Biotech Companies accounted for US$ 32 billion, highlighting their dominance in pipeline development and partnerships. Research Institutes, valued at US$ 13 billion, play a key role in clinical validation and technology innovation.

Regional Outlook: USA & Japan

United States

The U.S., valued at US$ 41 billion in 2024, is the largest market for RNA-based therapies, supported by:

• Strong venture capital and government funding for RNA research.
• High prevalence of chronic and rare diseases requiring innovative therapies.
• FDA's expedited approval pathways for RNA-based drugs and vaccines.

Recent trends in 2025 include Pfizer's expansion of its mRNA platform into oncology vaccines and Moderna's collaboration with Merck on personalized cancer vaccines.

Japan

Japan's RNA-based therapies market reached US$ 7.8 billion in 2024 and is projected to grow at double-digit rates. Key growth factors include:

• Increasing government investments in advanced biotechnology.
• Partnerships between Japanese firms and global biopharma for RNAi and ASO therapies.
• PMDA's regulatory flexibility, supporting clinical trials for RNA-based therapies targeting rare and genetic diseases.

In 2025, NS Pharma advanced multiple ASO-based therapies into late-stage trials, reinforcing Japan's leadership in neuromuscular indications.

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Industry Trends & Developments

• Expansion of mRNA Platforms: Moderna, Pfizer, and BioNTech are expanding beyond infectious diseases into oncology, rare genetic conditions, and cardiology.
• RNAi Breakthroughs: Alnylam's approved therapies for ATTR amyloidosis and ongoing trials in hypertension highlight RNAi's clinical potential.
• ASO Advancements: Sarepta's Duchenne muscular dystrophy therapies are setting new standards in rare disease treatment.
• CRISPR Integration: RNA editing systems are increasingly tested for blood disorders, with collaborations between CRISPR Therapeutics, Vertex, and major pharmaceutical players.
• M&A Activity: In 2025, Novo Nordisk announced a US$ 3.5 billion acquisition of an RNA biotech specializing in metabolic diseases, marking one of the largest deals of the year.

Competitive Landscape: Key Companies

Sarepta Therapeutics

A leader in ASO-based therapies, Sarepta's revenue from Duchenne muscular dystrophy treatments surpassed US$ 4 billion in 2024. The company continues to invest heavily in next-gen RNA platforms, including gene-edited RNA therapeutics.

Alnylam Pharmaceuticals

Alnylam dominates the RNAi segment, with products generating US$ 3.2 billion in 2024 revenues. In 2025, the company expanded its ATTR and hypertension portfolio, securing multiple FDA fast-track designations.

Pfizer

Pfizer remains a dominant force in mRNA vaccines, generating US$ 25 billion in 2024 from its COVID-19 and pipeline vaccines. The company is actively advancing oncology-focused mRNA vaccines in partnership with biotech firms.

Moderna Therapeutics

Moderna reported US$ 18 billion in 2024 revenues from its mRNA portfolio, with oncology and rare disease programs leading its post-COVID expansion.

Novartis

Novartis is strategically investing in RNAi and ASOs, with a growing rare disease pipeline. In 2025, the company partnered with Biogen on RNA-based therapies for neurodegenerative diseases.

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Strategic Outlook

The RNA-based Therapies Market is expected to more than double by 2033, supported by:

1. Diversification of RNA Modalities – Expanding beyond mRNA vaccines into RNAi, ASOs, and CRISPR-based platforms.
2. Oncology and Rare Disease Leadership – Both indications will remain the highest-revenue segments.
3. Regional Strength – The U.S. will drive volume growth, while Japan's regulatory and clinical ecosystem will accelerate Asia-Pacific innovation.
4. Investor Confidence – Large-scale acquisitions and licensing deals signal RNA therapeutics as one of the most attractive areas for biopharma investment.

Recent Developments

• January 2025: Danish biotech Nverna Therapeutics launched with a focus on RNA therapies for severe genetic diseases, starting with Huntington's disease.
• September 2024: Aurora Biosynthetics unveiled an end-to-end GMP solution for RNA therapeutics manufacturing in the Asia-Pacific, covering plasmid DNA, mRNA, and lipid nanoparticles.

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SOURCE DataM Intelligence 4 Market Research LLP