Nucleic Acid Based Therapeutics

Global Nucleic Acid Based Therapeutics Market to Reach US$12.6 Billion by 2030

The global market for Nucleic Acid Based Therapeutics estimated at US$5.4 Billion in the year 2024, is expected to reach US$12.6 Billion by 2030, growing at a CAGR of 15.1% over the analysis period 2024-2030. RNA Interference & Short Interfering RNAs, one of the segments analyzed in the report, is expected to record a 16.3% CAGR and reach US$8.5 Billion by the end of the analysis period. Growth in the Antisense Oligonucleotides segment is estimated at 13.3% CAGR over the analysis period.

The U.S. Market is Estimated at US$1.5 Billion While China is Forecast to Grow at 20.2% CAGR

The Nucleic Acid Based Therapeutics market in the U.S. is estimated at US$1.5 Billion in the year 2024. China, the world`s second largest economy, is forecast to reach a projected market size of US$2.8 Billion by the year 2030 trailing a CAGR of 20.2% over the analysis period 2024-2030. Among the other noteworthy geographic markets are Japan and Canada, each forecast to grow at a CAGR of 11.0% and 13.6% respectively over the analysis period. Within Europe, Germany is forecast to grow at approximately 12.0% CAGR.

Global Nucleic Acid Based Therapeutics Market - Key Trends & Drivers Summarized

How Are Nucleic Acid Therapeutics Redefining the Future of Disease Treatment?
Nucleic acid based therapeutics are reshaping modern medicine by enabling precise manipulation of gene expression and cellular function. These therapies, which include antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), aptamers, and messenger RNAs (mRNAs), offer targeted intervention at the genetic level, bypassing conventional protein-focused approaches. Unlike traditional drugs that modulate protein pathways post-translation, nucleic acid therapeutics intervene earlier-at the transcription or translation stage-enabling profound specificity in correcting genetic dysfunctions.

This transformative approach has opened doors to treating previously “undruggable” targets such as non-coding RNAs, gene fusions, and rare monogenic diseases. Therapies like nusinersen (for spinal muscular atrophy) and patisiran (for hereditary transthyretin-mediated amyloidosis) have validated the clinical viability of ASOs and siRNAs. Meanwhile, mRNA technologies-propelled to global attention by COVID-19 vaccines-are now being repurposed for oncology, autoimmune conditions, and personalized vaccines. This shift marks a transition from broad-spectrum interventions to highly individualized, gene-level medicine.

What Technological Advances Are Supporting Clinical Translation and Delivery?
The success of nucleic acid therapies hinges on delivery technologies that protect these fragile molecules and enable cell-specific uptake. Lipid nanoparticles (LNPs) have emerged as the most scalable and clinically validated carriers, particularly for mRNA and siRNA drugs. These LNPs shield therapeutic payloads from degradation, facilitate cellular entry via endocytosis, and trigger controlled release in the cytosol. Improvements in ionizable lipid design, PEGylation strategies, and targeting ligands have significantly boosted the efficacy and safety of LNP systems.

Beyond LNPs, polymer-based nanoparticles, dendrimers, exosomes, and conjugated delivery using GalNAc (for liver targeting) are expanding the range of administration routes and target tissues. For example, GalNAc-conjugated siRNAs have enabled subcutaneous administration for chronic liver conditions with monthly dosing schedules. Additionally, electroporation, viral vectors (AAV, lentivirus), and novel hydrogels are being tested for localized or tissue-selective delivery in oncology and CNS disorders. Bioinformatics tools now play a pivotal role in optimizing sequence design to avoid off-target effects and enhance intracellular stability.

Simultaneously, synthetic chemistry advancements are enhancing the stability, affinity, and immunogenicity profiles of oligonucleotide sequences. Backbone modifications (e.g., phosphorothioate), sugar ring alterations (2’-O-methyl, 2’-fluoro), and locked nucleic acid (LNA) architectures have improved pharmacokinetics and minimized immune activation. These advancements are enabling more frequent dosing, longer circulation half-lives, and reduced toxicity profiles-critical factors in expanding clinical adoption and regulatory acceptance.

Which Therapeutic Areas and Companies Are Driving Market Penetration?
Rare genetic diseases, oncology, and infectious diseases are at the forefront of nucleic acid therapeutic adoption. In rare diseases, where traditional small molecules often fall short, ASOs and siRNAs have demonstrated remarkable efficacy by restoring or silencing faulty gene products. Disorders such as Duchenne muscular dystrophy, amyotrophic lateral sclerosis (ALS), and Batten disease are among the key targets under active clinical investigation. The orphan drug designation and accelerated approval pathways in the U.S. and Europe are enabling faster market entry for developers in this segment.

Oncology represents a fast-emerging application area, with nucleic acid therapeutics being employed in gene silencing, neoantigen vaccine development, and immune cell reprogramming. Personalized mRNA cancer vaccines, developed in collaboration between pharma giants like Moderna and Merck, are advancing into late-stage trials for melanoma and lung cancer. Simultaneously, RNA interference platforms are being tailored to silence tumor drivers such as KRAS and STAT3. Delivery challenges in solid tumors remain a hurdle, but intratumoral and local delivery strategies are improving drug distribution and efficacy.

Leading players such as Ionis Pharmaceuticals, Alnylam, Moderna, BioNTech, and Sarepta Therapeutics dominate the landscape, leveraging deep expertise in oligonucleotide chemistry and delivery. Emerging biotechs are also innovating at the intersection of nucleic acid therapy and AI-driven drug design. Markets such as China and South Korea are accelerating homegrown development through policy support and IP harmonization, increasing regional participation in the global therapeutic pipeline.

What Is Fueling Growth in the Global Nucleic Acid Based Therapeutics Market?
The growth in the global nucleic acid based therapeutics market is driven by several factors, including rising genetic disease awareness, rapid vaccine platform development, and the growing demand for precision medicine. Increased understanding of human genomics and transcriptomics has enabled better identification of disease targets and biomarkers, expanding the pool of treatable indications through nucleic acid interventions.

Clinical success stories have reduced stakeholder skepticism and accelerated investments across preclinical and clinical stages. Strategic collaborations, licensing deals, and venture capital funding are flowing into RNA-focused startups and delivery technology platforms. The success of COVID-19 mRNA vaccines served as a global validation event, prompting healthcare systems and regulators to prioritize scalable and rapid-response nucleic acid therapies for future pandemics, cancer, and emerging diseases.

Moreover, regulatory agencies have established clearer pathways and expedited review programs for these therapies, especially when targeting life-threatening conditions with high unmet needs. Reimbursement models are evolving to accommodate the high costs of personalized nucleic acid therapies, supported by outcome-based pricing and value-based contracting. With expanding manufacturing capacity, digital drug design, and increasing therapeutic diversity, nucleic acid based therapeutics are transitioning from niche biotech applications to a foundational pillar of 21st-century medicine.

SCOPE OF STUDY:

The report analyzes the Nucleic Acid Based Therapeutics market in terms of units by the following Segments, and Geographic Regions/Countries:

Segments:
Product (RNA Interference & Short Interfering RNAs, Antisense Oligonucleotides, Other Products); Application (Autoimmune Disorders Application, Infectious Diseases Application, Genetic Disorders Application, Cancer Application, Other Applications) End-User (Hospitals & Clinics End-User, Academic & Research Institutes End-User)

Geographic Regions/Countries:
World; United States; Canada; Japan; China; Europe (France; Germany; Italy; United Kingdom; Spain; Russia; and Rest of Europe); Asia-Pacific (Australia; India; South Korea; and Rest of Asia-Pacific); Latin America (Argentina; Brazil; Mexico; and Rest of Latin America); Middle East (Iran; Israel; Saudi Arabia; United Arab Emirates; and Rest of Middle East); and Africa.

Select Competitors (Total 48 Featured) -

• Alnylam Pharmaceuticals
• Anima Biotech
• Arrowhead Pharmaceuticals
• BioNTech SE
• BridgeBio Pharma
• CureVac N.V.
• Dicerna Pharmaceuticals
• Dynavax Technologies
• Gilead Sciences Inc.
• Ionis Pharmaceuticals
• Moderna Inc.
• NOXXON Pharma N.V.
• Otsuka Pharmaceutical Co., Ltd.
• Pfizer Inc.
• Phio Pharmaceuticals Corp.
• Precision BioSciences
• ProQR Therapeutics N.V.
• Sarepta Therapeutics
• Silence Therapeutics
• Wave Life Sciences

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