Global Antisense & RNAi Therapeutics Market Growth, Share, Size, Trends and Forecast (2025 - 2031)
By Technology;
RNA Interference and Antisense RNABy Indication;
Autosomal Recessive Disease, Autosomal Dominant Disease, Chromosomal Disease, and OthersBy Route of Administration;
Pulmonary Delivery, Intravenous Injections, Intra-Dermal Injections, Intraperitoneal Injections, Topical Delivery, and OthersBy Geography;
North America, Europe, Asia Pacific, Middle East & Africa, and Latin America - Report Timeline (2021 - 2031)Antisense & RNAi Therapeutics Market Overview
Antisense & RNAi Therapeutics Market (USD Million)
Antisense & RNAi Therapeutics Market was valued at USD 3,560.80 million in the year 2024. The size of this market is expected to increase to USD 14,324.05 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 22.0%.
Global Antisense & RNAi Therapeutics Market Growth, Share, Size, Trends and Forecast
*Market size in USD million
CAGR 22.0 %
Study Period | 2025 - 2031 |
---|---|
Base Year | 2024 |
CAGR (%) | 22.0 % |
Market Size (2024) | USD 3,560.80 Million |
Market Size (2031) | USD 14,324.05 Million |
Market Concentration | Low |
Report Pages | 373 |
Major Players
- Olix Pharmaceuticals
- GlaxoSmithKline Plc
- Sanofi S.A
- Alnylam Pharmaceuticals
- Ionis Pharmaceuticals
- Benitec Biopharma Ltd.
- Acuitas Therapeutics
- Silence Therapeutics Plc
- Enzon Pharmaceuticals Inc
- Antisense Therapeutics Ltd.
- Bio-Path Holdings Inc.
- Gene Signal
- Gradalis
- iCo Therapeutics
- Lorus Therapeutics (Aptose Biosciences)
- Marina Biotech
- miRagen Therapeutics
- Mirna Therapeutics Inc
- OncoGeneX Pharmaceuticals
- Quark Pharmaceuticals
Market Concentration
Consolidated - Market dominated by 1 - 5 major players
Global Antisense & RNAi Therapeutics Market
Fragmented - Highly competitive market without dominant players
The Antisense & RNAi Therapeutics Market is rapidly transforming the landscape of precision medicine through gene-targeted therapies. Its growth is strongly supported by the increasing prevalence of chronic and inherited disorders, with over 60% of RNA-based drugs progressing through various clinical phases. Investments continue to surge as pharmaceutical innovators explore the potential of gene-silencing mechanisms to develop more effective treatments.
Technological Advancements Fueling Growth
The sector benefits from major strides in antisense oligonucleotide and RNA interference platforms, which now account for more than 55% of technological developments in this space. Innovations in chemical stability and delivery vectors have led to more target-specific and durable therapies, boosting clinical outcomes. These advances support a clear shift toward precision-based treatment models.
Rising Disease Burden and Therapeutic Applications
RNA-based therapeutics are becoming essential tools in the fight against serious diseases such as cancer, neurodegenerative disorders, and rare conditions. These treatments have delivered over 45% efficacy enhancements in key preclinical studies. Their capacity to modulate gene function with high specificity is revolutionizing approaches to previously untreatable illnesses.
Regulatory Approvals and Pipeline Expansion
Favorable regulatory dynamics are accelerating product development in this market. Around 35% of RNAi and antisense therapies have secured designations such as fast-track or orphan status, enabling quicker entry into commercial use. Increasing collaborations across the industry are fueling a diversified and expanding drug pipeline, with numerous therapies nearing market launch.
Antisense & RNAi Therapeutics Market Recent Developments
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In August 2023, Sirnaomics Ltd announced that the company had completed the phase I clinical study of STP707, an RNAi therapeutic for treating multiple solid tumors.
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In July 2023, Alnylam Pharmaceuticals, Inc. partnered with Roche to develop and market zilebesiran, an investigational ribonucleic acid interference (RNAi) therapeutic to treat hypertension.
Antisense & RNAi Therapeutics Market Segment Analysis
In this report, the Antisense & RNAi Therapeutics Market has been segmented by Technology, Indication, Route of administration and Geography.
Antisense & RNAi Therapeutics Market, Segmentation by Technology
The Antisense & RNAi Therapeutics Market has been segmented by Technology into RNA interference and Antisense RNA.
RNA Interference (RNAi)
RNA interference holds the larger market share, approximately 60%, due to its potent gene-silencing capabilities. RNAi technology uses small interfering RNA (siRNA) molecules to degrade target mRNA, effectively inhibiting gene expression. This approach is widely researched and applied in treating various genetic disorders and cancers.
Antisense RNA
Antisense RNA accounts for about 40% of the market. This technology involves the use of antisense oligonucleotides (ASOs) that bind to complementary mRNA sequences to block translation or alter splicing. It is utilized in therapies targeting neurological diseases, rare genetic conditions, and other complex disorders.
Antisense & RNAi Therapeutics Market, Segmentation by Indication
The Antisense & RNAi Therapeutics Market has been segmented by Indication into Autosomal Recessive Disease, Autosomal Dominant Disease, Chromosomal Disease and Others.
Autosomal Recessive Disease
Autosomal recessive diseases account for approximately 40% of the market. These genetic disorders require two copies of a mutated gene for the condition to manifest, and antisense & RNAi therapies are being developed to target and silence specific faulty genes, offering promising treatment options.
Autosomal Dominant Disease
Autosomal dominant diseases represent about 30% of the market. These conditions occur when a single copy of a mutated gene causes the disease. Antisense & RNAi therapeutics work by selectively inhibiting the expression of the harmful gene, aiming to alleviate symptoms and progression.
Chromosomal Disease
Chromosomal diseases make up roughly 20% of the market. These disorders arise from structural changes or abnormalities in chromosomes. Emerging RNA-based therapies focus on correcting or compensating for these abnormalities at the molecular level.
Others
The “Others” category includes various complex genetic and acquired diseases, comprising about 10% of the market. Continuous research is expanding the applications of antisense and RNAi therapeutics into new indications.
Antisense & RNAi Therapeutics Market, Segmentation by Route of Administration
The Antisense & RNAi Therapeutics Market has been segmented by Route of Admiistration into Pulmonary delivery, Intravenous injections, Intra-dermal injections, Intraperitoneal injections , Topical delivery and Others.
Pulmonary Delivery
Pulmonary delivery accounts for approximately 30% of the market. This non-invasive route allows for direct administration to the lungs, making it ideal for treating respiratory diseases with enhanced drug absorption and reduced systemic side effects.
Intravenous Injections
Intravenous injections represent about 25% of the market. This route enables rapid and controlled delivery of antisense and RNAi therapeutics into the bloodstream, suitable for systemic treatment of various genetic and acquired diseases.
Intra-dermal Injections
Intra-dermal injections make up roughly 15% of the market. This method targets delivery into the skin layers, facilitating localized treatment with potential applications in skin disorders and vaccine delivery.
Intraperitoneal Injections
Intraperitoneal injections contribute about 10% of the market. Administered into the peritoneal cavity, this route is used mainly in preclinical and some clinical settings for delivering therapeutics with enhanced bioavailability.
Topical Delivery
Topical delivery accounts for approximately 15% of the market. It offers a non-invasive approach to administer RNA-based therapies directly to the affected area, particularly for dermatological conditions.
Others
The “Others” category, including routes such as oral, subcutaneous, and intramuscular, makes up about 5% of the market. Emerging delivery methods are being explored to improve patient compliance and therapeutic outcomes.
Antisense & RNAi Therapeutics Market, Segmentation by Geography
In this report, the Antisense & RNAi Therapeutics Market has been segmented by Geography into five regions; North America, Europe, Asia Pacific, Middle East and Africa and Latin America.
Regions and Countries Analyzed in this Report
Antisense & RNAi Therapeutics Market Share (%), by Geographical Region
North America
North America leads the market with approximately 35% share. The region benefits from strong biotechnology infrastructure, extensive research and development activities, and the presence of major pharmaceutical companies focusing on antisense and RNAi therapeutics.
Europe
Europe accounts for about 28% of the market. Established biotech hubs, supportive regulatory frameworks, and increasing investment in genetic research in countries such as Germany, France, and the UK drive market growth.
Asia Pacific
Asia Pacific holds nearly 22% of the market and is expected to register the fastest growth. Factors include rising biotechnology adoption, expanding healthcare infrastructure, and growing government initiatives in countries like China, India, and Japan.
Middle East and Africa
Middle East and Africa contribute approximately 8% of the market. The growth is supported by improving healthcare facilities, increasing awareness about advanced therapeutics, and expanding pharmaceutical sectors in countries like Saudi Arabia, South Africa, and the UAE.
Latin America
Latin America makes up about 7% of the global market. Increasing investments in healthcare, rising biotechnology research, and growing patient awareness in countries such as Brazil, Argentina, and Mexico fuel market expansion.
Antisense & RNAi Therapeutics Market Trends
This report provides an in depth analysis of various factors that impact the dynamics of Antisense & RNAi Therapeutics Market. These factors include; Market Drivers, Restraints and Opportunities Analysis.
Comprehensive Market Impact Matrix
This matrix outlines how core market forces—Drivers, Restraints, and Opportunities—affect key business dimensions including Growth, Competition, Customer Behavior, Regulation, and Innovation.
Market Forces ↓ / Impact Areas → | Market Growth Rate | Competitive Landscape | Customer Behavior | Regulatory Influence | Innovation Potential |
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Drivers | High impact (e.g., tech adoption, rising demand) | Encourages new entrants and fosters expansion | Increases usage and enhances demand elasticity | Often aligns with progressive policy trends | Fuels R&D initiatives and product development |
Restraints | Slows growth (e.g., high costs, supply chain issues) | Raises entry barriers and may drive market consolidation | Deters consumption due to friction or low awareness | Introduces compliance hurdles and regulatory risks | Limits innovation appetite and risk tolerance |
Opportunities | Unlocks new segments or untapped geographies | Creates white space for innovation and M&A | Opens new use cases and shifts consumer preferences | Policy shifts may offer strategic advantages | Sparks disruptive innovation and strategic alliances |
Drivers, Restraints and Opportunities Analysis
Drivers:
- Increasing Incidence of Chronic Diseases
- Advancements in RNA Interference (RNAi) Technology
- Rising Investments in Biopharmaceutical Research and Development
- Growing Geriatric Population
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Increasing Focus on Precision Medicine - The growing focus on precision medicine is a significant driver of the antisense and RNAi therapeutics market, as healthcare shifts toward highly targeted, gene-specific treatments. Antisense oligonucleotides (ASOs) and RNA interference (RNAi) technologies offer the ability to modulate gene expression with exceptional specificity, making them ideal tools for treating rare genetic disorders, cancer, and other diseases with well-defined molecular origins. These therapies align closely with the objectives of precision medicine by addressing the root cause of diseases at the genetic level rather than merely managing symptoms.
Advancements in genomics, bioinformatics, and biomarker discovery are further accelerating the integration of antisense and RNAi therapies into clinical pipelines. With the support of personalized diagnostics and genetic profiling, researchers and clinicians can now identify patient subsets most likely to benefit from RNA-based treatments. This growing synergy between precision medicine and RNA therapeutics is enhancing therapeutic efficacy, reducing off-target effects, and driving increased investment and innovation in the field, ultimately expanding the market’s growth potential on a global scale.
Restraints:
- Immunogenicity & Short Half-life
- Specificity and Selectivity
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Manufacturing Complexity and Cost - Manufacturing complexity and cost remain significant restraints on the growth of the antisense and RNAi therapeutics market. Producing stable, biologically active oligonucleotides involves highly specialized processes, including chemical synthesis, purification, and formulation, all under stringent regulatory and quality standards. These steps are not only technically demanding but also time-intensive, requiring advanced infrastructure and skilled expertise. The high cost of production often results in expensive end products, limiting affordability and access, particularly in cost-sensitive healthcare systems.
Moreover, large-scale manufacturing and supply chain logistics for RNA-based therapies present additional challenges, such as maintaining product stability, ensuring cold chain requirements, and scaling up production without compromising quality. These barriers can delay commercialization, hinder broader adoption, and discourage smaller biotech firms from entering the market. Addressing these cost and complexity issues is essential to unlock the full therapeutic potential of antisense and RNAi technologies and support their integration into mainstream clinical practice.
Opportunities:
- Expanding Application Scope
- Investment and Partnerships
- Pipeline Development Medicine
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Targeting Undruggable Targets - The ability to target undruggable targets presents a transformative opportunity for the antisense and RNAi therapeutics market. Traditional small-molecule and monoclonal antibody drugs are often limited in their ability to engage intracellular or structurally complex targets, leaving many disease-driving genes beyond therapeutic reach. Antisense and RNAi technologies overcome these limitations by directly modulating gene expression at the RNA level, enabling the silencing or correction of genes that were previously considered inaccessible by conventional approaches.
This capability opens the door to treating a wide range of conditions, including rare genetic disorders, neurodegenerative diseases, metabolic syndromes, and certain cancers, where traditional therapies have failed or shown limited efficacy. RNA-based interventions can be specifically designed to bind to messenger RNA (mRNA) or pre-mRNA, preventing the production of faulty proteins or correcting splicing errors. This precision enhances both efficacy and safety, making antisense and RNAi therapeutics especially valuable in targeting unique or poorly understood molecular mechanisms.
Pharmaceutical and biotech companies are increasingly investing in research programs aimed at identifying novel undruggable targets, supported by advances in genomics, transcriptomics, and high-throughput screening technologies. These tools enable more accurate mapping of disease-associated gene networks and the development of customized RNA molecules to intervene early in the disease pathway. As the understanding of complex diseases deepens, the demand for RNA-based therapeutics is expected to grow in tandem, creating new market avenues and therapeutic categories.
With regulatory agencies showing growing interest in RNA therapies and granting expedited approvals for first-in-class drugs, companies that successfully develop treatments for undruggable targets stand to gain significant market share and clinical recognition. This focus on addressing previously untreatable diseases through RNA-based precision medicine not only expands the therapeutic landscape but also strengthens the long-term innovation potential of the antisense and RNAi therapeutics market.
Antisense & RNAi Therapeutics Market Competitive Landscape Analysis
Key players in Antisense & RNAi Therapeutics Market include:
- Olix Pharmaceuticals
- GlaxoSmithKline Plc
- Sanofi S.A
- Alnylam Pharmaceuticals
- Ionis Pharmaceuticals
- Benitec Biopharma Ltd.
- Acuitas Therapeutics
- Silence Therapeutics Plc
- Enzon Pharmaceuticals Inc
- Antisense Therapeutics Ltd.
- Bio-Path Holdings Inc.
- Gene Signal
- Marina Biotech
- Mirna Therapeutics Inc
- OncoGeneX Pharmaceuticals
- Quark Pharmaceuticals
In this report, the profile of each market player provides following information:
- Company Overview and Product Portfolio
- Market Share Analysis
- Key Developments
- Financial Overview
- Strategies
- Company SWOT Analysis
- Introduction
- Research Objectives and Assumptions
- Research Methodology
- Abbreviations
- Market Definition & Study Scope
- Executive Summary
- Market Snapshot, By Technology
- Market Snapshot, By Indication
- Market Snapshot, By Route of Administration
- Market Snapshot, By Region
- Antisense & RNAi Therapeutics Market Dynamics
- Drivers, Restraints and Opportunities
- Drivers
- Increasing Incidence of Chronic Diseases
- Advancements in RNA Interference (RNAi) Technology
- Rising Investments in Biopharmaceutical Research and Development
- Growing Geriatric Population
- Increasing Focus on Precision Medicine
- Restraints
- Immunogenicity & Short Half-life
- Specificity and Selectivity
- Manufacturing Complexity and Cost
- Opportunities
- Expanding Application Scope
- Investment and Partnerships
- Pipeline Development Medicine
- Targeting Undruggable Targets
- Drivers
- PEST Analysis
- Political Analysis
- Economic Analysis
- Social Analysis
- Technological Analysis
- Porter's Analysis
- Bargaining Power of Suppliers
- Bargaining Power of Buyers
- Threat of Substitutes
- Threat of New Entrants
- Competitive Rivalry
- Drivers, Restraints and Opportunities
- Market Segmentation
- Antisense & RNAi Therapeutics Market, By Technology, 2021 - 2031 (USD Million)
- RNA interference
- Antisense RNA
- Antisense & RNAi Therapeutics Market, By Indication, 2021 - 2031 (USD Million)
- Autosomal Recessive Disease
- Autosomal Dominant Disease
- Chromosomal Disease
- Others
- Antisense & RNAi Therapeutics Market, By Route of Admiistration, 2021 - 2031 (USD Million)
- Pulmonary delivery
- Intravenous injections
- Intra-dermal injections
- Intraperitoneal injections
- Topical delivery
- Others
- Antisense & RNAi Therapeutics Market, By Geography, 2021 - 2031 (USD Million)
- North America
- United States
- Canada
- Europe
- Germany
- United Kingdom
- France
- Italy
- Spain
- Nordic
- Benelux
- Rest of Europe
- Asia Pacific
- Japan
- China
- India
- Australia & New Zealand
- South Korea
- ASEAN (Association of South East Asian Countries)
- Rest of Asia Pacific
- Middle East & Africa
- GCC
- Israel
- South Africa
- Rest of Middle East & Africa
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- North America
- Antisense & RNAi Therapeutics Market, By Technology, 2021 - 2031 (USD Million)
- Competitive Landscape
- Company Profiles
- Olix Pharmaceuticals
- GlaxoSmithKline Plc
- Sanofi S.A
- Alnylam Pharmaceuticals
- Ionis Pharmaceuticals
- Benitec Biopharma Ltd.
- Acuitas Therapeutics
- Silence Therapeutics Plc
- Enzon Pharmaceuticals Inc
- Antisense Therapeutics Ltd.
- Bio-Path Holdings Inc.
- Gene Signal
- Gradalis
- iCo Therapeutics
- Lorus Therapeutics (Aptose Biosciences)
- Marina Biotech
- miRagen Therapeutics
- Mirna Therapeutics Inc
- OncoGeneX Pharmaceuticals
- Quark Pharmaceuticals
- Company Profiles
- Analyst Views
- Future Outlook of the Market