核酸治療薬の世界市場-2025～2033年

核酸治療薬の世界市場は、2024年に56億4,000万米ドルに達し、2032年には144億2,000万米ドルに達すると予測され、予測期間2025年のCAGRは10.9％です。

核酸治療薬は、病気の治療や予防のためにDNAやRNAなどの核酸、あるいはこれらの分子を改変したものを使用する治療法の一種です。これらの治療法は、細胞内の遺伝物質を標的として、遺伝子変異を修正したり、遺伝子発現を調節したり、病気の原因となるタンパク質の活性を阻害したりします。

核酸ベースの治療には、遺伝子治療、RNA干渉（RNAi）、アンチセンスオリゴヌクレオチド（ASOs）、メッセンジャーRNA（mRNA）治療など幅広いアプローチがあり、それぞれ異なるタイプの疾患、特にがん、神経疾患、稀な遺伝性疾患など遺伝的な起源を持つ疾患に対処するように設計されています。

市場力学：

促進要因と抑制要因

研究開発投資の増加

研究開発（R&D）への投資の増加は、核酸治療薬市場の成長を牽引する重要な役割を果たすと予想されます。研究開発への資金が増加するにつれて、製薬会社は革新的な核酸ベースの治療法でポートフォリオを拡大し、臨床試験から実際の使用へと進めています。例えば、2022年3月、RNAimmune, Inc.は約2,700万米ドルのシリーズA資金調達ラウンドを確保しました。当社はこの資金を、感染症、がん、希少疾病に焦点を当てたmRNAワクチンと創薬の研究開発を加速させるために使用する予定です。

これらの投資は、学術機関、研究機関、バイオテクノロジー企業間の協力を促し、画期的な治療法の開発を促進する強力なネットワークを構築しています。例えば、2024年8月、NovartisとVersant Venturesは、腎臓病に対する次世代RNA医薬品をターゲットに、シリーズ資金調達と戦略的研究協力資金を合わせて1億5,000万米ドルを拠出し、Borealis Biosciencesを発表しました。

さらに、研究開発努力は、核酸治療薬の有効性を向上させるために不可欠なデリバリー方法、安定性、ターゲティングにおける重要な課題に取り組んでいます。研究開発がより重視される中、市場では、治療の選択肢を一変させ、患者の転帰を大幅に改善する可能性を秘めた画期的な治療法が登場しています。

核酸治療薬を提供する上での課題

核酸治療薬の提供における課題は、効果的に標的を定め、細胞に入り込むことに伴う固有の複雑さにより、市場を大きく制限します。RNAやDNAのような核酸は大きく、負に荷電した分子であるため、体液中のヌクレアーゼによる分解を受けやすく、安定性や生物学的利用可能性を複雑にしています。さらに、効率的な細胞内への取り込みを達成することは大きなハードルです。ウイルスや非ウイルスベクターを含む多くのデリバリーシステムは、生体内での遺伝子導入効率が低く、苦戦しています。

目次

第1章 調査手法と調査範囲

第2章 定義と概要

第3章 エグゼクティブサマリー

第4章 市場力学

• 影響要因
  • 促進要因
    • バイオ医薬品の需要増加
  • 抑制要因
    • 高い開発コスト
  • 機会
  • 影響分析

第5章 産業分析

• ポーターのファイブフォース分析
• サプライチェーン分析
• 価格分析
• 特許分析
• 規制分析
• SWOT分析
• アンメットニーズ

第6章 カテゴリ別

• アンチセンスオリゴヌクレオチド（ASO）
• 小分子干渉RNA（siRNA）
• メッセンジャーRNA（mRNA）
• 自己増幅RNA（saRNA）

第7章 用途別

• がん
• ウイルス感染
• 遺伝性疾患
• その他

第8章 エンドユーザー別

• 病院
• 学術研究機関

第9章 地域別

• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • スペイン
  • イタリア
  • その他欧州地域
• 南米
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋地域
  • 中国
  • インド
  • 日本
  • 韓国
  • その他アジア太平洋地域
• 中東・アフリカ

第10章 競合情勢

• 競合シナリオ
• 市況・シェア分析
• M&A分析

第11章 企業プロファイル

• Ionis Pharmaceuticals
  • 会社概要
  • 製品ポートフォリオと概要
  • 財務概要
  • 主な発展
• Alnylam Pharmaceuticals, Inc.
• Gilead Sciences, Inc.
• Sarepta Therapeutics, Inc.
• Creative Biolabs
• GSK plc
• Novartis AG
• BioNTech SE.
• Silence Therapeutics
• AstraZeneca

第12章 付録

The global nucleic acid therapeutics market reached US$ 5.64 billion in 2024 and is expected to reach US$ 14.42 billion by 2032, growing at a CAGR of 10.9% during the forecast period 2025-2032.

Nucleic acid therapeutics are a class of treatments that involve the use of nucleic acids, such as DNA, RNA, or modified forms of these molecules, to treat or prevent diseases. These therapies work by targeting the genetic material within cells to either correct genetic mutations, modulate gene expression, or inhibit the activity of disease-causing proteins.

Nucleic acid-based treatments can include a wide range of approaches, such as gene therapy, RNA interference (RNAi), antisense oligonucleotides (ASOs), and messenger RNA (mRNA) therapies, each designed to address different types of diseases, particularly those with genetic origins like cancer, neurological disorders, and rare inherited conditions.

Market Dynamics: Drivers & Restraints

Growing Investments in Research and Development

Increasing investments in research and development (R&D) are expected to play a crucial role in driving the growth of the nucleic acid therapeutics market. As funding for R&D rises, pharmaceutical companies are expanding their portfolios with innovative nucleic acid-based treatments, advancing them from clinical trials to real-world use. For instance, in March 2022, RNAimmune, Inc. secured an approximately US$27 million Series A round of financing. The Company intends to use the funding to accelerate its research and development of mRNA vaccine and drug discovery focused on infectious diseases, cancer, and rare diseases.

These investments are encouraging collaboration between academic institutions, research organizations, and biotech firms, creating a strong network that fosters the development of breakthrough therapies. For instance, in August 2024, Novartis and Versant Ventures unveiled Borealis Biosciences with $150 million in combined Series financing and strategic research collaboration funding to target next-generation RNA medicines for kidney diseases.

Additionally, R&D efforts are addressing critical challenges in delivery methods, stability, and targeting, which are essential for improving the effectiveness of nucleic acid therapeutics. With a greater emphasis on R&D, the market is witnessing the emergence of transformative therapies that have the potential to revolutionize treatment options and significantly improve patient outcomes.

Challenges in delivering nucleic acid therapeutics

Challenges in delivering nucleic acid therapeutics significantly restrain the market due to the inherent complexities associated with effectively targeting and entering cells. Nucleic acids, such as RNA and DNA, are large and negatively charged molecules that are vulnerable to degradation by nucleases in bodily fluids, which complicates their stability and bioavailability. Additionally, achieving efficient cellular uptake is a major hurdle; many delivery systems, including viral and non-viral vectors, struggle with low transfection efficiencies in vivo.

Segment Analysis

The global Nucleic Acid Therapeutics market is segmented based on category, application, end-user, and region.

Category:

Antisense Oligonucleotides (ASOs) segment is expected to dominate the global Nucleic Acid Therapeutics share

Antisense oligonucleotides (ASOs) are expected to dominate the nucleic acid therapeutics market due to their unique ability to target and regulate specific genes involved in a variety of diseases, including genetic disorders, cancer, and viral infections. ASOs work by binding to messenger RNA (mRNA) and modifying gene expression, effectively silencing harmful genes or correcting genetic mutations at the molecular level. This precision in targeting makes ASOs a powerful tool for personalized medicine, particularly for conditions that have limited treatment options.

The growing success of ASOs in clinical trials, along with their approval for treating diseases like spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD), is driving their widespread adoption. Additionally, advancements in delivery technologies are improving the effectiveness and safety of ASOs, further boosting their market potential. As more ASO-based therapies receive regulatory approval and new applications are explored, the demand for ASOs is expected to continue rising.

Geographical Analysis

North America is expected to hold a significant position in the global Nucleic Acid Therapeutics share

North America is expected to hold a significant portion of the nucleic acid therapeutics market share. This is due to the high level of investment in biotechnology and pharmaceutical research, and the presence of leading biopharmaceutical companies that are actively developing and commercializing nucleic acid-based therapies.

The presence of a large number of market players is encouraging the players to invest and develop new therapeutics. Several players are investing in the research and development of advanced therapeutics in the region. For instance, in October 2024, Leal Therapeutics, Inc. announced a $45 million financing. The round was led by Newpath Partners, with additional new investors Chugai Venture Fund and Alexandria Venture Investments, in addition to existing investors OrbiMed, Euclidean Capital and PhiFund. This latest financing round follows a previously disclosed $39 million financing.

The increasing prevalence of genetic disorders, cancer, and infectious diseases in North America is expected to create a strong demand for innovative and personalized treatment options, including nucleic acid therapeutics.

Asia Pacific is at the fastest-growing pace in the global Nucleic Acid Therapeutics market share

The Asia-Pacific region is experiencing the fastest growth in the global nucleic acid therapeutics market, driven by several key factors. Rapid advancements in biotechnology and an increasing focus on innovative medical treatments are positioning the region as a hub for cutting-edge research and development.

The growing prevalence of genetic disorders, cancer, and infectious diseases, coupled with a rising demand for personalized medicine, is fuelling the adoption of nucleic acid-based therapies. Additionally, significant investments in healthcare infrastructure, particularly in countries like China, Japan, and India, are enabling greater access to advanced therapeutics.

Competitive Landscape

The major global players in the market include Ionis Pharmaceuticals, Alnylam Pharmaceuticals, Inc., Gilead Sciences, Inc., AstraZeneca, Sarepta Therapeutics, Inc., Creative Biolabs, GSK plc, Novartis AG, BioNTech SE., and Silence Therapeutics among others.

Why Purchase the Report?

• Pipeline & Innovations: Reviews ongoing clinical trials, and product pipelines, and forecasts upcoming advancements in medical devices and pharmaceuticals.
• Product Performance & Market Positioning: Analyzes product performance, market positioning, and growth potential to optimize strategies.
• Real-World Evidence: Integrates patient feedback and data into product development for improved outcomes.
• Physician Preferences & Health System Impact: Examines healthcare provider behaviors and the impact of health system mergers on adoption strategies.
• Market Updates & Industry Changes: Covers recent regulatory changes, new policies, and emerging technologies.
• Competitive Strategies: Analyzes competitor strategies, market share, and emerging players.
• Pricing & Market Access: Reviews pricing models, reimbursement trends, and market access strategies.
• Market Entry & Expansion: Identifies optimal strategies for entering new markets and partnerships.
• Regional Growth & Investment: Highlights high-growth regions and investment opportunities.
• Supply Chain Optimization: Assesses supply chain risks and distribution strategies for efficient product delivery.
• Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
• Post-market Surveillance: Uses post-market data to enhance product safety and access.
• Pharmacoeconomics & Value-Based Pricing: Analyzes the shift to value-based pricing and data-driven decision-making in R&D.

The Global Nucleic Acid Therapeutics Market report delivers a detailed analysis with 60+ key tables, more than 50 visually impactful figures, and 176 pages of expert insights, providing a complete view of the market landscape.

Target Audience 2023

• Manufacturers: Pharmaceutical, Medical Device, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Technology & Innovation: AI/Robotics Providers, R&D Professionals, Clinical Trial Managers, Pharmacovigilance Experts.
• Investors: Healthcare Investors, Venture Fund Investors, Pharma Marketing & Sales.
• Consulting & Advisory: Healthcare Consultants, Industry Associations, Analysts.
• Supply Chain: Distribution and Supply Chain Managers.
• Consumers & Advocacy: Patients, Advocacy Groups, Insurance Companies.
• Academic & Research: Academic Institutions.

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Category
• 3.2. Snippet by Application
• 3.3. Snippet by End-User
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Rising demand for biopharmaceuticals
  • 4.1.2. Restraints
    • 4.1.2.1. High development costs
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Patent Analysis
• 5.5. Regulatory Analysis
• 5.6. SWOT Analysis
• 5.7. Unmet Needs

6. By Category

• 6.1. Introduction
  • 6.1.1. Analysis and Y-o-Y Growth Analysis (%), By Category
  • 6.1.2. Market Attractiveness Index, By Category
• 6.2. Antisense oligonucleotides (ASOs)*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Small interfering RNA (siRNA)
• 6.4. Messenger RNA (mRNA)
• 6.5. Self-amplifying RNA (saRNA)

7. By Application

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 7.1.2. Market Attractiveness Index, By Application
• 7.2. Cancers*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Viral Infections
• 7.4. Genetic Disorders
• 7.5. Others

8. By End-User

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
  • 8.1.2. Market Attractiveness Index, By End-User
• 8.2. Hospitals*
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 8.3. Academic & Research Institutes

9. By Region

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 9.1.2. Market Attractiveness Index, By Region
• 9.2. North America
  • 9.2.1. Introduction
  • 9.2.2. Key Region-Specific Dynamics
  • 9.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Category
  • 9.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 9.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
  • 9.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 9.2.6.1. U.S.
    • 9.2.6.2. Canada
    • 9.2.6.3. Mexico
• 9.3. Europe
  • 9.3.1. Introduction
  • 9.3.2. Key Region-Specific Dynamics
  • 9.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Category
  • 9.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 9.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
  • 9.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 9.3.6.1. Germany
    • 9.3.6.2. U.K.
    • 9.3.6.3. France
    • 9.3.6.4. Spain
    • 9.3.6.5. Italy
    • 9.3.6.6. Rest of Europe
• 9.4. South America
  • 9.4.1. Introduction
  • 9.4.2. Key Region-Specific Dynamics
  • 9.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Category
  • 9.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 9.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
  • 9.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 9.4.6.1. Brazil
    • 9.4.6.2. Argentina
    • 9.4.6.3. Rest of South America
• 9.5. Asia-Pacific
  • 9.5.1. Introduction
  • 9.5.2. Key Region-Specific Dynamics
  • 9.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Category
  • 9.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 9.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User
  • 9.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 9.5.6.1. China
    • 9.5.6.2. India
    • 9.5.6.3. Japan
    • 9.5.6.4. South Korea
    • 9.5.6.5. Rest of Asia-Pacific
• 9.6. Middle East and Africa
  • 9.6.1. Introduction
  • 9.6.2. Key Region-Specific Dynamics
  • 9.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Category
  • 9.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
  • 9.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End-User

10. Competitive Landscape

• 10.1. Competitive Scenario
• 10.2. Market Positioning/Share Analysis
• 10.3. Mergers and Acquisitions Analysis

11. Company Profiles

• 11.1. Ionis Pharmaceuticals*
  • 11.1.1. Company Overview
  • 11.1.2. Product Portfolio and Description
  • 11.1.3. Financial Overview
  • 11.1.4. Key Developments
• 11.2. Alnylam Pharmaceuticals, Inc.
• 11.3. Gilead Sciences, Inc.
• 11.4. Sarepta Therapeutics, Inc.
• 11.5. Creative Biolabs
• 11.6. GSK plc
• 11.7. Novartis AG
• 11.8. BioNTech SE.
• 11.9. Silence Therapeutics
• 11.10. AstraZeneca

LIST NOT EXHAUSTIVE

12. Appendix

• 12.1. About Us and Services
• 12.2. Contact Us