アンチセンスオリゴヌクレオチドの世界市場 (2025年～2033年)

世界のアンチセンスオリゴヌクレオチド市場は、2024年に29億8,597万米ドルに達し、2033年には52億8,696万米ドルに達すると予測され、予測期間（2025年～2033年）のCAGRは6.5%で成長する見込みです。

エグゼクティブサマリー

アンチセンスオリゴヌクレオチド（ASO）は、相補的な核酸配列に結合し、その機能に影響を与える合成RNA分子です。病気の原因となる遺伝子やその変異体を直接制御する新たな薬剤クラス別であり、従来のタンパク質特異的治療に代わる治療法を提供します。

市場力学：

促進要因と抑制要因

遺伝性疾患の有病率の上昇

遺伝性疾患、特にDMDとSMAの急激な増加は、世界のASO市場の開拓に影響を与える重要な要因です。例えば、希少疾患の罹患者数は少ないですが、その種類は7000を超え、世界の負担は大きいです。特に低所得国では、3億人が希少疾病に罹患しています。遺伝的原因が80%を占め、小児期では70%を占め、診断には4～8年かかります。

遺伝性希少疾患の有病率が高く、世界全体で3億人が罹患しているため、アンチセンスオリゴヌクレオチド（ASO）のような標的療法を使用する必要があります。希少疾患の80％が遺伝性であり、診断に時間がかかることから、ASOは有望な治療選択肢となります。

高い開発コストと規制上のハードル

大きな可能性を秘めているにもかかわらず、ASOの開発には莫大な費用がかかり、高く厳しい規制に従わなければなりません。臨床試験にはかなりの予算がかかり、安全性の問題に集中するため承認への道筋は複雑です。このため、中小のバイオテクノロジー企業の参入は最終的に制限され、全体的な成長は減速します。例えば、脊髄性筋萎縮症（SMA）治療では、1回の注射に12万5,000米ドルかかるASOが使われ、その後、初期費用75万米ドル、年間費用37万5,000米ドルがかかります。

目次

第1章 分析手法と分析範囲

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
    • 遺伝性疾患の罹患率の上昇
    • RNAベースの治療法とドラッグデリバリーシステムの進歩
  • 抑制要因
    • 高い開発コストと規制上のハードル
    • 発展途上地域での認知度・普及率の制約
  • 機会
    • 創薬における人工知能と計算生物学の導入増加
  • 影響分析

第5章 戦略的洞察と業界展望

• 市場のリーダーと先駆者
  • 新たな先駆者と主要企業
  • 最大の売上を誇るブランドを確立したリーダー
  • 確立された製品を持つ市場リーダー
• CXOの視点
• 最新の開発とブレークスルー
• ケーススタディ/進行中の研究
• 規制と償還の情勢
  • 北米
  • 欧州
  • アジア太平洋
  • ラテンアメリカ
  • 中東・アフリカ
• 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
• 特許分析
• SWOT分析
• アンメットニーズとギャップ
• 市場参入と拡大のための推奨戦略
• シナリオ分析：ベストケース・ベースケース・ワーストケースの予測
• 価格分析と価格市場力学
• キーオピニオンリーダー (KOL)

第6章 薬剤の種類別

• エテプリルセン
• フォミビルセン
• ミポメルセン
• デフィブロチド
• ヴォラネゾルセン
• インクリシラン
• ジボシラン
• ヌシネルセン
• ルマシラン
• その他

第7章 適応症別

• 成人
• 小児

第8章 地域別

• 北米
  • 米国
  • カナダ
• 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
• ラテンアメリカ
  • メキシコ
  • ブラジル
  • アルゼンチン
  • その他南米
• アジア太平洋
  • 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9章 競合情勢

• 競合シナリオ
• 市況・シェア分析
• M&A分析

第10章 企業プロファイル

• Ionis Pharmaceuticals, Inc
  • 企業概要
  • 製品ポートフォリオ
    • 製品説明
    • 製品の主要業績評価指標（KPI）
    • 製品販売の実績・予測
    • 製品販売量
  • 財務概要
    • 企業の収益
    • 収益シェア：地域別
    • 収益予測
  • 主な発展
    • 企業合併・買収 (M&A)
    • 主な製品開発活動
    • 規制当局の承認等
  • SWOT分析
• Sarepta Therapeutics
• Alnylam Pharmaceuticals, Inc.
• Percheron Therapeutics
• Isarna Therapeutics GmbH.
• Novartis AG
• Jazz Pharmaceuticals
• Akcea Pharma
• Genzyme
• Biogen
• Similar data will be provided for each market player.
• Wave Life Sciences Ltd
  • パイプライン製品の説明
  • 製品の主要業績評価指標（KPI）
  • 主な活動
  • 市場参入のタイムライン
  • 製品普及率
  • 売上予測と予測
• Cenna Biosciences, Inc.
• Amarna Therapeutics B.V.
• Idera Pharmaceuticals, Inc
• 各市場参入企業に対しても同様のデータが提供されます。

第12章 付録

The global antisense oligonucleotides market reached US$ 2,985.97 million in 2024 and is expected to reach US$ 5,286.96 million by 2033, growing at a CAGR of 6.5% during the forecast period 2025-2033.

Executive Summary

Antisense oligonucleotides (ASOs) are synthetic RNA molecules that bind to complementary nucleic acid sequences, affecting their functions. They are an emerging class of drugs that directly regulate disease-causing genes and their variants, offering an alternative to traditional protein-specific therapies.

Market Dynamics: Drivers & Restraints

Rising Prevalence of Genetic Disorders

The exponential rise in genetic disorders, particularly DMD and SMA, is a crucial factor influencing the development of the worldwide ASO market. For instance, rare diseases affect a small number of individuals, but with over 7000 types, the global burden is significant. 300 million people live with rare diseases, especially in low-income countries. Genetic causes account for 80%, and 70% in childhood, and diagnosis takes 4-8 years.

The high prevalence of genetic rare diseases, affecting 300 million people globally, necessitates the use of targeted therapies like antisense oligonucleotides (ASOs). With 80% of rare diseases being genetic and diagnosis time-consuming, ASOs offer a promising treatment option.

High Development Costs and Regulatory Hurdles

In spite of having considerable potential, ASO development incurs huge costs and follows high and mighty stringent regulations, both of which cause serious impediments. Clinical trials involve a pretty large budget, and the pathways for approval are complicated because they concentrate on safety issues. This ultimately limits entry for smaller biotech companies and decelerates overall growth. For instance, the spinal muscular atrophy (SMA) treatment involves an ASO with a cost of $125,000 per injection, followed by a $750,000 initial cost and an annual cost of $375,000 thereafter.

Segment Analysis

The global antisense oligonucleotides market is segmented based on drug type, indication and region.

Drug Type:

Eteplirsen segment is expected to dominate the antisense oligonucleotides with the highest market share

Eteplirsen injection is utilized to treat Duchenne muscular dystrophy (DMD) in patients with a confirmed dystrophin gene mutation, which results in exon 51 skipping, a rare, inherited muscle disease caused by the absence of dystrophin protein.

These are the first antisense oligonucleotide therapy products marketed as (Exondys 51) Eteplirsen by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). The approval of the above product by the U.S. FDA in September 2016 has been heralded as a great leap forward in the medical management of DMD.

This product (Exondys 51) has a revenue of 540.57 million which is qute dominating segment than other drugs involved in this market and indeed expected to dominate during the forecast period.

Geographical Analysis

North America is expected to hold a significant position in the antisense oligonucleotides market share

North America holds a substantial position in the antisense oligonucleotides market and is expected to hold most of the market share owing to highest revenue by the drugs manufactured by different companies. For instance, OXLUMO (Lumasiran) has total revenue of 1,09,839 out of which 38,159 are generated from United States whereas Givosiran (GIVLAARI) has total revenue of 2,19,251 out of which 1,41,954 has been generated from United States.

Hence, this implies United States are expected to dominate due to significant investments, rise in research and developments, collaborations and partnerships and others factors help the overall country and region to dominate during the forecast period.

Also, other factors include, strong investments in R&D, well-established biotechnology infrastructure, and increasing incidences of genetic disorders like Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA).

Competitive Landscape

The major global players in the Antisense Oligonucleotides market include Ionis Pharmaceuticals, Inc. Sarepta Therapeutics, Alnylam Pharmaceuticals, Inc. Percheron Therapeutics, Isarna Therapeutics GmbH, Novartis AG, Jazz Pharmaceuticals, Akcea Pharma, Genzyme and Biogen among others.

Key Developments

• In January 2024, Vanda Pharmaceuticals has received approval from the FDA for an Investigational New Drug application to evaluate VCA-894A (Antisense Oligonucleotide) for treating Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants in the IGHMBP2 gene.

Why Purchase the Report?

• Pipeline & Innovations: Reviews ongoing clinical trials, 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.

Target Audience 2024

• Manufacturers: Pharmaceutical, Medical Device, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Drug Type & 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.

The global antisense oligonucleotides market report delivers a detailed analysis with 61 key tables, more than 59 visually impactful figures, and 220 pages of expert insights, providing a complete view of the market landscape.

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 Drug Type
• 3.2. Snippet by Indication
• 3.3. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Rising Prevalence of Genetic Disorders
    • 4.1.1.2. Advancements in RNA-based therapeutics and drug delivery systems
  • 4.1.2. Restraints
    • 4.1.2.1. High Development Costs and Regulatory Hurdles
    • 4.1.2.2. Limited awareness and adoption in developing regions
  • 4.1.3. Opportunity
    • 4.1.3.1. Increasing adoption of artificial intelligence and computational biology in drug discovery
  • 4.1.4. Impact Analysis

5. Strategic Insights and Industry Outlook

• 5.1. Market Leaders and Pioneers
  • 5.1.1. Emerging Pioneers and Prominent Players
  • 5.1.2. Established leaders with largest selling Brand
  • 5.1.3. Market leaders with established Product
• 5.2. CXO Perspectives
• 5.3. Latest Developments and Breakthroughs
• 5.4. Case Studies/Ongoing Research
• 5.5. Regulatory and Reimbursement Landscape
  • 5.5.1. North America
  • 5.5.2. Europe
  • 5.5.3. Asia Pacific
  • 5.5.4. Latin America
  • 5.5.5. Middle East & Africa
• 5.6. Porter's Five Force Analysis
• 5.7. Pipeline Analysis
• 5.8. Supply Chain Analysis
• 5.9. Patent Analysis
• 5.10. SWOT Analysis
• 5.11. Unmet Needs and Gaps
• 5.12. Recommended Strategies for Market Entry and Expansion
• 5.13. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
• 5.14. Pricing Analysis and Price Dynamics
• 5.15. Key Opinion Leaders

6. By Drug Type

• 6.1. Introduction
  • 6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 6.1.2. Market Attractiveness Index, By Drug Type
• 6.2. Eteplirsen*
  • 6.2.1. Introduction
  • 6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 6.3. Fomivirsen
• 6.4. Mipomersen
• 6.5. Defibrotide
• 6.6. Volanesorsen
• 6.7. Inclisiran
• 6.8. Givosiran
• 6.9. Nusinersen
• 6.10. Lumasiran
• 6.11. Others

7. By Indication

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
  • 7.1.2. Market Attractiveness Index, By Indication
• 7.2. Adult*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Pediatric

8. By Region

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 8.1.2. Market Attractiveness Index, By Region
• 8.2. North America
  • 8.2.1. Introduction
  • 8.2.2. Key Region-Specific Dynamics
  • 8.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 8.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
  • 8.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.2.5.1. U.S.
    • 8.2.5.2. Canada
• 8.3. Europe
  • 8.3.1. Introduction
  • 8.3.2. Key Region-Specific Dynamics
  • 8.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 8.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
  • 8.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.3.5.1. Germany
    • 8.3.5.2. U.K.
    • 8.3.5.3. France
    • 8.3.5.4. Spain
    • 8.3.5.5. Italy
    • 8.3.5.6. Rest of Europe
• 8.4. Latin America
  • 8.4.1. Introduction
  • 8.4.2. Key Region-Specific Dynamics
  • 8.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 8.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
  • 8.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.4.5.1. Mexico
    • 8.4.5.2. Brazil
    • 8.4.5.3. Argentina
    • 8.4.5.4. Rest of South America
• 8.5. Asia-Pacific
  • 8.5.1. Introduction
  • 8.5.2. Key Region-Specific Dynamics
  • 8.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 8.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication
  • 8.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 8.5.5.1. China
    • 8.5.5.2. India
    • 8.5.5.3. Japan
    • 8.5.5.4. South Korea
    • 8.5.5.5. Rest of Asia-Pacific
• 8.6. Middle East and Africa
  • 8.6.1. Introduction
  • 8.6.2. Key Region-Specific Dynamics
  • 8.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Drug Type
  • 8.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Indication

9. Competitive Landscape

• 9.1. Competitive Scenario
• 9.2. Market Positioning/Share Analysis
• 9.3. Mergers and Acquisitions Analysis

10. Company Profiles

Key Market Players

• 10.1. Ionis Pharmaceuticals, Inc*
  • 10.1.1. Company Overview
  • 10.1.2. Product Portfolio
    • 10.1.2.1. Product Description
    • 10.1.2.2. Product Key Performance Indicators (KPIs)
    • 10.1.2.3. Historic and Forecasted Product Sales
    • 10.1.2.4. Product Sales Volume
  • 10.1.3. Financial Overview
    • 10.1.3.1. Company Revenue's
    • 10.1.3.2. Geographical Revenue Shares
    • 10.1.3.3. Revenue Forecasts
  • 10.1.4. Key Developments
    • 10.1.4.1. Mergers & Acquisitions
    • 10.1.4.2. Key Product Development Activities
    • 10.1.4.3. Regulatory Approvals etc.
  • 10.1.5. SWOT Analysis
• 10.2. Sarepta Therapeutics
• 10.3. Alnylam Pharmaceuticals, Inc.
• 10.4. Percheron Therapeutics
• 10.5. Isarna Therapeutics GmbH.
• 10.6. Novartis AG
• 10.7. Jazz Pharmaceuticals
• 10.8. Akcea Pharma
• 10.9. Genzyme
• 10.10. Biogen
• Similar data will be provided for each market player.

Emerging Market Players

• 10.11. Wave Life Sciences Ltd*
  • 10.11.1. Pipeline Products Description
  • 10.11.2. Product Key Performance Indicators (KPIs)
  • 10.11.3. Key Activities
  • 10.11.4. Market Entry Timelines
  • 10.11.5. Product Penetration Rate
  • 10.11.6. Sales Estimation and Projections
• 10.12. Cenna Biosciences, Inc.
• 10.13. Amarna Therapeutics B.V.
• 10.14. Idera Pharmaceuticals, Inc
• Similar data will be provided for each market player.

LIST NOT EXHAUSTIVE

12. Appendix

• 12.1 About Us and Services
• 12.2 Contact Us