한글목차

세계 안티센스 올리고뉴클레오타이드 시장은 2024년에 29억 8,597만 달러에 달했습니다. 2033년에는 52억 8,696만 달러에 달할 것으로 예측되며, 예측 기간(2025-2033) 동안 6.5%의 연평균 복합 성장률(CAGR)을 보일 것으로 예상됩니다.

주요 요약

안티센스 올리고뉴클레오티드(ASO)는 상보적인 핵산 서열에 결합하여 그 기능에 영향을 미치는 합성 RNA 분자입니다. 질병을 유발하는 유전자나 그 변이체를 직접적으로 조절하는 새로운 약제군으로, 기존의 단백질 특이적 치료제를 대체할 수 있는 치료법을 제공합니다.

시장 역학:

성장 촉진요인과 억제요인

유전성 질환의 유병률 증가

유전성 질환, 특히 DMD와 SMA의 급격한 증가는 세계 ASO 시장 개척에 영향을 미치는 중요한 요인입니다. 예를 들어, 희귀질환의 환자 수는 적지만 그 유형은 7000여 종에 달하고, 전 세계적으로 부담이 큽니다. 특히 저소득 국가에서는 3억 명이 희귀질환을 앓고 있습니다. 유전적 원인이 80%를 차지하고, 소아기에는 70%를 차지하며, 진단에 4-8년이 걸립니다.

유전성 희귀질환의 유병률이 높고, 전 세계적으로 3억 명이 앓고 있기 때문에 항센스 올리고뉴클레오티드(ASO)와 같은 표적치료제를 사용할 필요가 있습니다. 희귀질환의 80%가 유전성이고, 진단에 시간이 오래 걸리기 때문에 ASO는 유망한 치료 옵션이 될 수 있습니다.

높은 개발 비용과 규제 장벽

큰 잠재력을 가지고 있음에도 불구하고, ASO의 개발에는 막대한 비용이 소요되며, 높고 엄격한 규제를 따라야 합니다. 임상시험에는 상당한 예산이 소요되고, 안전성 문제에 집중하기 때문에 승인으로 가는 길은 복잡합니다. 이 때문에 결국 중소 바이오 기업의 진입이 제한되고 전반적인 성장이 둔화될 수밖에 없습니다. 예를 들어, 척수성 근위축증(SMA) 치료의 경우 1회 주사에 12만 5,000달러의 ASO가 사용되며, 이후 75만 달러의 초기 비용과 37만 5,000달러의 연간 비용이 소요됩니다.

목차

제1장 분석 방법이및 분석 범위

제2장 정의와 개요

제3장 주요 요약

제4장 시장 역학

• 영향요인
  • 성장 촉진요인
  • 성장 억제요인
  • 기회
  • 영향 분석

제5장 전략적 인사이트와 업계 전망

• 시장 리더와 선구자
  • 새로운 선구자와 주요 기업
  • 최대 판매를 자랑하는 브랜드를 확립한 리더
  • 확립된 제품을 보유한 시장 리더
• CXO 시점
• 최신 개발과 Break through
• 사례 연구/진행 중인 연구
• 규제와 상환 상황
  • 북미
  • 유럽
  • 아시아태평양
  • 라틴아메리카
  • 중동 및 아프리카
• Porter의 Five Forces 분석
• 공급망 분석
• 특허 분석
• SWOT 분석
• 미충족 요구와 갭
• 시장 진입 및 확장을 위한 추천 전략
• 시나리오 분석 : 베스트 케이스/베이스 케이스/워스트 케이스 예측
• 가격 분석과 가격 시장 역학
• KOL(Key Opinion Leader)

제6장 약제 유형별

• Eteplirsen
• Fomivirsen
• Mipomersen
• Defibrotide
• Volanesorsen
• Inclisiran
• Givosiran
• Nusinersen
• Lumasiran
• 기타

제7장 적응증별

• 성인
• 소아

제8장 지역별

• 북미
  • 미국
  • 캐나다
• 유럽
  • 독일
  • 영국
  • 프랑스
  • 스페인
  • 이탈리아
  • 기타 유럽
• 라틴아메리카
  • 멕시코
  • 브라질
  • 아르헨티나
  • 기타 남미
• 아시아태평양
  • 중국
  • 인도
  • 일본
  • 한국
  • 기타 아시아태평양
• 중동 및 아프리카

제9장 경쟁 구도

• 경쟁 시나리오
• 시장 현황 및 점유율 분석
• 인수합병(M&A) 분석

제10장 기업 개요

• Ionis Pharmaceuticals, Inc
  • 기업 개요
  • 제품 포트폴리오
    • 제품 설명
    • 제품 핵심성과지표(KPI)
    • 제품 판매 실적과 예측
    • 제품 판매량
  • 재무 개요
    • 기업 매출
    • 매출 점유율 : 지역별
    • 매출 예측
  • 주요 발전
    • 인수합병(M&A)
    • 주요 제품 개발 활동
    • 규제기관 승인 등
  • SWOT 분석
• 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
  • 파이프라인 제품 설명
  • 제품 핵심성과지표(KPI)
  • 주요 활동
  • 시장 참여 타임라인
  • 제품 보급률
  • 판매 예측과 예측
• Cenna Biosciences, Inc.
• Amarna Therapeutics B.V.
• Idera Pharmaceuticals, Inc
• 각 시장 진출기업에 대해서도 같은 데이터가 제공됩니다.

제12장 부록

영문목차

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