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단일유전질환 치료 시장 보고서(2026년)

Monogenetic Disease Therapy Global Market Report 2026

발행일: | 리서치사: 구분자 The Business Research Company | 페이지 정보: 영문 250 Pages | 배송안내 : 2-10일 (영업일 기준)

    
    
    




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단일유전질환 치료 시장 규모는 최근 몇 년간 크게 성장했습니다. 2025년 283억 달러에서 2026년에는 310억 8,000만 달러에 이르고, CAGR 9.8%로 성장할 전망입니다. 그동안의 성장 요인으로는 인간 유전체 연구의 발전, 유전자 치료의 초기 임상시험, 희귀질환 진단 건수 증가, 희귀질환 치료제(희귀의약품)에 대한 우대 정책, 바이러스 벡터 기술의 발전 등을 꼽을 수 있습니다.

단일유전질환 치료 시장 규모는 향후 몇 년간 급성장이 전망되고 있습니다. 2030년에는 455억 8,000만 달러에 이르고, CAGR은 10.0%를 나타낼 전망입니다. 예측 기간 동안 이러한 성장은 유전자 편집 플랫폼의 확장, 희귀질환 파이프라인의 확대, 유전자 스크리닝 비율 증가, 맞춤치료에 대한 자금 투입 증가, 규제 당국의 승인 범위 확대에 기인하는 것으로 보입니다. 예측 기간의 주요 동향으로는 단일 유전자 표적치료제, 유전자 대체 치료 프로그램, RNA 기반 유전자 치료, 희귀질환 유전자 교정, 정밀유전자 의약품 설계 등을 들 수 있습니다.

정밀의료 및 맞춤의료의 보급 확대는 향후 단일유전질환 치료제 시장의 성장을 견인할 것으로 예측됩니다. 정밀의료 및 맞춤의료는 치료 효과를 높이고 부작용을 줄이기 위해 개인의 고유한 유전적, 분자적 또는 생활습관적 특성에 따라 질병을 예방, 진단, 치료하는 맞춤형 의료 접근법입니다. 정밀의료와 맞춤의료의 성장은 효과를 극대화하고 부작용을 최소화하며 특정 유전적, 분자적 또는 생활습관적 요인에 대응하는 맞춤형 치료를 원하는 의료진과 환자들에 의해 주도되고 있습니다. 단일유전질환 치료는 환자의 특정 유전자 돌연변이를 표적으로 삼아 고도로 맞춤화된 효과적인 치료 솔루션을 가능하게 하여 정밀의료와 맞춤형 의료를 뒷받침하고 있습니다. 예를 들어, 미국 기반 병원 및 의료 기관인 Personalized Medicine Coalition에 따르면, 2024년 2월 기준 FDA는 2023년 희귀질환 환자를 위한 16개의 새로운 맞춤형 치료제를 승인했습니다. 이는 2022년 6건에서 증가한 수치입니다. 따라서 정밀의료 및 맞춤형 의료의 도입 확대는 단일유전질환 치료 시장의 성장을 가속화하고 있습니다.

단일유전질환 치료제 시장에서 사업을 전개하는 주요 기업들은 듀센형 근이영양증(DMD)을 대상으로 하는 프로토콜 등 혁신적인 유전자 치료제 개발에 집중하고 있으며, 질병의 진행을 늦추고 환자의 장기적인 예후를 개선하는 것을 목표로 하고 있습니다. Duchenne 근이영양증 프로토콜은 Duchenne 근이영양증 환자의 진단, 모니터링 및 관리를 위해 특별히 고안된 체계적인 임상 또는 치료 지침으로, 질환의 진행을 늦추고 근육 기능을 개선하며 관련 합병증에 대처하는 데 중점을 두고 있습니다. 중점을 두고 있습니다. 예를 들어, 2026년 1월 미국 소재 바이오 제약사 Sarepta Therapeutics Inc는 4세 이상 보행이 가능한 뒤쉔형 근이영양증(DMD) 환자를 대상으로 하는 'Elevidys'를 출시하였습니다. 회사 측은 외부 대조군 대비 3년 동안 기능 저하 속도가 최대 73% 감소했다고 보고했으며, 이는 질병 경과를 바꾸고 삶의 질(QOL)을 개선하기 위한 조기 개입의 중요성을 강조하고 있습니다.

자주 묻는 질문

  • 단일유전질환 치료 시장 규모는 어떻게 변화하고 있나요?
  • 단일유전질환 치료 시장의 성장 요인은 무엇인가요?
  • 정밀의료 및 맞춤의료가 단일유전질환 치료 시장에 미치는 영향은 무엇인가요?
  • 단일유전질환 치료제 시장에서 주요 기업들은 어떤 방향으로 사업을 전개하고 있나요?
  • Sarepta Therapeutics Inc의 최근 성과는 무엇인가요?

목차

제1장 주요 요약

제2장 시장 특징

제3장 시장 공급망 분석

제4장 세계 시장 동향과 전략

제5장 최종 이용 산업 시장 분석

제6장 시장 : 금리, 인플레이션, 지정학, 무역 전쟁과 관세의 영향, 관세 전쟁과 무역 보호주의의 공급망에 대한 영향, 코로나 팬데믹이 시장에 미치는 영향을 포함한 거시경제 시나리오

제7장 세계 전략 분석 프레임워크, 현재 시장 규모, 시장 비교 및 성장률 분석

제8장 TAM(Total Addressable Market) 규모

제9장 시장 세분화

제10장 지역별/국가별 분석

제11장 아시아태평양 시장

제12장 중국 시장

제13장 인도 시장

제14장 일본 시장

제15장 호주 시장

제16장 인도네시아 시장

제17장 한국 시장

제18장 대만 시장

제19장 동남아시아 시장

제20장 서유럽 시장

제21장 영국 시장

제22장 독일 시장

제23장 프랑스 시장

제24장 이탈리아 시장

제25장 스페인 시장

제26장 동유럽 시장

제27장 러시아 시장

제28장 북미 시장

제29장 미국 시장

제30장 캐나다 시장

제31장 남미 시장

제32장 브라질 시장

제33장 중동 시장

제34장 아프리카 시장

제35장 시장 규제 상황과 투자환경

제36장 경쟁 구도와 기업 개요

제37장 기타 주요 기업 및 혁신 기업

제38장 세계 시장 경쟁 벤치마킹과 대시보드

제39장 시장에서 주목 받는 신생 기업

제40장 주요 인수합병(M&A)

제41장 시장 잠재력이 높은 국가, 부문, 전략

제42장 부록

LSH 26.04.22

Monogenetic disease therapy refers to advanced medical treatments designed to target diseases caused by mutations or defects in a single gene, by correcting, replacing, silencing, or compensating for the abnormal gene or its resulting protein function. These therapies include gene therapy, RNA-based interventions, enzyme replacement therapies, and targeted small-molecule drugs, all aimed at addressing the underlying genetic cause rather than merely managing clinical symptoms.

The primary types of monogenetic disease therapy include pharmacological, surgical, blood transfusion, gene therapies, and other therapy types. Pharmacological therapies refer to treatment approaches that use drugs to control symptoms or alter disease progression in patients affected by single-gene disorders. These therapies are categorized based on inheritance patterns such as autosomal and chromosomal. They are used for conditions including cystic fibrosis, sickle cell anemia, phenylketonuria, and other applications. They are distributed through hospital pharmacies, retail pharmacies, and online pharmacies, and are utilized by end users such as clinics, hospitals, diagnostic centres, home healthcare providers, and other end users.

Tariffs on viral vectors, specialized biologic inputs, and advanced therapy manufacturing materials are affecting the monogenetic disease therapy market by increasing production and development costs. Gene and RNA based therapy segments are most impacted where cross border sourcing of biotech materials and reagents is common, especially across north america and europe. Higher import duties can raise therapy manufacturing expenses and treatment pricing. However, tariffs are also encouraging regional biomanufacturing capacity and local supply chain development. This supports domestic gene therapy producers and specialized biotech suppliers.

The monogenetic disease therapy market research report is one of a series of new reports from The Business Research Company that provides monogenetic disease therapy market statistics, including monogenetic disease therapy industry global market size, regional shares, competitors with a monogenetic disease therapy market share, detailed monogenetic disease therapy market segments, market trends and opportunities, and any further data you may need to thrive in the monogenetic disease therapy industry. This monogenetic disease therapy market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenario of the industry.

The monogenetic disease therapy market size has grown strongly in recent years. It will grow from $28.3 billion in 2025 to $31.08 billion in 2026 at a compound annual growth rate (CAGR) of 9.8%. The growth in the historic period can be attributed to human genome research progress, early gene therapy trials, rise in rare disease diagnosis, orphan drug incentives, advances in viral vectors.

The monogenetic disease therapy market size is expected to see rapid growth in the next few years. It will grow to $45.58 billion in 2030 at a compound annual growth rate (CAGR) of 10.0%. The growth in the forecast period can be attributed to growth in gene editing platforms, expansion of rare disease pipelines, higher genetic screening rates, increased personalized therapy funding, broader regulatory approvals. Major trends in the forecast period include single gene targeted therapies, gene replacement treatment programs, rna based genetic treatments, rare disease gene correction, precision genetic drug design.

The increasing adoption of precision and personalized medicine is expected to drive the growth of the monogenetic disease therapy market going forward. Precision and personalized medicine is a medical approach that customizes disease prevention, diagnosis, and treatment based on an individual's unique genetic, molecular, or lifestyle characteristics to enhance therapeutic effectiveness and reduce adverse effects. The growth of precision and personalized medicine is driven by healthcare providers and patients seeking individualized treatments that maximize efficacy, minimize side effects, and address specific genetic, molecular, or lifestyle factors. Monogenetic disease therapy supports precision and personalized medicine by targeting a patient's specific genetic mutation, enabling highly customized and effective treatment solutions. For instance, in February 2024, according to the Personalized Medicine Coalition, a US-based hospital and health care organization, the FDA approved 16 new personalized treatments for rare disease patients in 2023, up from six approvals in 2022. Therefore, the rising adoption of precision and personalized medicine is accelerating the growth of the monogenetic disease therapy market.

Leading companies operating in the monogenetic disease therapy market are focusing on developing innovative gene therapies, such as Duchenne muscular dystrophy (DMD)-targeted protocols, to slow disease progression and improve long-term patient outcomes. Duchenne muscular dystrophy-targeted protocols are structured clinical or therapeutic guidelines specifically designed to diagnose, monitor, and manage patients with Duchenne muscular dystrophy, focusing on slowing disease progression, improving muscle function, and addressing associated complications. For example, in January 2026, Sarepta Therapeutics Inc, a US-based biopharmaceutical company, launched Elevidys for ambulatory patients aged four and older with DMD, reporting up to a 73% reduction in the rate of functional decline over three years compared with an external control group, highlighting the importance of early intervention to alter disease course and improve quality of life.

In October 2024, Ocuphire Pharma Inc., a US-based clinical-stage ophthalmic biopharmaceutical company, acquired Opus Genetics Inc. for an undisclosed amount. With this acquisition, Ocuphire aimed to establish a leading gene therapy franchise by combining its late-stage drug development expertise with Opus Genetics' pipeline of AAV-based gene therapy candidates targeting conditions such as Leber congenital amaurosis and other inherited retinal disorders. Opus Genetics Inc. is a US-based clinical-stage gene therapy company focused on monogenetic disease therapy, specializing in the development of AAV-based, one-time treatments for rare inherited retinal diseases caused by single-gene mutations.

Major companies operating in the monogenetic disease therapy market are Pfizer Inc., Sanofi S.A., Astellas Pharma Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics Inc., Ptc therapeutics inc., Crispr therapeutics ag, CRISPR Therapeutics, Voyager Therapeutics Inc., Oxford Biomedica plc, REGENXBIO INC., Arcturus therapeutics holdings inc., Editas medicine Inc., American Gene Technologies, UniQure NV., MeiraGTx Limited, Prime medicine, Abeona therapeutics Inc., Blue Cross Blue Shield Association, Rocket pharmaceuticals Inc.

North America was the largest region in the monogenetic disease therapy market in 2025. Asia-Pacific is expected to be the fastest-growing region in the forecast period. The regions covered in the monogenetic disease therapy market report are Asia-Pacific, South East Asia, Western Europe, Eastern Europe, North America, South America, Middle East, Africa.

The countries covered in the monogenetic disease therapy market report are Australia, Brazil, China, France, Germany, India, Indonesia, Japan, Taiwan, Russia, South Korea, UK, USA, Canada, Italy, Spain.

The monogenetic disease therapy market includes revenues earned by entities through gene replacement therapies, gene editing therapies, RNA-based therapies, cell-based gene therapies, and gene silencing or modulation therapies. The market value includes the value of related goods sold by the service provider or included within the service offering. Only goods and services traded between entities or sold to end consumers are included.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD unless otherwise specified).

The revenues for a specified geography are consumption values that are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

Monogenetic Disease Therapy Market Global Report 2026 from The Business Research Company provides strategists, marketers and senior management with the critical information they need to assess the market.

This report focuses monogenetic disease therapy market which is experiencing strong growth. The report gives a guide to the trends which will be shaping the market over the next ten years and beyond.

Reasons to Purchase

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  • Understand customers based on end user analysis.
  • Benchmark performance against key competitors based on market share, innovation, and brand strength.
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Where is the largest and fastest growing market for monogenetic disease therapy ? How does the market relate to the overall economy, demography and other similar markets? What forces will shape the market going forward, including technological disruption, regulatory shifts, and changing consumer preferences? The monogenetic disease therapy market global report from the Business Research Company answers all these questions and many more.

The report covers market characteristics, size and growth, segmentation, regional and country breakdowns, total addressable market (TAM), market attractiveness score (MAS), competitive landscape, market shares, company scoring matrix, trends and strategies for this market. It traces the market's historic and forecast market growth by geography.

  • The market characteristics section of the report defines and explains the market. This section also examines key products and services offered in the market, evaluates brand-level differentiation, compares product features, and highlights major innovation and product development trends.
  • The supply chain analysis section provides an overview of the entire value chain, including key raw materials, resources, and supplier analysis. It also provides a list competitor at each level of the supply chain.
  • The updated trends and strategies section analyses the shape of the market as it evolves and highlights emerging technology trends such as digital transformation, automation, sustainability initiatives, and AI-driven innovation. It suggests how companies can leverage these advancements to strengthen their market position and achieve competitive differentiation.
  • The regulatory and investment landscape section provides an overview of the key regulatory frameworks, regularity bodies, associations, and government policies influencing the market. It also examines major investment flows, incentives, and funding trends shaping industry growth and innovation.
  • The market size section gives the market size ($b) covering both the historic growth of the market, and forecasting its development.
  • The forecasts are made after considering the major factors currently impacting the market. These include the technological advancements such as AI and automation, Russia-Ukraine war, trade tariffs (government-imposed import/export duties), elevated inflation and interest rates.
  • The total addressable market (TAM) analysis section defines and estimates the market potential compares it with the current market size, and provides strategic insights and growth opportunities based on this evaluation.
  • The market attractiveness scoring section evaluates the market based on a quantitative scoring framework that considers growth potential, competitive dynamics, strategic fit, and risk profile. It also provides interpretive insights and strategic implications for decision-makers.
  • Market segmentations break down the market into sub markets.
  • The regional and country breakdowns section gives an analysis of the market in each geography and the size of the market by geography and compares their historic and forecast growth.
  • Expanded geographical coverage includes Taiwan and Southeast Asia, reflecting recent supply chain realignments and manufacturing shifts in the region. This section analyzes how these markets are becoming increasingly important hubs in the global value chain.
  • The competitive landscape chapter gives a description of the competitive nature of the market, market shares, and a description of the leading companies. Key financial deals which have shaped the market in recent years are identified.
  • The company scoring matrix section evaluates and ranks leading companies based on a multi-parameter framework that includes market share or revenues, product innovation, and brand recognition.

Scope

  • Markets Covered:1) By Therapy Type: Pharmacological; Surgical; Blood Transfusion; Gene Therapies; Other Therapy Types
  • 2) By Inheritance Pattern: Autosomal; Chromosomal
  • 3) By Application: Cystic Fibrosis; Sickle Cell Anemia; Phenylketonuria; Other Applications
  • 4) By Distribution Channel: Hospital Pharmacy; Retail Pharmacy; Online Pharmacy
  • 5) By End-Users: Clinics; Hospitals; Diagnostic Centres; Home Healthcare; Other End-Users
  • Subsegments:
  • 1) By Pharmacological: Small Molecule Drugs; Enzyme Replacement Therapies; Protein Replacement Therapies; Immunomodulatory Drugs; Targeted Molecular Therapies
  • 2) By Surgical: Organ Transplant Procedures; Tissue Repair Surgeries; Gene Editing Assisted Surgeries; Minimally Invasive Surgeries; Corrective Surgical Interventions
  • 3) By Blood Transfusion: Red Blood Cell Transfusions; Platelet Transfusions; Plasma Transfusions; Stem Cell Infusions; Autologous Blood Transfusions
  • 4) By Gene Therapies: Ex Vivo Gene Therapy; In Vivo Gene Therapy; Viral Vector Based Gene Therapy; Non Viral Vector Gene Therapy; Genome Editing Based Therapy
  • 5) By Other Therapy Types: Cell Based Therapies; RNA Based Therapies; Protein Based Therapies; Combination Therapies; Experimental Therapies
  • Companies Mentioned: Pfizer Inc.; Sanofi S.A.; Astellas Pharma Inc.; Vertex Pharmaceuticals Incorporated; Sarepta Therapeutics Inc.; Ptc therapeutics inc.; Crispr therapeutics ag; CRISPR Therapeutics; Voyager Therapeutics Inc.; Oxford Biomedica plc; REGENXBIO INC.; Arcturus therapeutics holdings inc.; Editas medicine Inc.; American Gene Technologies; UniQure NV.; MeiraGTx Limited; Prime medicine; Abeona therapeutics Inc.; Blue Cross Blue Shield Association; Rocket pharmaceuticals Inc.
  • Countries: Australia; Brazil; China; France; Germany; India; Indonesia; Japan; Taiwan; Russia; South Korea; UK; USA; Canada; Italy; Spain
  • Regions: Asia-Pacific; South East Asia; Western Europe; Eastern Europe; North America; South America; Middle East; Africa
  • Time Series: Five years historic and ten years forecast.
  • Data: Ratios of market size and growth to related markets, GDP proportions, expenditure per capita,
  • Data Segmentations: country and regional historic and forecast data, market share of competitors, market segments.
  • Sourcing and Referencing: Data and analysis throughout the report is sourced using end notes.
  • Delivery Format: Word, PDF or Interactive Report
  • + Excel Dashboard
  • Added Benefits
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Added Benefits available all on all list-price licence purchases, to be claimed at time of purchase. Customisations within report scope and limited to 20% of content and consultant support time limited to 8 hours.

Table of Contents

1. Executive Summary

  • 1.1. Key Market Insights (2020-2035)
  • 1.2. Visual Dashboard: Market Size, Growth Rate, Hotspots
  • 1.3. Major Factors Driving the Market
  • 1.4. Top Three Trends Shaping the Market

2. Monogenetic Disease Therapy Market Characteristics

  • 2.1. Market Definition & Scope
  • 2.2. Market Segmentations
  • 2.3. Overview of Key Products and Services
  • 2.4. Global Monogenetic Disease Therapy Market Attractiveness Scoring And Analysis
    • 2.4.1. Overview of Market Attractiveness Framework
    • 2.4.2. Quantitative Scoring Methodology
    • 2.4.3. Factor-Wise Evaluation
  • Growth Potential Analysis, Competitive Dynamics Assessment, Strategic Fit Assessment And Risk Profile Evaluation
    • 2.4.4. Market Attractiveness Scoring and Interpretation
    • 2.4.5. Strategic Implications and Recommendations

3. Monogenetic Disease Therapy Market Supply Chain Analysis

  • 3.1. Overview of the Supply Chain and Ecosystem
  • 3.2. List Of Key Raw Materials, Resources & Suppliers
  • 3.3. List Of Major Distributors and Channel Partners
  • 3.4. List Of Major End Users

4. Global Monogenetic Disease Therapy Market Trends And Strategies

  • 4.1. Key Technologies & Future Trends
    • 4.1.1 Biotechnology, Genomics & Precision Medicine
    • 4.1.2 Artificial Intelligence & Autonomous Intelligence
    • 4.1.3 Digitalization, Cloud, Big Data & Cybersecurity
    • 4.1.4 Industry 4.0 & Intelligent Manufacturing
    • 4.1.5 Sustainability, Climate Tech & Circular Economy
  • 4.2. Major Trends
    • 4.2.1 Single Gene Targeted Therapies
    • 4.2.2 Gene Replacement Treatment Programs
    • 4.2.3 Rna Based Genetic Treatments
    • 4.2.4 Rare Disease Gene Correction
    • 4.2.5 Precision Genetic Drug Design

5. Monogenetic Disease Therapy Market Analysis Of End Use Industries

  • 5.1 Hospitals
  • 5.2 Specialty Clinics
  • 5.3 Diagnostic Centres
  • 5.4 Research Hospitals
  • 5.5 Gene Therapy Centers

6. Monogenetic Disease Therapy Market - Macro Economic Scenario Including The Impact Of Interest Rates, Inflation, Geopolitics, Trade Wars and Tariffs, Supply Chain Impact from Tariff War & Trade Protectionism, And Covid And Recovery On The Market

7. Global Monogenetic Disease Therapy Strategic Analysis Framework, Current Market Size, Market Comparisons And Growth Rate Analysis

  • 7.1. Global Monogenetic Disease Therapy PESTEL Analysis (Political, Social, Technological, Environmental and Legal Factors, Drivers and Restraints)
  • 7.2. Global Monogenetic Disease Therapy Market Size, Comparisons And Growth Rate Analysis
  • 7.3. Global Monogenetic Disease Therapy Historic Market Size and Growth, 2020 - 2025, Value ($ Billion)
  • 7.4. Global Monogenetic Disease Therapy Forecast Market Size and Growth, 2025 - 2030, 2035F, Value ($ Billion)

8. Global Monogenetic Disease Therapy Total Addressable Market (TAM) Analysis for the Market

  • 8.1. Definition and Scope of Total Addressable Market (TAM)
  • 8.2. Methodology and Assumptions
  • 8.3. Global Total Addressable Market (TAM) Estimation
  • 8.4. TAM vs. Current Market Size Analysis
  • 8.5. Strategic Insights and Growth Opportunities from TAM Analysis

9. Monogenetic Disease Therapy Market Segmentation

  • 9.1. Global Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Pharmacological, Surgical, Blood Transfusion, Gene Therapies, Other Therapy Types
  • 9.2. Global Monogenetic Disease Therapy Market, Segmentation By Inheritance Pattern, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Autosomal, Chromosomal
  • 9.3. Global Monogenetic Disease Therapy Market, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Cystic Fibrosis, Sickle Cell Anemia, Phenylketonuria, Other Applications
  • 9.4. Global Monogenetic Disease Therapy Market, Segmentation By Distribution Channel, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Hospital Pharmacy, Retail Pharmacy, Online Pharmacy
  • 9.5. Global Monogenetic Disease Therapy Market, Segmentation By End-Users, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Clinics, Hospitals, Diagnostic Centres, Home Healthcare, Other End-Users
  • 9.6. Global Monogenetic Disease Therapy Market, Sub-Segmentation Of Pharmacological, By Type, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Small Molecule Drugs, Enzyme Replacement Therapies, Protein Replacement Therapies, Immunomodulatory Drugs, Targeted Molecular Therapies
  • 9.7. Global Monogenetic Disease Therapy Market, Sub-Segmentation Of Surgical, By Type, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Organ Transplant Procedures, Tissue Repair Surgeries, Gene Editing Assisted Surgeries, Minimally Invasive Surgeries, Corrective Surgical Interventions
  • 9.8. Global Monogenetic Disease Therapy Market, Sub-Segmentation Of Blood Transfusion, By Type, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Red Blood Cell Transfusions, Platelet Transfusions, Plasma Transfusions, Stem Cell Infusions, Autologous Blood Transfusions
  • 9.9. Global Monogenetic Disease Therapy Market, Sub-Segmentation Of Gene Therapies, By Type, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Ex Vivo Gene Therapy, In Vivo Gene Therapy, Viral Vector Based Gene Therapy, Non Viral Vector Gene Therapy, Genome Editing Based Therapy
  • 9.10. Global Monogenetic Disease Therapy Market, Sub-Segmentation Of Other Therapy Types, By Type, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • Cell Based Therapies, RNA Based Therapies, Protein Based Therapies, Combination Therapies, Experimental Therapies

10. Monogenetic Disease Therapy Market Regional And Country Analysis

  • 10.1. Global Monogenetic Disease Therapy Market, Split By Region, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion
  • 10.2. Global Monogenetic Disease Therapy Market, Split By Country, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

11. Asia-Pacific Monogenetic Disease Therapy Market

  • 11.1. Asia-Pacific Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 11.2. Asia-Pacific Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

12. China Monogenetic Disease Therapy Market

  • 12.1. China Monogenetic Disease Therapy Market Overview
  • Country Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 12.2. China Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

13. India Monogenetic Disease Therapy Market

  • 13.1. India Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

14. Japan Monogenetic Disease Therapy Market

  • 14.1. Japan Monogenetic Disease Therapy Market Overview
  • Country Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 14.2. Japan Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

15. Australia Monogenetic Disease Therapy Market

  • 15.1. Australia Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

16. Indonesia Monogenetic Disease Therapy Market

  • 16.1. Indonesia Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

17. South Korea Monogenetic Disease Therapy Market

  • 17.1. South Korea Monogenetic Disease Therapy Market Overview
  • Country Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 17.2. South Korea Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

18. Taiwan Monogenetic Disease Therapy Market

  • 18.1. Taiwan Monogenetic Disease Therapy Market Overview
  • Country Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 18.2. Taiwan Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

19. South East Asia Monogenetic Disease Therapy Market

  • 19.1. South East Asia Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 19.2. South East Asia Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

20. Western Europe Monogenetic Disease Therapy Market

  • 20.1. Western Europe Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 20.2. Western Europe Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

21. UK Monogenetic Disease Therapy Market

  • 21.1. UK Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

22. Germany Monogenetic Disease Therapy Market

  • 22.1. Germany Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

23. France Monogenetic Disease Therapy Market

  • 23.1. France Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

24. Italy Monogenetic Disease Therapy Market

  • 24.1. Italy Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

25. Spain Monogenetic Disease Therapy Market

  • 25.1. Spain Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

26. Eastern Europe Monogenetic Disease Therapy Market

  • 26.1. Eastern Europe Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 26.2. Eastern Europe Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

27. Russia Monogenetic Disease Therapy Market

  • 27.1. Russia Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

28. North America Monogenetic Disease Therapy Market

  • 28.1. North America Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 28.2. North America Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

29. USA Monogenetic Disease Therapy Market

  • 29.1. USA Monogenetic Disease Therapy Market Overview
  • Country Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 29.2. USA Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

30. Canada Monogenetic Disease Therapy Market

  • 30.1. Canada Monogenetic Disease Therapy Market Overview
  • Country Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 30.2. Canada Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

31. South America Monogenetic Disease Therapy Market

  • 31.1. South America Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 31.2. South America Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

32. Brazil Monogenetic Disease Therapy Market

  • 32.1. Brazil Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

33. Middle East Monogenetic Disease Therapy Market

  • 33.1. Middle East Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 33.2. Middle East Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

34. Africa Monogenetic Disease Therapy Market

  • 34.1. Africa Monogenetic Disease Therapy Market Overview
  • Region Information, Market Information, Background Information, Government Initiatives, Regulations, Regulatory Bodies, Major Associations, Taxes Levied, Corporate Tax Structure, Investments, Major Companies
  • 34.2. Africa Monogenetic Disease Therapy Market, Segmentation By Therapy Type, Segmentation By Inheritance Pattern, Segmentation By Application, Historic and Forecast, 2020-2025, 2025-2030F, 2035F, $ Billion

35. Monogenetic Disease Therapy Market Regulatory and Investment Landscape

36. Monogenetic Disease Therapy Market Competitive Landscape And Company Profiles

  • 36.1. Monogenetic Disease Therapy Market Competitive Landscape And Market Share 2024
    • 36.1.1. Top 10 Companies (Ranked by revenue/share)
  • 36.2. Monogenetic Disease Therapy Market - Company Scoring Matrix
    • 36.2.1. Market Revenues
    • 36.2.2. Product Innovation Score
    • 36.2.3. Brand Recognition
  • 36.3. Monogenetic Disease Therapy Market Company Profiles
    • 36.3.1. Pfizer Inc. Overview, Products and Services, Strategy and Financial Analysis
    • 36.3.2. Sanofi S.A. Overview, Products and Services, Strategy and Financial Analysis
    • 36.3.3. Astellas Pharma Inc. Overview, Products and Services, Strategy and Financial Analysis
    • 36.3.4. Vertex Pharmaceuticals Incorporated Overview, Products and Services, Strategy and Financial Analysis
    • 36.3.5. Sarepta Therapeutics Inc. Overview, Products and Services, Strategy and Financial Analysis

37. Monogenetic Disease Therapy Market Other Major And Innovative Companies

  • Ptc therapeutics inc., Crispr therapeutics ag, CRISPR Therapeutics, Voyager Therapeutics Inc., Oxford Biomedica plc, REGENXBIO INC., Arcturus therapeutics holdings inc., Editas medicine Inc., American Gene Technologies, UniQure NV., MeiraGTx Limited, Prime medicine, Abeona therapeutics Inc., Blue Cross Blue Shield Association, Rocket pharmaceuticals Inc.

38. Global Monogenetic Disease Therapy Market Competitive Benchmarking And Dashboard

39. Upcoming Startups in the Market

40. Key Mergers And Acquisitions In The Monogenetic Disease Therapy Market

41. Monogenetic Disease Therapy Market High Potential Countries, Segments and Strategies

  • 41.1 Monogenetic Disease Therapy Market In 2030 - Countries Offering Most New Opportunities
  • 41.2 Monogenetic Disease Therapy Market In 2030 - Segments Offering Most New Opportunities
  • 41.3 Monogenetic Disease Therapy Market In 2030 - Growth Strategies
    • 41.3.1 Market Trend Based Strategies
    • 41.3.2 Competitor Strategies

42. Appendix

  • 42.1. Abbreviations
  • 42.2. Currencies
  • 42.3. Historic And Forecast Inflation Rates
  • 42.4. Research Inquiries
  • 42.5. The Business Research Company
  • 42.6. Copyright And Disclaimer
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