유전자 치료 시장 규모, 점유율, 성장 및 세계 산업 분석 : 유형별, 용도별, 지역별 인사이트와 예측(2026-2034년)

Growth Factors of gene therapy Market

The global gene therapy market is experiencing strong momentum due to rapid advancements in genetic science and the increasing prevalence of rare and inherited disorders. According to Fortune Business Insights, the gene therapy market was valued at USD 4.29 billion in 2025. The market is projected to grow from USD 5.20 billion in 2026 to USD 11.33 billion by 2034, registering a CAGR of 10.20% during the forecast period. In 2025, North America dominated the market with a share of 24.60%, supported by strong research funding and early regulatory approvals.

Gene therapy involves modifying or replacing defective genes to treat or prevent diseases. These therapies are increasingly used for congenital genetic disorders such as spinal muscular atrophy, hemophilia, B-thalassemia, and inherited retinal diseases, as well as acquired conditions such as leukemia. The rising global burden of genetic and rare diseases is significantly driving demand for curative therapies, thereby supporting market growth.

Market Dynamics

Market Drivers

The rising burden of genetic disorders worldwide is a key driver of the gene therapy market. The growing awareness of gene-based treatments that address the root cause of diseases has increased clinical adoption. According to the World Federation of Hemophilia, 218,804 hemophilia patients were reported globally in 2023, highlighting the unmet need for long-term therapeutic solutions. Increasing research activities and funding for rare disease treatment further accelerate market expansion.

Market Restraints

The high cost of gene therapies remains a major restraint limiting patient access. These therapies often involve complex manufacturing processes and one-time treatment models, resulting in extremely high prices. For instance, in December 2022, CSL received U.S. FDA approval for the first gene therapy for hemophilia B, priced at USD 3.5 million per treatment. Such pricing creates financial challenges for patients, healthcare providers, and payers, slowing widespread adoption.

Market Opportunities

Advancements in gene delivery systems present significant growth opportunities. Innovations aimed at improving vector efficiency, tissue specificity, and scalability are transforming therapy development. In May 2025, Dyno Therapeutics launched new adeno-associated virus (AAV) capsid vectors targeting the eye, musculoskeletal system, and central nervous system. These developments are expected to improve therapeutic outcomes and support market expansion during the forecast period.

Market Challenges

A key challenge in the gene therapy market is maintaining long-term clinical efficacy. While early treatment responses are often strong, durability concerns remain due to immune responses and declining transgene expression. This necessitates extended clinical follow-up and increases development costs. Regulatory scrutiny regarding durability has also delayed approvals, impacting market growth.

Market Trends

A prominent trend in the gene therapy market is the focus on improving manufacturing scalability. With the growing number of clinical candidates, companies are investing in advanced bioprocessing technologies and automated manufacturing systems. In October 2024, the Cell and Gene Therapy Catapult received USD 1.5 million in funding to reduce AAV production costs and improve scalability, reflecting industry-wide efforts to enhance supply capabilities.

Segmentation Analysis

By Product

The market is segmented into Zolgensma, Luxturna, Roctavian, and others.

The Zolgensma segment dominated the market in 2024, with a value of USD 1.24 billion in 2025, driven by its proven durability and effectiveness in treating spinal muscular atrophy.

The Roctavian segment is projected to grow at a CAGR of 38.72% over the forecast period.

By Vector Type

Viral vectors dominated the market, accounting for 98.46% share in 2026, due to high transduction efficiency and long-lasting gene expression. The non-viral vector segment is expected to grow at a CAGR of 57.65%.

By Indication

The genetic disorders segment held the largest share in 2024 and is expected to account for 31.4% of the market in 2025, driven by increasing demand for personalized therapies. The ophthalmology segment is projected to grow at a CAGR of 19.35%.

By End User

The hospitals & clinics segment dominated the market with a 55.99% share in 2025, supported by their role in clinical trials and treatment administration.

Regional Outlook

North America led the market with a value of USD 1.05 billion in 2025 and USD 1.12 billion in 2026, driven by strong R&D activity and FDA approvals.

Europe reached USD 2.32 billion in 2025, supported by favorable regulatory frameworks.

Asia Pacific recorded USD 0.53 billion in 2026, with India and China valued at USD 0.04 billion and USD 0.19 billion, respectively.

Competitive Landscape

The market is semi-consolidated, led by Novartis AG, F. Hoffmann-La Roche Ltd., and BioMarin Pharmaceutical Inc. These companies benefit from strong product portfolios, global reach, and active R&D pipelines. Other key players include CSL, Vertex Pharmaceuticals, Krystal Biotech, and Rocket Pharmaceuticals.

Conclusion

The global gene therapy market is set for sustained growth from USD 4.29 billion in 2025 to USD 11.33 billion by 2034, driven by rising genetic disease prevalence, technological advancements, and increasing regulatory approvals. Despite challenges related to cost and long-term efficacy, continued innovation in delivery systems and manufacturing scalability is expected to support long-term market expansion.

Segmentation By Product

• Zolgensma
• Luxturna
• Roctavian
• Others

By Vector Product

• Viral Vectors
• Non-Viral Vectors

By Indication

• Genetic Disorders
• Ophthalmology
• Hematology
• Others

By End User

• Hospitals & Clinics
• Specialty Clinics
• Others

By Geography

• North America (By Product, Vector Type, Indication, End User, and Country)
  • U.S.
  • Canada
• Europe (By Product, Vector Type, Indication, End User, and Country/Sub-region)
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Scandinavia
  • Rest of Europe
• Asia Pacific (By Product, Vector Type, Indication, End User, and Country/Sub-region)
  • China
  • Japan
  • India
  • Australia
  • Southeast Asia
  • Rest of Asia Pacific
• Latin America (By Product, Vector Type, Indication, End User, and Country/Sub-region)
  • Brazil
  • Mexico
  • Rest of Latin America
• Middle East & Africa (By Product, Vector Type, Indication, End User, and Country/Sub-region)
  • GCC
  • South Africa
• Rest of the Middle East & Africa

Table of Content

1. Introduction

• 1.1. Research Scope
• 1.2. Market Segmentation
• 1.3. Research Methodology
• 1.4. Definitions and Assumptions

2. Executive Summary

3. Market Dynamics

• 3.1. Market Drivers
• 3.2. Market Restraints
• 3.3. Market Opportunities
• 3.4. Market Trends

4. Key Insights

• 4.1. Development of Gene Therapy for New Disease Indications
• 4.2. Regulatory Scenarios, By Key Countries/Regions
• 4.3. Technological Advancements in Gene Therapy Market
• 4.4. New Product Launches, By Key Players
• 4.5. Pipeline Analysis, By Key Companies
• 4.6. Key Industry Developments (Mergers, Acquisitions, Partnerships, Launches, and Others)
• 4.7. Pricing & Reimbursement Landscape in the Gene Therapy Market
• 4.8. Manufacturing & Supply Chain Insights
• 4.9. Investment & Funding Trends in the Market
• 4.10. Patient Access to the Therapy & Ethical Considerations

5. Global Gene Therapy Market Analysis, Insights and Forecast, 2021-2034

• 5.1. Market Analysis, Insights and Forecast - By Product
  • 5.1.1. Zolgensma
  • 5.1.2. Luxturna
  • 5.1.3. Roctavian
  • 5.1.4. Others
• 5.2. Market Analysis, Insights and Forecast - By Vector Type
  • 5.2.1. Viral Vectors
  • 5.2.2. Non-Viral Vectors
• 5.3. Market Analysis, Insights and Forecast - By Indication
  • 5.3.1. Genetic Disorders
  • 5.3.2. Ophthalmology
  • 5.3.3. Hematology
  • 5.3.4. Others
• 5.4. Market Analysis, Insights and Forecast - By End User
  • 5.4.1. Hospitals & Clinics
  • 5.4.2. Specialty Clinics
  • 5.4.3. Others
• 5.5. Market Analysis, Insights and Forecast - Region
  • 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

6. North America Gene Therapy Market Analysis, Insights and Forecast, 2021-2034

• 6.1. Market Analysis, Insights and Forecast - By Product
  • 6.1.1. Zolgensma
  • 6.1.2. Luxturna
  • 6.1.3. Roctavian
  • 6.1.4. Others
• 6.2. Market Analysis, Insights and Forecast - By Vector Type
  • 6.2.1. Viral Vectors
  • 6.2.2. Non-Viral Vectors
• 6.3. Market Analysis, Insights and Forecast - By Indication
  • 6.3.1. Genetic Disorders
  • 6.3.2. Ophthalmology
  • 6.3.3. Hematology
  • 6.3.4. Others
• 6.4. Market Analysis, Insights and Forecast - By End User
  • 6.4.1. Hospitals & Clinics
  • 6.4.2. Specialty Clinics
  • 6.4.3. Others
• 6.5. Market Analysis, Insights and Forecast - By Country
  • 6.5.1. U.S.
  • 6.5.2. Canada

7. Europe Gene Therapy Market Analysis, Insights and Forecast, 2021-2034

• 7.1. Market Analysis, Insights and Forecast - By Product
  • 7.1.1. Zolgensma
  • 7.1.2. Luxturna
  • 7.1.3. Roctavian
  • 7.1.4. Others
• 7.2. Market Analysis, Insights and Forecast - By Vector Type
  • 7.2.1. Viral Vectors
  • 7.2.2. Non-Viral Vectors
• 7.3. Market Analysis, Insights and Forecast - By Indication
  • 7.3.1. Genetic Disorders
  • 7.3.2. Ophthalmology
  • 7.3.3. Hematology
  • 7.3.4. Others
• 7.4. Market Analysis, Insights and Forecast - By End User
  • 7.4.1. Hospitals & Clinics
  • 7.4.2. Specialty Clinics
  • 7.4.3. Others
• 7.5. Market Analysis, Insights and Forecast - By Country/ Sub-Region
  • 7.5.1. Germany
  • 7.5.2. U.K.
  • 7.5.3. France
  • 7.5.4. Italy
  • 7.5.5. Spain
  • 7.5.6. Scandinavia
  • 7.5.7. Rest of Europe

8. Asia Pacific Gene Therapy Market Analysis, Insights and Forecast, 2021-2034

• 8.1. Market Analysis, Insights and Forecast - By Product
  • 8.1.1. Zolgensma
  • 8.1.2. Luxturna
  • 8.1.3. Roctavian
  • 8.1.4. Others
• 8.2. Market Analysis, Insights and Forecast - By Vector Type
  • 8.2.1. Viral Vectors
  • 8.2.2. Non-Viral Vectors
• 8.3. Market Analysis, Insights and Forecast - By Indication
  • 8.3.1. Genetic Disorders
  • 8.3.2. Ophthalmology
  • 8.3.3. Hematology
  • 8.3.4. Others
• 8.4. Market Analysis, Insights and Forecast - By End User
  • 8.4.1. Hospitals & Clinics
  • 8.4.2. Specialty Clinics
  • 8.4.3. Others
• 8.5. Market Analysis, Insights and Forecast - By Country/ Sub-Region
  • 8.5.1. China
  • 8.5.2. Japan
  • 8.5.3. India
  • 8.5.4. Australia
  • 8.5.5. Southeast Asia
  • 8.5.6. Rest of Asia Pacific

9. Latin America Gene Therapy Market Analysis, Insights and Forecast, 2021-2034

• 9.1. Market Analysis, Insights and Forecast - By Product
  • 9.1.1. Zolgensma
  • 9.1.2. Luxturna
  • 9.1.3. Roctavian
  • 9.1.4. Others
• 9.2. Market Analysis, Insights and Forecast - By Vector Type
  • 9.2.1. Viral Vectors
  • 9.2.2. Non-Viral Vectors
• 9.3. Market Analysis, Insights and Forecast - By Indication
  • 9.3.1. Genetic Disorders
  • 9.3.2. Ophthalmology
  • 9.3.3. Hematology
  • 9.3.4. Others
• 9.4. Market Analysis, Insights and Forecast - By End User
  • 9.4.1. Hospitals & Clinics
  • 9.4.2. Specialty Clinics
  • 9.4.3. Others
• 9.5. Market Analysis, Insights and Forecast - By Country/ Sub-Region
  • 9.5.1. Brazil
  • 9.5.2. Mexico
  • 9.5.3. Rest of Latin America

10. Middle East & Africa Gene Therapy Market Analysis, Insights and Forecast, 2021-2034

• 10.1. Market Analysis, Insights and Forecast - By Product
  • 10.1.1. Zolgensma
  • 10.1.2. Luxturna
  • 10.1.3. Roctavian
  • 10.1.4. Others
• 10.2. Market Analysis, Insights and Forecast - By Vector Type
  • 10.2.1. Viral Vectors
  • 10.2.2. Non-Viral Vectors
• 10.3. Market Analysis, Insights and Forecast - By Indication
  • 10.3.1. Genetic Disorders
  • 10.3.2. Ophthalmology
  • 10.3.3. Hematology
  • 10.3.4. Others
• 10.4. Market Analysis, Insights and Forecast - By End User
  • 10.4.1. Hospitals & Clinics
  • 10.4.2. Specialty Clinics
  • 10.4.3. Others
• 10.5. Market Analysis, Insights and Forecast - By Country/ Sub-Region
  • 10.5.1. GCC
  • 10.5.2. South Africa
  • 10.5.3. Rest of the Middle East & Africa

11. Competitive Analysis

• 11.1. Global Market Share Analysis (2025)
• 11.2. Company Profiles (Overview, Products, SWOT Analysis, Recent Developments, Strategies, Financials (Based on Availability))
  • 11.2.1. Novartis AG
  • 11.2.2. F. Hoffmann-La Roche Ltd
  • 11.2.3. BioMarin Pharmaceutical Inc.
  • 11.2.4. CSL
  • 11.2.5. Vertex Pharmaceuticals Incorporated
  • 11.2.6. Krystal Biotech, Inc.
  • 11.2.7. Rocket Pharmaceuticals
  • 11.2.8. Dyno Therapeutics, Inc.