The global gene therapy market reached US$ 4.79 billion in 2024 and is expected to reach US$ 35.91 by 2033, growing at a CAGR of 23.1% during the forecast period 2025-2033.
Gene therapy is a modern medical technique that seeks to cure and prevent genetic disorders by modifying or introducing genetic material into the cells of a patient. This is achieved by using vectors, such as viruses, to deliver functional genes or gene-editing tools to the patient's body. By focusing on the specific genes that cause diseases, scientists aim to rectify or replace faulty genetic information, which could offer enduring solutions.
Gene therapy has immense potential for addressing inherited disorders, cardiovascular diseases, infectious diseases, and cancer. Gene therapy works by replacing or inactivating disease-causing genes. In some cases, gene therapy introduces new genes into the body to treat a specific disease. With gene therapy, healthcare providers deliver a healthy copy of a gene to cells inside the body. This healthy gene replaces a damaged gene, inactivates a mutated gene or introduces an entirely new gene.
Market Dynamics: Drivers & Restraints
Growing Utilization of Advanced Delivery Vectors
The development and utilization of advanced delivery vectors are one of the most significant factors driving the growth of the gene therapy market. Delivery vectors are essential for delivering therapeutic genes to specific cells in the body. The efficiency, precision, and safety of these vectors can significantly influence the effectiveness of gene therapies and their widespread adoption.
The development of more efficient vectors is enabling gene therapies to be applied to a wider range of diseases, including solid tumors, neurological disorders, and cardiovascular diseases, which were previously difficult to treat due to delivery challenges.
For instance, in October 2024, Roche applied Dyno Therapeutics’ engineered adeno-associated virus (AAV) capsid platform to develop next-generation AAV vectors for gene therapies targeting unspecified neurological diseases, through a collaboration that could generate more than $1 billion for Dyno.
Adeno-associated virus (AAV) vectors are among the most commonly used delivery systems due to their ability to target a variety of tissues, including the liver, muscle, and eye. AAV-based gene therapies, such as Zolgensma for Spinal Muscular Atrophy (SMA), are already proving highly effective.
High Cost associated with the Gene Therapies
The high cost of gene therapies is one of the most significant challenges impacting the growth and accessibility of the gene therapy market. While gene therapies have shown curative potential for many serious diseases, their expensive price tags pose barriers to widespread adoption.
For instance, the gene therapies Zynteglo and Skysona are priced at US$ 2.8 million and US$ 3 million per dose. Moreover, Zolgensma has a reported list price of US$ 2.1 million. The development of gene therapies is an expensive, lengthy process. Clinical trials for gene therapies often span several years and involve high patient monitoring and expensive regulatory approval procedures.
For instance, Luxturna, a gene therapy for Leber’s congenital amaurosis, took over 10 years from development to approval, and its cost is $850,000 per patient. Many gene therapies are personalized treatments that are tailored to the genetic makeup of individual patients, requiring bespoke production for each case. This individualization further increases costs.
For instance, CAR-T therapies such as Yescarta and Kymriah are customized for each patient by collecting and modifying the patient’s T-cells, a process that involves complex manufacturing and quality control measures. These therapies can cost over $300,000 to $400,000 per patient.
Segment Analysis
The global gene therapy market is segmented based on the approach, vector type, technique, application, and region.
Gene addition in the technique segment is expected to dominate the gene therapy market.
Gene Addition therapy involves introducing a functional gene into a patient's cells to compensate for deficiencies caused by loss-of-function mutations. This approach is particularly effective in addressing recessive monogenic diseases, where a faulty gene fails to produce a necessary protein.
According to John Wiley & Sons, Inc.'s research study in August 2024, stated that while various vectors can be used to deliver the functional gene, adeno-associated virus (AAV) vectors are often favored for gene therapy due to their small size, lack of human pathogenicity, weakened post-injection immune responses, and ease of engineering. However, Gene Addition cannot address all gene mutations, including dominant mutations, polygenic mutation conditions, and large genes that are incompatible with delivery vectors.
Furthermore, key players in the industry are product approvals that would drive this segment's growth. For instance, in November 2024, PTC Therapeutics, Inc. received accelerated approval from the U.S. Food and Drug Administration (FDA) for its gene therapy to treat Aromatic L-Amino Acid Decarboxylase (AADC) deficiency.
This marks the first gene therapy approved in the U.S. that is directly administered to the brain. The gene therapy will be marketed in the U.S. under the brand name Kebilidi and is approved for both children and adults with AADC deficiency, covering the full spectrum of disease severity. KEBILIDI is a Gene Addition therapy administered directly into the putamen region of the brain using a precise neurosurgical procedure called stereotactic surgery. It works by replacing a faulty gene.
Also, in August 2022, Bluebird Bio, Inc. received FDA approval for ZYNTEGLO (betibeglogene autotemcel) is a one-time, custom-made gene therapy that uses Gene Silencing to address the underlying genetic cause of beta-thalassemia in patients who require regular red blood cell (RBC) transfusions. Zynteglo addresses the underlying genetic cause of the disease.Top of FormBottom of Form
Geographical Analysis
North America is expected to dominate the Gene Therapy market
The gene therapy market in the North America region is expected to be driven by various factors like the increasing prevalence of cancer, genetic disorders, and other rare diseases. As per clinical Case Reports research publication in February 2024, SMA, a genetic disorder resulting from mutations in both copies of the SMN1 gene in cells, affects 1 in 6,000 to 1 in 10,000 children.
According to the National Heart, Lung, and Blood Institute data in September 2024, Sickle cell disease (SCD) affects approximately 100,000 people in the United States, with over 90% being non-Hispanic Black or African American, and 8 million people worldwide. The demand for gene-based treatments is expected to accelerate, driving the overall growth of the gene therapy market.
Furthermore, in this region, a major number of key players’ presence, a well-advanced healthcare infrastructure, technological advances, product launches, approvals, and increasing research activities are driving the gene therapy market in the North American region.
For instance, in September 2024, Vertex Pharmaceuticals Incorporated announced that Health Canada granted marketing authorization for CASGEVY (exagamglogene autotemcel) for individuals 12 years and older with sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) or transfusion-dependent beta-thalassemia (TDT). CASGEVY is an autologous genome-edited hematopoietic stem cell-based therapy. It is estimated that approximately 2,000 patients in Canada are eligible for this treatment, with the majority having SCD.
Similarly, in March 2024, Orchard Therapeutics, acquired by Kyowa Kirin, announced its U.S. launch plans for Lenmeldy (atidarsagene autotemcel), the first FDA-approved gene therapy for children with early-onset metachromatic leukodystrophy (MLD). MLD is a rare, rapidly progressive, and ultimately fatal neurometabolic disease affecting roughly one in 100,000 live births.
Competitive Landscape
The global market players in the gene therapy market are Alnylam Pharmaceuticals, Inc., Spark Therapeutics, Inc., Novartis AG, bluebird bio, Inc., Ferring Pharmaceuticals Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics group, Krystal Biotech, Inc. among others.
Why Purchase the Report?
• Pipeline & Innovations: Reviews ongoing clinical trials, product pipelines, and forecasts upcoming pharmaceutical advancements.
• Type 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 Type 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 gene therapy market report would provide approximately 45 tables, 46 figures, and 180 pages.
Target Audience 2024
• Manufacturers: Pharmaceutical, Biotech Companies, Contract Manufacturers, Distributors, Hospitals.
• Regulatory & Policy: Compliance Officers, Government, Health Economists, Market Access Specialists.
• Technology & Innovation: 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.
- 1. Market Introduction and Scope
- 1.1. Objectives of the Report
- 1.2. Report Coverage & Definitions
- 1.3. Report Scope
- 2. Executive Insights and Key Takeaways
- 3. Market Highlights and Strategic Takeaways
- 3.1. Key Trends and Future Projections
- 4. Snippet by Approach
- 4.1. Snippet by Vector Type
- 4.2. Snippet by Technique
- 4.3. Snippet by Application
- 4.4. Snippet by Region
- 5. Dynamics
- 5.1. Impacting Factors
- 5.1.1. Drivers
- 5.1.1.1. Growing Utilization of Advanced Delivery Vectors
- 5.1.1.2. Expansion into the Rare Diseases
- 5.1.1.3. XX
- 5.1.2. Restraints
- 5.1.2.1. High Cost Associated with the Gene Therapies
- 5.1.2.2. Safety and Long-Term Efficacy Concerns
- 5.1.2.3. XX
- 5.1.3. Opportunity
- 5.1.3.1. Personalized Cancer Gene Therapies
- 5.1.3.2. XX
- 5.1.4. Impact Analysis
- 6. Strategic Insights and Industry Outlook
- 6.1. Market Leaders and Pioneers
- 6.1.1. Emerging Pioneers and Prominent Players
- 6.1.2. Established leaders with the largest selling Brand
- 6.1.3. Market leaders with established Product
- 6.2. CXO Perspectives
- 6.3. Latest Developments and Breakthroughs
- 6.4. Case Studies/Ongoing Research
- 6.5. Regulatory and Reimbursement Landscape
- 6.5.1. North America
- 6.5.2. Europe
- 6.5.3. Asia Pacific
- 6.5.4. Latin America
- 6.5.5. Middle East & Africa
- 6.6. Porter’s Five Force Analysis
- 6.7. Supply Chain Analysis
- 6.8. Patent Analysis
- 6.9. SWOT Analysis
- 6.10. Unmet Needs and Gaps
- 6.11. Recommended Strategies for Market Entry and Expansion
- 6.12. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
- 6.13. Pricing Analysis and Price Dynamics
- 6.14. Key Opinion Leaders
- 7. Gene Therapy Market, By Approach
- 7.1. Introduction
- 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
- 7.1.2. Market Attractiveness Index, By Approach
- 7.2. Ex-Vivo*
- 7.2.1. Introduction
- 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 7.3. In-Vivo
- 8. Gene Therapy Market, By Vector Type
- 8.1. Introduction
- 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
- 8.1.2. Market Attractiveness Index, By Vector Type
- 8.2. Viral Vectors*
- 8.2.1. Introduction
- 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 8.2.3. Adeno-Associated Virus
- 8.2.4. Herpes Simplex Virus
- 8.2.5. Lentivirus
- 8.3. Non-Viral Vectors
- 9. Gene Therapy Market, By Technique
- 9.1. Introduction
- 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
- 9.1.2. Market Attractiveness Index, By Technique
- 9.2. Gene Addition*
- 9.2.1. Introduction
- 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 9.3. Gene Silencing
- 9.4. Gene Editing
- 10. Gene Therapy Market, By Application
- 10.1. Introduction
- 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
- 10.1.2. Market Attractiveness Index, By Application
- 10.2. Oncology*
- 10.2.1. Introduction
- 10.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
- 10.3. Musculoskeletal Conditions
- 10.4. Blood Disorders
- 10.5. Rare Diseases
- 10.6. Ophthalmology
- 10.7. Others
- 11. Gene Therapy Market, By Regional Market Analysis and Growth Opportunities
- 11.1. Introduction
- 11.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
- 11.1.1.1. Market Attractiveness Index, By Region
- 11.2. North America
- 11.2.1. Introduction
- 11.2.2. Key Region-Specific Dynamics
- 11.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
- 11.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
- 11.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
- 11.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
- 11.2.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.2.7.1. U.S.
- 11.2.7.2. Canada
- 11.3. Europe
- 11.3.1. Introduction
- 11.3.2. Key Region-Specific Dynamics
- 11.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
- 11.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
- 11.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
- 11.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
- 11.3.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.3.7.1. Germany
- 11.3.7.2. U.K.
- 11.3.7.3. France
- 11.3.7.4. Spain
- 11.3.7.5. Italy
- 11.3.7.6. Rest of Europe
- 11.4. Latin America
- 11.4.1. Introduction
- 11.4.2. Key Region-Specific Dynamics
- 11.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
- 11.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
- 11.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
- 11.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
- 11.4.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.4.7.1. Mexico
- 11.4.7.2. Brazil
- 11.4.7.3. Argentina
- 11.4.7.4. Rest of Latin America
- 11.5. Asia-Pacific
- 11.5.1. Introduction
- 11.5.2. Key Region-Specific Dynamics
- 11.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
- 11.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
- 11.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
- 11.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
- 11.5.7. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
- 11.5.7.1. China
- 11.5.7.2. India
- 11.5.7.3. Japan
- 11.5.7.4. South Korea
- 11.5.7.5. Rest of Asia-Pacific
- 11.6. Middle East and Africa
- 11.6.1. Introduction
- 11.6.2. Key Region-Specific Dynamics
- 11.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Approach
- 11.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
- 11.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
- 11.6.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
- 12. Competitive Landscape and Market Positioning
- 12.1. Competitive Overview and Key Market Players
- 12.2. Market Share Analysis and Positioning Matrix
- 12.3. Strategic Partnerships, Mergers & Acquisitions
- 12.4. Key Developments in Product Portfolios and Innovations
- 12.5. Company Benchmarking
- 13. Company Profiles
- 13.1. Alnylam Pharmaceuticals, Inc.*
- 13.1.1. Company Overview
- 13.1.2. Product Portfolio
- 13.1.2.1. Product Description
- 13.1.2.2. Product Key Performance Indicators (KPIs)
- 13.1.2.3. Historic and Forecasted Product Sales
- 13.1.2.4. Product Sales Volume
- 13.1.3. Financial Overview
- 13.1.4. Company Revenue
- 13.1.4.1. Geographical Revenue Shares
- 13.1.4.1.1. Revenue Forecasts
- 13.1.4.2. Key Developments
- 13.1.4.2.1. Mergers & Acquisitions
- 13.1.4.2.2. Key Product Development Activities
- 13.1.4.2.3. Regulatory Approvals, etc.
- 13.1.4.3. SWOT Analysis
- 13.2. Spark Therapeutics, Inc.
- 13.3. Novartis AG
- 13.4. bluebird bio, Inc.
- 13.5. Ferring Pharmaceuticals Inc.
- 13.6. Vertex Pharmaceuticals Incorporated
- 13.7. Sarepta Therapeutics, Inc.
- 13.8. CSL Behring LLC
- 13.9. Amgen, Inc.
- 13.10. Orchard Therapeutics group
- 13.11. Krystal Biotech, Inc. (LIST NOT EXHAUSTIVE)
- 14. Assumptions and Research Methodology
- 14.1. Data Collection Methods
- 14.2. Data Triangulation
- 14.3. Forecasting Techniques
- 14.4. Data Verification and Validation
- 15. Appendix
- 15.1. About Us and Services
- 15.2. Contact Us
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