US Gene Therapy Market - 2025-2033
Overview
The US gene therapy market reached US$ 2,812.92 million in 2024 and is expected to reach US$ 21,701.51 million by 2033, growing at a CAGR of 23.5 % 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 US 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, in San Francisco, 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 $1 billion for Dyno.
Delivery vectors are critical technologies used to transport genetic material into a patient's cells. Adeno-associated virus (AAV) vectors are among the most commonly used delivery systems due to their ability to target a variety of tissues, including the liver, muscle, and eye. AAV-based gene therapies, such as Zolgensma for spinal muscular atrophy (SMA), are already proving highly effective.
High Cost associated with the Gene Therapies
The high cost of gene therapies is one of the most significant challenges impacting the growth and accessibility of gene therapies in the U.S.. While gene therapies have shown curative potential for many serious diseases, their expensive price tags pose barriers to widespread adoption.
For instance, the gene therapies Zynteglo and Skysona are priced at US$ 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 also an expensive and 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 US gene therapy market is segmented based on approach, vector type, technique, and application.
Approach:
The in vivo segment in the approach is expected to dominate the US gene therapy market share
In-vivo gene therapy refers to the direct delivery of genetic material either intravenously (through an IV) or locally to a specific organ (eg, directly into the eye). In-vivo gene therapy works through the help of a vector, which directly inserts functional copies of a gene into target cells to treat a mutated or missing gene.
In vivo delivery of gene therapy has been proven in many areas of research. Some of the currently approved gene therapies deliver genetic material in vivo. Targeted in vivo gene therapy will continue to evolve as scientists continue to refine methods of gene delivery.
Furthermore, key players’ strategies, such as investments, agreements, and technological innovations in AI, would propel this market growth. For instance, in June 2024, in the US, 2seventy Bio completed a $40 million asset purchase agreement (APA) with Novo Nordisk, selling its Hemophilia A program. Novo Nordisk also gains rights to 2seventy's MegaTAL in vivo gene editing technology, excluding its use in oncology and certain autoimmune-related cell therapies.
Also, in January 2025, Dyno Therapeutics, a genetic technology company specializing in AI-driven in vivo gene delivery, announced that Roche has exercised its option to license a novel adeno-associated virus (AAV) capsid for a gene therapy program targeting an undisclosed neurological disease. Dyno's platform uses AI and in vivo data to address gene delivery challenges. Overall, as research and development continue and technological evolution in in-vivo delivery methods plays an important role in this segment’s growth during the forecast period.
Competitive Landscape
The major players in the US gene therapy market include Alnylam Pharmaceuticals, Inc., NOVARTIS AG, Sarepta Therapeutics, Inc., Krystal Biotech, Inc., CSL, Bluebird Bio, Inc., SPARK THERAPEUTICS, INC., Ferring, Vertex Pharmaceuticals Incorporated, Amgen, Inc., and Orchard Therapeutics Plc, among others.
Key Developments
Target Audience 2024
The US gene therapy market reached US$ 2,812.92 million in 2024 and is expected to reach US$ 21,701.51 million by 2033, growing at a CAGR of 23.5 % 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 US 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, in San Francisco, 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 $1 billion for Dyno.
Delivery vectors are critical technologies used to transport genetic material into a patient's cells. Adeno-associated virus (AAV) vectors are among the most commonly used delivery systems due to their ability to target a variety of tissues, including the liver, muscle, and eye. AAV-based gene therapies, such as Zolgensma for spinal muscular atrophy (SMA), are already proving highly effective.
High Cost associated with the Gene Therapies
The high cost of gene therapies is one of the most significant challenges impacting the growth and accessibility of gene therapies in the U.S.. While gene therapies have shown curative potential for many serious diseases, their expensive price tags pose barriers to widespread adoption.
For instance, the gene therapies Zynteglo and Skysona are priced at US$ 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 also an expensive and 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 US gene therapy market is segmented based on approach, vector type, technique, and application.
Approach:
The in vivo segment in the approach is expected to dominate the US gene therapy market share
In-vivo gene therapy refers to the direct delivery of genetic material either intravenously (through an IV) or locally to a specific organ (eg, directly into the eye). In-vivo gene therapy works through the help of a vector, which directly inserts functional copies of a gene into target cells to treat a mutated or missing gene.
In vivo delivery of gene therapy has been proven in many areas of research. Some of the currently approved gene therapies deliver genetic material in vivo. Targeted in vivo gene therapy will continue to evolve as scientists continue to refine methods of gene delivery.
Furthermore, key players’ strategies, such as investments, agreements, and technological innovations in AI, would propel this market growth. For instance, in June 2024, in the US, 2seventy Bio completed a $40 million asset purchase agreement (APA) with Novo Nordisk, selling its Hemophilia A program. Novo Nordisk also gains rights to 2seventy's MegaTAL in vivo gene editing technology, excluding its use in oncology and certain autoimmune-related cell therapies.
Also, in January 2025, Dyno Therapeutics, a genetic technology company specializing in AI-driven in vivo gene delivery, announced that Roche has exercised its option to license a novel adeno-associated virus (AAV) capsid for a gene therapy program targeting an undisclosed neurological disease. Dyno's platform uses AI and in vivo data to address gene delivery challenges. Overall, as research and development continue and technological evolution in in-vivo delivery methods plays an important role in this segment’s growth during the forecast period.
Competitive Landscape
The major players in the US gene therapy market include Alnylam Pharmaceuticals, Inc., NOVARTIS AG, Sarepta Therapeutics, Inc., Krystal Biotech, Inc., CSL, Bluebird Bio, Inc., SPARK THERAPEUTICS, INC., Ferring, Vertex Pharmaceuticals Incorporated, Amgen, Inc., and Orchard Therapeutics Plc, among others.
Key Developments
- In February 2025, a global biotechnology leader in the US, CSL, announced the four-year results from the pivotal HOPE-B study confirming the long-term durability and safety of a one-time infusion of HEMGENIX (etranacogene dezaparvovec-drlb) for adults living with hemophilia B.
- In September 2024, uniQure N.V., a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease.
- In April 2024, Ferring Pharmaceuticals announced a strategic agreement with SK pharmteco, a leading contract development and manufacturing organization (CDMO), to scale up commercial production of the drug substance for Adstiladrin (nadofaragene firadenovec-vncg), an FDA-approved intravesical gene therapy for bladder cancer.
- Pipeline & Innovations: Reviews ongoing clinical trials and product pipelines and forecasts upcoming advancements in medical devices and pharmaceuticals.
- Approach Performance & Market Positioning: Analyze product performance, market positioning, and growth potential to optimize strategies.
- Real-World Evidence: Integrates patient feedback and data into Approach 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: This covers recent regulatory changes, new policies, and emerging technologies.
- Competitive Strategies: Analyze 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 an efficient Approach to delivery.
- Sustainability & Regulatory Impact: Focuses on eco-friendly practices and evolving regulations in healthcare.
- Post-market Surveillance: Uses post-market data to enhance the product's 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.
- Vector 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.
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
2.1. Market Highlights and Strategic Takeaways
2.2. Key Trends and Future Projections
2.3. Snippet by Approach
2.4. Snippet by Vector Type
2.5. Snippet by Technique
2.6. Snippet by Application
3. DYNAMICS
3.1. Impacting Factors
3.1.1. Drivers
3.1.1.1. Growing Utilization of Advanced Delivery Vectors
3.1.1.2. Expansion of Gene Therapy Applications into the Rare Diseases
3.1.1.3. XX
3.1.2. Restraints
3.1.2.1. High Cost Associated with Gene Therapies
3.1.2.2. Safety and Long-Term Efficacy Concerns
3.1.2.3. XX
3.1.3. Opportunity
3.1.3.1. Personalized Cancer Gene Therapies
3.1.3.2. XX
3.1.4. Impact Analysis
4. STRATEGIC INSIGHTS AND INDUSTRY OUTLOOK
4.1. Market Leaders and Pioneers
4.1.1. Emerging Pioneers and Prominent Players
4.1.2. Established leaders with the largest-selling Brand
4.1.3. Market leaders with established Approach
4.2. CXO Perspectives
4.3. Latest Developments and Breakthroughs
4.4. Case Studies/Ongoing Research
4.5. Regulatory and Reimbursement Landscape
4.6. Porter’s Five Forces Analysis
4.7. Supply Chain Analysis
4.8. Patent Analysis
4.9. SWOT Analysis
4.10. Unmet Needs and Gaps
4.11. Recommended Strategies for Market Entry and Expansion
4.12. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
4.13. Pricing Analysis and Price Dynamics
4.14. Key Opinion Leaders
5. US GENE THERAPY MARKET, BY APPROACH
5.1. Introduction
5.1.1. Analysis and Y-o-Y Growth Analysis (%), By Approach
5.1.2. Market Attractiveness Index, By Approach
5.2. In-Vivo*
5.2.1. Introduction
5.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
5.3. Ex vivo
6. US GENE THERAPY MARKET, BY VECTOR TYPE
6.1. Introduction
6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
6.1.2. Market Attractiveness Index, By Vector Type
6.2. Viral Vectors*
6.2.1. Introduction
6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
6.2.3. Adeno-Associated Virus
6.2.4. Herpes Simplex Virus
6.2.5. Lentivirus
6.3. Non-Viral Vectors
7. US GENE THERAPY MARKET, BY TECHNIQUE
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
7.1.2. Market Attractiveness Index, By Technique
7.2. Gene Addition*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Gene Silencing
7.4. Gene Editing
8. US GENE THERAPY MARKET, BY APPLICATION
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
8.1.2. Market Attractiveness Index, By Application
8.2. Rare Diseases*
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Musculoskeletal Conditions
8.4. Blood Disorders
8.5. Oncology
8.6. Ophthalmology
8.7. Others
9. COMPETITIVE LANDSCAPE AND MARKET POSITIONING
9.1. Competitive Overview and Key Market Players
9.2. Market Share Analysis and Positioning Matrix
9.3. Strategic Partnerships, Mergers & Acquisitions
9.4. Key Developments in Approach Portfolios and Innovations
9.5. Company Benchmarking
10. COMPANY PROFILES
10.1. Alnylam Pharmaceuticals, Inc.
10.1.1. Company Overview
10.1.2. Approach Portfolio
10.1.2.1. Approach Description
10.1.2.2. Approach Key Performance Indicators (KPIs)
10.1.2.3. Historic and Forecasted Approach Sales
10.1.2.4. Approach Sales Volume
10.1.3. Financial Overview
10.1.3.1. Company Revenue
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 Approach Development Activities
10.1.4.3. Regulatory Approvals, etc.
10.1.5. SWOT Analysis
10.2. NOVARTIS AG
10.3. Sarepta Therapeutics, Inc.
10.4. Krystal Biotech, Inc.
10.5. CSL
10.6. Bluebird Bio, Inc.
10.7. SPARK THERAPEUTICS, INC.
10.8. Ferring
10.9. Vertex Pharmaceuticals Incorporated
10.10. Amgen, Inc
10.11. Orchard Therapeutics Plc
LIST NOT EXHAUSTIVE
11. ASSUMPTIONS AND RESEARCH METHODOLOGY
11.1. Data Collection Methods
11.2. Data Triangulation
11.3. Forecasting Techniques
11.4. Data Verification and Validation
12. APPENDIX
12.1. About Us and Services
12.2. Contact Us
1.1. Objectives of the Report
1.2. Report Coverage & Definitions
1.3. Report Scope
2. EXECUTIVE INSIGHTS AND KEY TAKEAWAYS
2.1. Market Highlights and Strategic Takeaways
2.2. Key Trends and Future Projections
2.3. Snippet by Approach
2.4. Snippet by Vector Type
2.5. Snippet by Technique
2.6. Snippet by Application
3. DYNAMICS
3.1. Impacting Factors
3.1.1. Drivers
3.1.1.1. Growing Utilization of Advanced Delivery Vectors
3.1.1.2. Expansion of Gene Therapy Applications into the Rare Diseases
3.1.1.3. XX
3.1.2. Restraints
3.1.2.1. High Cost Associated with Gene Therapies
3.1.2.2. Safety and Long-Term Efficacy Concerns
3.1.2.3. XX
3.1.3. Opportunity
3.1.3.1. Personalized Cancer Gene Therapies
3.1.3.2. XX
3.1.4. Impact Analysis
4. STRATEGIC INSIGHTS AND INDUSTRY OUTLOOK
4.1. Market Leaders and Pioneers
4.1.1. Emerging Pioneers and Prominent Players
4.1.2. Established leaders with the largest-selling Brand
4.1.3. Market leaders with established Approach
4.2. CXO Perspectives
4.3. Latest Developments and Breakthroughs
4.4. Case Studies/Ongoing Research
4.5. Regulatory and Reimbursement Landscape
4.6. Porter’s Five Forces Analysis
4.7. Supply Chain Analysis
4.8. Patent Analysis
4.9. SWOT Analysis
4.10. Unmet Needs and Gaps
4.11. Recommended Strategies for Market Entry and Expansion
4.12. Scenario Analysis: Best-Case, Base-Case, and Worst-Case Forecasts
4.13. Pricing Analysis and Price Dynamics
4.14. Key Opinion Leaders
5. US GENE THERAPY MARKET, BY APPROACH
5.1. Introduction
5.1.1. Analysis and Y-o-Y Growth Analysis (%), By Approach
5.1.2. Market Attractiveness Index, By Approach
5.2. In-Vivo*
5.2.1. Introduction
5.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
5.3. Ex vivo
6. US GENE THERAPY MARKET, BY VECTOR TYPE
6.1. Introduction
6.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Vector Type
6.1.2. Market Attractiveness Index, By Vector Type
6.2. Viral Vectors*
6.2.1. Introduction
6.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
6.2.3. Adeno-Associated Virus
6.2.4. Herpes Simplex Virus
6.2.5. Lentivirus
6.3. Non-Viral Vectors
7. US GENE THERAPY MARKET, BY TECHNIQUE
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Technique
7.1.2. Market Attractiveness Index, By Technique
7.2. Gene Addition*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.3. Gene Silencing
7.4. Gene Editing
8. US GENE THERAPY MARKET, BY APPLICATION
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Application
8.1.2. Market Attractiveness Index, By Application
8.2. Rare Diseases*
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Musculoskeletal Conditions
8.4. Blood Disorders
8.5. Oncology
8.6. Ophthalmology
8.7. Others
9. COMPETITIVE LANDSCAPE AND MARKET POSITIONING
9.1. Competitive Overview and Key Market Players
9.2. Market Share Analysis and Positioning Matrix
9.3. Strategic Partnerships, Mergers & Acquisitions
9.4. Key Developments in Approach Portfolios and Innovations
9.5. Company Benchmarking
10. COMPANY PROFILES
10.1. Alnylam Pharmaceuticals, Inc.
10.1.1. Company Overview
10.1.2. Approach Portfolio
10.1.2.1. Approach Description
10.1.2.2. Approach Key Performance Indicators (KPIs)
10.1.2.3. Historic and Forecasted Approach Sales
10.1.2.4. Approach Sales Volume
10.1.3. Financial Overview
10.1.3.1. Company Revenue
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 Approach Development Activities
10.1.4.3. Regulatory Approvals, etc.
10.1.5. SWOT Analysis
10.2. NOVARTIS AG
10.3. Sarepta Therapeutics, Inc.
10.4. Krystal Biotech, Inc.
10.5. CSL
10.6. Bluebird Bio, Inc.
10.7. SPARK THERAPEUTICS, INC.
10.8. Ferring
10.9. Vertex Pharmaceuticals Incorporated
10.10. Amgen, Inc
10.11. Orchard Therapeutics Plc
LIST NOT EXHAUSTIVE
11. ASSUMPTIONS AND RESEARCH METHODOLOGY
11.1. Data Collection Methods
11.2. Data Triangulation
11.3. Forecasting Techniques
11.4. Data Verification and Validation
12. APPENDIX
12.1. About Us and Services
12.2. Contact Us
LIST OF TABLES
Table 1 US Gene Therapy Market Value, By Approach, 2025, 2029 & 2033 (US$ Million)
Table 2 US Gene Therapy Market Value, By Vector Type, 2025, 2029 & 2033 (US$ Million)
Table 3 US Gene Therapy Market Value, By Technique, 2025, 2029 & 2033 (US$ Million)
Table 4 US Gene Therapy Market Value, By Application, 2025, 2029 & 2033 (US$ Million)
Table 5 US Gene Therapy Market Value, By Approach, 2025, 2029 & 2033 (US$ Million)
Table 6 US Gene Therapy Market Value, By Approach, 2022-2033 (US$ Million)
Table 7 US Gene Therapy Market Value, By Vector Type, 2025, 2029 & 2033 (US$ Million)
Table 8 US Gene Therapy Market Value, By Vector Type, 2022-2033 (US$ Million)
Table 9 US Gene Therapy Market Value, By Technique, 2025, 2029 & 2033 (US$ Million)
Table 10 US Gene Therapy Market Value, By Technique, 2022-2033 (US$ Million)
Table 11 US Gene Therapy Market Value, By Application, 2025, 2029 & 2033 (US$ Million)
Table 12 US Gene Therapy Market Value, By Application, 2022-2033 (US$ Million)
Table 13 Alnylam Pharmaceuticals, Inc.: Overview
Table 14 Alnylam Pharmaceuticals, Inc.: Product Portfolio
Table 15 Alnylam Pharmaceuticals, Inc.: Key Developments
Table 16 NOVARTIS AG: Overview
Table 17 NOVARTIS AG: Product Portfolio
Table 18 NOVARTIS AG: Key Developments
Table 19 Sarepta Therapeutics, Inc.: Overview
Table 20 Sarepta Therapeutics, Inc.: Product Portfolio
Table 21 Sarepta Therapeutics, Inc.: Key Developments
Table 22 Krystal Biotech, Inc.: Overview
Table 23 Krystal Biotech, Inc.: Product Portfolio
Table 24 Krystal Biotech, Inc.: Key Developments
Table 25 CSL: Overview
Table 26 CSL: Product Portfolio
Table 27 CSL: Key Developments
Table 28 Bluebird Bio, Inc.: Overview
Table 29 Bluebird Bio, Inc.: Product Portfolio
Table 30 Bluebird Bio, Inc.: Key Developments
Table 31 SPARK THERAPEUTICS, INC.: Overview
Table 32 SPARK THERAPEUTICS, INC.: Product Portfolio
Table 33 SPARK THERAPEUTICS, INC.: Key Developments
Table 34 Ferring: Overview
Table 35 Ferring: Product Portfolio
Table 36 Ferring: Key Developments
Table 37 Vertex Pharmaceuticals Incorporated: Overview
Table 38 Vertex Pharmaceuticals Incorporated: Product Portfolio
Table 39 Vertex Pharmaceuticals Incorporated: Key Developments
Table 40 Amgen, Inc.: Overview
Table 41 Amgen, Inc.: Product Portfolio
Table 42 Amgen, Inc.: Key Developments
Table 43 Orchard Therapeutics Plc: Overview
Table 44 Orchard Therapeutics Plc: Product Portfolio
Table 45 Orchard Therapeutics Plc: Key Developments
Table 1 US Gene Therapy Market Value, By Approach, 2025, 2029 & 2033 (US$ Million)
Table 2 US Gene Therapy Market Value, By Vector Type, 2025, 2029 & 2033 (US$ Million)
Table 3 US Gene Therapy Market Value, By Technique, 2025, 2029 & 2033 (US$ Million)
Table 4 US Gene Therapy Market Value, By Application, 2025, 2029 & 2033 (US$ Million)
Table 5 US Gene Therapy Market Value, By Approach, 2025, 2029 & 2033 (US$ Million)
Table 6 US Gene Therapy Market Value, By Approach, 2022-2033 (US$ Million)
Table 7 US Gene Therapy Market Value, By Vector Type, 2025, 2029 & 2033 (US$ Million)
Table 8 US Gene Therapy Market Value, By Vector Type, 2022-2033 (US$ Million)
Table 9 US Gene Therapy Market Value, By Technique, 2025, 2029 & 2033 (US$ Million)
Table 10 US Gene Therapy Market Value, By Technique, 2022-2033 (US$ Million)
Table 11 US Gene Therapy Market Value, By Application, 2025, 2029 & 2033 (US$ Million)
Table 12 US Gene Therapy Market Value, By Application, 2022-2033 (US$ Million)
Table 13 Alnylam Pharmaceuticals, Inc.: Overview
Table 14 Alnylam Pharmaceuticals, Inc.: Product Portfolio
Table 15 Alnylam Pharmaceuticals, Inc.: Key Developments
Table 16 NOVARTIS AG: Overview
Table 17 NOVARTIS AG: Product Portfolio
Table 18 NOVARTIS AG: Key Developments
Table 19 Sarepta Therapeutics, Inc.: Overview
Table 20 Sarepta Therapeutics, Inc.: Product Portfolio
Table 21 Sarepta Therapeutics, Inc.: Key Developments
Table 22 Krystal Biotech, Inc.: Overview
Table 23 Krystal Biotech, Inc.: Product Portfolio
Table 24 Krystal Biotech, Inc.: Key Developments
Table 25 CSL: Overview
Table 26 CSL: Product Portfolio
Table 27 CSL: Key Developments
Table 28 Bluebird Bio, Inc.: Overview
Table 29 Bluebird Bio, Inc.: Product Portfolio
Table 30 Bluebird Bio, Inc.: Key Developments
Table 31 SPARK THERAPEUTICS, INC.: Overview
Table 32 SPARK THERAPEUTICS, INC.: Product Portfolio
Table 33 SPARK THERAPEUTICS, INC.: Key Developments
Table 34 Ferring: Overview
Table 35 Ferring: Product Portfolio
Table 36 Ferring: Key Developments
Table 37 Vertex Pharmaceuticals Incorporated: Overview
Table 38 Vertex Pharmaceuticals Incorporated: Product Portfolio
Table 39 Vertex Pharmaceuticals Incorporated: Key Developments
Table 40 Amgen, Inc.: Overview
Table 41 Amgen, Inc.: Product Portfolio
Table 42 Amgen, Inc.: Key Developments
Table 43 Orchard Therapeutics Plc: Overview
Table 44 Orchard Therapeutics Plc: Product Portfolio
Table 45 Orchard Therapeutics Plc: Key Developments
LIST OF FIGURES
Figure 1 US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 2 US Gene Therapy Market Share, By Approach, 2024 & 2033 (%)
Figure 3 US Gene Therapy Market Share, By Vector Type, 2024 & 2033 (%)
Figure 4 US Gene Therapy Market Share, By Technique, 2024 & 2033 (%)
Figure 5 US Gene Therapy Market Share, By Application, 2024 & 2033 (%)
Figure 6 US Gene Therapy Market Y-o-Y Growth, By Approach, 2023-2033 (%)
Figure 7 In-Vivo Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 8 Ex vivo Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 9 US Gene Therapy Market Y-o-Y Growth, By Vector Type, 2023-2033 (%)
Figure 10 Viral Vectors Vector Type in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 11 Non-Viral Vectors Vector Type in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 12 US Gene Therapy Market Y-o-Y Growth, By Technique, 2023-2033 (%)
Figure 13 Gene Addition Technique in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 14 Gene Silencing Technique in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 15 Gene Editing Technique in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 16 US Gene Therapy Market Y-o-Y Growth, By Application, 2023-2033 (%)
Figure 17 Rare Diseases Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 18 Musculoskeletal Conditions Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 19 Blood Disorders Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 20 Oncology Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 21 Ophthalmology Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 22 Others Application in the US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 23 Alnylam Pharmaceuticals, Inc.: Financials
Figure 24 NOVARTIS AG: Financials
Figure 25 Sarepta Therapeutics, Inc.: Financials
Figure 26 Krystal Biotech, Inc.: Financials
Figure 27 CSL: Financials
Figure 28 Bluebird Bio, Inc.: Financials
Figure 29 SPARK THERAPEUTICS, INC.: Financials
Figure 30 Ferring: Financials
Figure 31 Vertex Pharmaceuticals Incorporated: Financials
Figure 32 Amgen, Inc: Financials
Figure 33 Orchard Therapeutics Plc: Financials
Figure 1 US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 2 US Gene Therapy Market Share, By Approach, 2024 & 2033 (%)
Figure 3 US Gene Therapy Market Share, By Vector Type, 2024 & 2033 (%)
Figure 4 US Gene Therapy Market Share, By Technique, 2024 & 2033 (%)
Figure 5 US Gene Therapy Market Share, By Application, 2024 & 2033 (%)
Figure 6 US Gene Therapy Market Y-o-Y Growth, By Approach, 2023-2033 (%)
Figure 7 In-Vivo Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 8 Ex vivo Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 9 US Gene Therapy Market Y-o-Y Growth, By Vector Type, 2023-2033 (%)
Figure 10 Viral Vectors Vector Type in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 11 Non-Viral Vectors Vector Type in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 12 US Gene Therapy Market Y-o-Y Growth, By Technique, 2023-2033 (%)
Figure 13 Gene Addition Technique in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 14 Gene Silencing Technique in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 15 Gene Editing Technique in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 16 US Gene Therapy Market Y-o-Y Growth, By Application, 2023-2033 (%)
Figure 17 Rare Diseases Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 18 Musculoskeletal Conditions Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 19 Blood Disorders Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 20 Oncology Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 21 Ophthalmology Application in US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 22 Others Application in the US Gene Therapy Market Value, 2022-2033 (US$ Million)
Figure 23 Alnylam Pharmaceuticals, Inc.: Financials
Figure 24 NOVARTIS AG: Financials
Figure 25 Sarepta Therapeutics, Inc.: Financials
Figure 26 Krystal Biotech, Inc.: Financials
Figure 27 CSL: Financials
Figure 28 Bluebird Bio, Inc.: Financials
Figure 29 SPARK THERAPEUTICS, INC.: Financials
Figure 30 Ferring: Financials
Figure 31 Vertex Pharmaceuticals Incorporated: Financials
Figure 32 Amgen, Inc: Financials
Figure 33 Orchard Therapeutics Plc: Financials
