Summary

Hemophilia Gene Therapy Market
The hemophilia gene therapy market was valued at $0.4 billion in 2023 and is projected to reach $3.0 billion by 2033, growing at a CAGR of 23.6% from 2024 to 2033.
Hemophilia is a genetic blood disorder that involves improper coagulation of blood due to deficiency or abnormality in certain clotting factors. Gene therapy for this disease is an innovative approach that includes the delivery of an active copy of the dysfunctional gene responsible for the production of clotting factors. The delivered gene induces the patient’s cells to produce the clotting factors, preventing the requirement for frequent factor replacement infusions. This treatment remains highly reliable as it cures the root cause of the disease, eliminating the risk of reversion.

Advancements in gene transfer technology have enhanced the reliability and efficacy of gene therapies, thereby propelling the development of the market. In addition, the ability of gene therapy to treat the root cause of hemophilia entirely prevents patients from recurrent factor replacement sessions and augments the market growth significantly. Furthermore, upsurge in government support in the form of funding & incentives for boosting the R&D of healthcare and biotechnology has elevated the availability of gene therapy for patients. This acts as a key driver of the hemophilia gene therapy market. A notable therapeutic approach currently being explored by researchers and doctors is gene editing. Genome editing tools such as zinc finger nucleases and transcription activator-like effector nucleases are being used to modify a patient’s DNA to cure the disease. This approach is poised to be trending in the future due to its highly targeted and fast-curing ability.

However, the high cost of gene therapies prevents several budget-sensitive individuals from undergoing the treatment, thereby hampering the development of the market. For instance, the two new gene therapies approved by the U.S. Food and Drug Administration in 2022-2023 have list prices of $2.9 million and 3.5 million. On the contrary, rise in strategic collaborations between pharmaceutical firms, research institutions, and regulatory bodies is anticipated to accelerate therapeutic development & reduce the associated expenses. This surge in collaborations & reduction in expenses is expected to present lucrative opportunities for the market growth.

Segment Review
The hemophilia gene therapy market is segmented into type, end user, and region. On the basis of type, the market is bifurcated into hemophilia A and hemophilia B. Depending on end user, it is divided into hospitals, specialty clinics, and others. Region wise, it is analyzed across North America, Europe, Asia-Pacific, and LAMEA.
Key Findings
On the basis of type, the hemophilia A segment is expected to dominate the market by 2033.
Region wise, North America was the highest revenue generator in 2023.
Competition Analysis
The major players in the global hemophilia gene therapy market include BioMarin Pharmaceutical Inc., Spark Therapeutics, Inc., uniQure N.V., Pfizer Inc., Freeline Therapeutics Holdings plc, Sangamo Therapeutics, Inc., Bioverativ, CSL Behring, Ultragenyx, and Shire Plc. These major players have adopted various key development strategies such as business expansion, new product launches, and partnerships to strengthen their foothold in the competitive market.

Additional benefits you will get with this purchase are:
● Quarterly Update and* (only available with a corporate license, on listed price)
● 5 additional Company Profile of client Choice pre- or Post-purchase, as a free update.
● Free Upcoming Version on the Purchase of Five and Enterprise User License.
● 16 analyst hours of support* (post-purchase, if you find additional data requirements upon review of the report, you may receive support amounting to 16 analyst hours to solve questions, and post-sale queries)
● 15% Free Customization* (in case the scope or segment of the report does not match your requirements, 15% is equivalent to 3 working days of free work, applicable once)
● Free data Pack on the Five and Enterprise User License. (Excel version of the report)
● Free Updated report if the report is 6-12 months old or older.
● 24-hour priority response*
● Free Industry updates and white papers.

Possible Customization with this report (with additional cost and timeline, please talk to the sales executive to know more)
● Patient/epidemiology data at country, region, global level
● Regulatory Guidelines
● Additional company profiles with specific to client's interest
● Expanded list for Company Profiles
● Historic market data
● SWOT Analysis
Key Market Segments
By Type
● Hemophilia A
● Hemophilia B
By End User
● Hospitals
● Specialty Clinics
● Others
By Region
● North America
○ U.S.
○ Canada
○ Mexico
● Europe
○ France
○ Germany
○ Italy
○ Spain
○ UK
○ Rest of Europe
● Asia-Pacific
○ China
○ Japan
○ India
○ South Korea
○ Australia
○ Rest of Asia-Pacific
● LAMEA
○ Brazil
○ South Africa
○ Saudi Arabia
○ Rest of LAMEA
● Key Market Players
○ BioMarin Pharmaceutical Inc.
○ Spark Therapeutics, Inc.
○ uniQure N.V.
○ Pfizer Inc.
○ Freeline Therapeutics Holdings plc.
○ Sangamo Therapeutics, Inc
○ Bioverativ
○ CSL Behring
○ Ultragenyx
○ Shire Plc

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Table of Contents

CHAPTER 1: INTRODUCTION
1.1. Report Description
1.2. Key Market Segments
1.3. Key Benefits
1.4. Research Methodology
1.4.1. Primary Research
1.4.2. Secondary Research
1.4.3. Analyst Tools and Models
CHAPTER 2: EXECUTIVE SUMMARY
2.1. CXO Perspective
CHAPTER 3: MARKET LANDSCAPE
3.1. Market Definition and Scope
3.2. Key Findings
3.2.1. Top Investment Pockets
3.2.2. Top Winning Strategies
3.3. Porter's Five Forces Analysis
3.3.1. Bargaining Power of Suppliers
3.3.2. Threat of New Entrants
3.3.3. Threat of Substitutes
3.3.4. Competitive Rivalry
3.3.5. Bargaining Power among Buyers
3.4. Market Dynamics
3.4.1. Drivers
3.4.2. Restraints
3.4.3. Opportunities
CHAPTER 4: HEMOPHILIA GENE THERAPY MARKET, BY TYPE
4.1. Market Overview
4.1.1 Market Size and Forecast, By Type
4.2. Hemophilia A
4.2.1. Key Market Trends, Growth Factors and Opportunities
4.2.2. Market Size and Forecast, By Region
4.2.3. Market Share Analysis, By Country
4.3. Hemophilia B
4.3.1. Key Market Trends, Growth Factors and Opportunities
4.3.2. Market Size and Forecast, By Region
4.3.3. Market Share Analysis, By Country
CHAPTER 5: HEMOPHILIA GENE THERAPY MARKET, BY END USER
5.1. Market Overview
5.1.1 Market Size and Forecast, By End User
5.2. Hospitals
5.2.1. Key Market Trends, Growth Factors and Opportunities
5.2.2. Market Size and Forecast, By Region
5.2.3. Market Share Analysis, By Country
5.3. Specialty Clinics
5.3.1. Key Market Trends, Growth Factors and Opportunities
5.3.2. Market Size and Forecast, By Region
5.3.3. Market Share Analysis, By Country
5.4. Others
5.4.1. Key Market Trends, Growth Factors and Opportunities
5.4.2. Market Size and Forecast, By Region
5.4.3. Market Share Analysis, By Country
CHAPTER 6: HEMOPHILIA GENE THERAPY MARKET, BY REGION
6.1. Market Overview
6.1.1 Market Size and Forecast, By Region
6.2. North America
6.2.1. Key Market Trends and Opportunities
6.2.2. Market Size and Forecast, By Type
6.2.3. Market Size and Forecast, By End User
6.2.4. Market Size and Forecast, By Country
6.2.5. U.S. Hemophilia Gene Therapy Market
6.2.5.1. Market Size and Forecast, By Type
6.2.5.2. Market Size and Forecast, By End User
6.2.6. Canada Hemophilia Gene Therapy Market
6.2.6.1. Market Size and Forecast, By Type
6.2.6.2. Market Size and Forecast, By End User
6.2.7. Mexico Hemophilia Gene Therapy Market
6.2.7.1. Market Size and Forecast, By Type
6.2.7.2. Market Size and Forecast, By End User
6.3. Europe
6.3.1. Key Market Trends and Opportunities
6.3.2. Market Size and Forecast, By Type
6.3.3. Market Size and Forecast, By End User
6.3.4. Market Size and Forecast, By Country
6.3.5. France Hemophilia Gene Therapy Market
6.3.5.1. Market Size and Forecast, By Type
6.3.5.2. Market Size and Forecast, By End User
6.3.6. Germany Hemophilia Gene Therapy Market
6.3.6.1. Market Size and Forecast, By Type
6.3.6.2. Market Size and Forecast, By End User
6.3.7. Italy Hemophilia Gene Therapy Market
6.3.7.1. Market Size and Forecast, By Type
6.3.7.2. Market Size and Forecast, By End User
6.3.8. Spain Hemophilia Gene Therapy Market
6.3.8.1. Market Size and Forecast, By Type
6.3.8.2. Market Size and Forecast, By End User
6.3.9. UK Hemophilia Gene Therapy Market
6.3.9.1. Market Size and Forecast, By Type
6.3.9.2. Market Size and Forecast, By End User
6.3.10. Rest Of Europe Hemophilia Gene Therapy Market
6.3.10.1. Market Size and Forecast, By Type
6.3.10.2. Market Size and Forecast, By End User
6.4. Asia-Pacific
6.4.1. Key Market Trends and Opportunities
6.4.2. Market Size and Forecast, By Type
6.4.3. Market Size and Forecast, By End User
6.4.4. Market Size and Forecast, By Country
6.4.5. China Hemophilia Gene Therapy Market
6.4.5.1. Market Size and Forecast, By Type
6.4.5.2. Market Size and Forecast, By End User
6.4.6. Japan Hemophilia Gene Therapy Market
6.4.6.1. Market Size and Forecast, By Type
6.4.6.2. Market Size and Forecast, By End User
6.4.7. India Hemophilia Gene Therapy Market
6.4.7.1. Market Size and Forecast, By Type
6.4.7.2. Market Size and Forecast, By End User
6.4.8. South Korea Hemophilia Gene Therapy Market
6.4.8.1. Market Size and Forecast, By Type
6.4.8.2. Market Size and Forecast, By End User
6.4.9. Australia Hemophilia Gene Therapy Market
6.4.9.1. Market Size and Forecast, By Type
6.4.9.2. Market Size and Forecast, By End User
6.4.10. Rest of Asia-Pacific Hemophilia Gene Therapy Market
6.4.10.1. Market Size and Forecast, By Type
6.4.10.2. Market Size and Forecast, By End User
6.5. LAMEA
6.5.1. Key Market Trends and Opportunities
6.5.2. Market Size and Forecast, By Type
6.5.3. Market Size and Forecast, By End User
6.5.4. Market Size and Forecast, By Country
6.5.5. Brazil Hemophilia Gene Therapy Market
6.5.5.1. Market Size and Forecast, By Type
6.5.5.2. Market Size and Forecast, By End User
6.5.6. South Africa Hemophilia Gene Therapy Market
6.5.6.1. Market Size and Forecast, By Type
6.5.6.2. Market Size and Forecast, By End User
6.5.7. Saudi Arabia Hemophilia Gene Therapy Market
6.5.7.1. Market Size and Forecast, By Type
6.5.7.2. Market Size and Forecast, By End User
6.5.8. Rest of LAMEA Hemophilia Gene Therapy Market
6.5.8.1. Market Size and Forecast, By Type
6.5.8.2. Market Size and Forecast, By End User
CHAPTER 7: COMPETITIVE LANDSCAPE
7.1. Introduction
7.2. Top Winning Strategies
7.3. Product Mapping Of Top 10 Player
7.4. Competitive Dashboard
7.5. Competitive Heatmap
7.6. Top Player Positioning, 2023
CHAPTER 8: COMPANY PROFILES
8.1. BioMarin Pharmaceutical Inc.
8.1.1. Company Overview
8.1.2. Key Executives
8.1.3. Company Snapshot
8.1.4. Operating Business Segments
8.1.5. Product Portfolio
8.1.6. Business Performance
8.1.7. Key Strategic Moves and Developments
8.2. Spark Therapeutics, Inc.
8.2.1. Company Overview
8.2.2. Key Executives
8.2.3. Company Snapshot
8.2.4. Operating Business Segments
8.2.5. Product Portfolio
8.2.6. Business Performance
8.2.7. Key Strategic Moves and Developments
8.3. UniQure N.V.
8.3.1. Company Overview
8.3.2. Key Executives
8.3.3. Company Snapshot
8.3.4. Operating Business Segments
8.3.5. Product Portfolio
8.3.6. Business Performance
8.3.7. Key Strategic Moves and Developments
8.4. Pfizer Inc.
8.4.1. Company Overview
8.4.2. Key Executives
8.4.3. Company Snapshot
8.4.4. Operating Business Segments
8.4.5. Product Portfolio
8.4.6. Business Performance
8.4.7. Key Strategic Moves and Developments
8.5. Freeline Therapeutics Holdings Plc.
8.5.1. Company Overview
8.5.2. Key Executives
8.5.3. Company Snapshot
8.5.4. Operating Business Segments
8.5.5. Product Portfolio
8.5.6. Business Performance
8.5.7. Key Strategic Moves and Developments
8.6. Sangamo Therapeutics, Inc
8.6.1. Company Overview
8.6.2. Key Executives
8.6.3. Company Snapshot
8.6.4. Operating Business Segments
8.6.5. Product Portfolio
8.6.6. Business Performance
8.6.7. Key Strategic Moves and Developments
8.7. Bioverativ
8.7.1. Company Overview
8.7.2. Key Executives
8.7.3. Company Snapshot
8.7.4. Operating Business Segments
8.7.5. Product Portfolio
8.7.6. Business Performance
8.7.7. Key Strategic Moves and Developments
8.8. CSL Behring
8.8.1. Company Overview
8.8.2. Key Executives
8.8.3. Company Snapshot
8.8.4. Operating Business Segments
8.8.5. Product Portfolio
8.8.6. Business Performance
8.8.7. Key Strategic Moves and Developments
8.9. Ultragenyx
8.9.1. Company Overview
8.9.2. Key Executives
8.9.3. Company Snapshot
8.9.4. Operating Business Segments
8.9.5. Product Portfolio
8.9.6. Business Performance
8.9.7. Key Strategic Moves and Developments
8.10. Shire Plc
8.10.1. Company Overview
8.10.2. Key Executives
8.10.3. Company Snapshot
8.10.4. Operating Business Segments
8.10.5. Product Portfolio
8.10.6. Business Performance
8.10.7. Key Strategic Moves and Developments