Global Hemophilia Gene Therapy Market 2019 by Company, Regions, Type and Application, Forecast to 2024


Report ID : RNR-533466 Category : Biotechnology Published Date : May 2019
Publisher : GlobalInfo Research Pages : 125 Format : PDF
Global Hemophilia Gene Therapy Market 2019 by Company, Regions, Type and Application, Forecast to 2024

Hemophilia is a rare bleeding disorder in which the blood does not clot normally. Hemophilia is a monogenic disease (a disease that is caused by a genetic defect in a single gene). There are two types of hemophilia caused by mutations in genes that encode protein factors which help the blood clot and stop bleeding when blood vessels are injured. Individuals with hemophilia experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis. The most frequent forms of hemophilia affect males.

Scope of the Report:
About 80% of them have hemophilia A, which affects the clotting factor VIII. The second most common form, hemophilia B, is due to a deficiency of the clotting factor IX. Several biotechs are racing to launch the first gene therapy for hemophilia. Currently, uniQure in the Netherlands and Spark Therapeutics in the US have the most advanced programs. Spark scored a victory in December when it presented Phase I/II for its candidate SPK-9001. The gene therapy was able to reduce annual bleeding episodes by 97%, as compared to its competitor uniQure’s candidate, AMT-060. However, uniQure has fought back. The company added a modification in its gene therapy that is known to increase clotting activity by 8- to 9- fold. This improved version has already been cleared to start a Phase III clinical trial in both Europe and the US.
Hemophilia B has traditionally been the main focus of biotechs, since most big pharma efforts were focused on the bigger hemophilia A market. In addition, applying gene therapy to hemophilia A is more challenging; The gene coding for the factor IX protein missing in hemophilia B is simply smaller than that for factor VIII missing in hemophilia A, and therefore easier to fit in the viral vectors used for gene delivery. US-based BioMarin is leading the development of a gene therapy. To overcome the size limit, the company has deleted a region from the factor VIII protein that is not necessary for clotting.
Hemophilia treatment is currently in the pre-clinical stage. And the multiple treatments that are underway might significantly improve the quality of life of patients with hemophilia, by getting rid of frequent infusions and hospital visits, and transitioning patients from severe to mild hemophilia.
The global Hemophilia Gene Therapy market is valued at xx million USD in 2018 and is expected to reach xx million USD by the end of 2024, growing at a CAGR of xx% between 2019 and 2024.
The Asia-Pacific will occupy for more market share in following years, especially in China, also fast growing India and Southeast Asia regions.
North America, especially The United States, will still play an important role which cannot be ignored. Any changes from United States might affect the development trend of Hemophilia Gene Therapy.
Europe also play important roles in global market, with market size of xx million USD in 2019 and will be xx million USD in 2024, with a CAGR of xx%.
This report studies the Hemophilia Gene Therapy market status and outlook of Global and major regions, from angles of players, countries, product types and end industries; this report analyzes the top players in global market, and splits the Hemophilia Gene Therapy market by product type and applications/end industries.

Market Segment by Companies, this report covers
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

Market Segment by Regions, regional analysis covers 
North America (United States, Canada and Mexico)
Europe (Germany, France, UK, Russia and Italy)
Asia-Pacific (China, Japan, Korea, India and Southeast Asia)
South America (Brazil, Argentina, Colombia)
Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa)

Market Segment by Type, covers
Hemophilia A
Hemophilia B

Market Segment by Applications, can be divided into
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy
1 Hemophilia Gene Therapy Market Overview
1.1 Product Overview and Scope of Hemophilia Gene Therapy
1.2 Classification of Hemophilia Gene Therapy by Types
1.2.1 Global Hemophilia Gene Therapy Revenue Comparison by Types (2019-2024)
1.2.2 Global Hemophilia Gene Therapy Revenue Market Share by Types in 2018
1.2.3 Hemophilia A
1.2.4 Hemophilia B
1.3 Global Hemophilia Gene Therapy Market by Application
1.3.1 Global Hemophilia Gene Therapy Market Size and Market Share Comparison by Applications (2014-2024)
1.3.2 Hemophilia A Gene Therapy
1.3.3 Hemophilia B Gene Therapy
1.4 Global Hemophilia Gene Therapy Market by Regions
1.4.1 Global Hemophilia Gene Therapy Market Size (Million USD) Comparison by Regions (2014-2024)
1.4.1 North America (USA, Canada and Mexico) Hemophilia Gene Therapy Status and Prospect (2014-2024)
1.4.2 Europe (Germany, France, UK, Russia and Italy) Hemophilia Gene Therapy Status and Prospect (2014-2024)
1.4.3 Asia-Pacific (China, Japan, Korea, India and Southeast Asia) Hemophilia Gene Therapy Status and Prospect (2014-2024)
1.4.4 South America (Brazil, Argentina, Colombia) Hemophilia Gene Therapy Status and Prospect (2014-2024)
1.4.5 Middle East and Africa (Saudi Arabia, UAE, Egypt, Nigeria and South Africa) Hemophilia Gene Therapy Status and Prospect (2014-2024)
1.5 Global Market Size of Hemophilia Gene Therapy (2014-2024)
2 Manufacturers Profiles
2.1 Spark Therapeutics
2.1.1 Business Overview
2.1.2 Hemophilia Gene Therapy Type and Applications
2.1.2.1 Product A
2.1.2.2 Product B
2.1.3 Spark Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.2 Ultragenyx
2.2.1 Business Overview
2.2.2 Hemophilia Gene Therapy Type and Applications
2.2.2.1 Product A
2.2.2.2 Product B
2.2.3 Ultragenyx Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.3 Shire PLC
2.3.1 Business Overview
2.3.2 Hemophilia Gene Therapy Type and Applications
2.3.2.1 Product A
2.3.2.2 Product B
2.3.3 Shire PLC Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.4 Sangamo Therapeutics
2.4.1 Business Overview
2.4.2 Hemophilia Gene Therapy Type and Applications
2.4.2.1 Product A
2.4.2.2 Product B
2.4.3 Sangamo Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.5 Bioverativ
2.5.1 Business Overview
2.5.2 Hemophilia Gene Therapy Type and Applications
2.5.2.1 Product A
2.5.2.2 Product B
2.5.3 Bioverativ Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.6 BioMarin
2.6.1 Business Overview
2.6.2 Hemophilia Gene Therapy Type and Applications
2.6.2.1 Product A
2.6.2.2 Product B
2.6.3 BioMarin Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.7 uniQure
2.7.1 Business Overview
2.7.2 Hemophilia Gene Therapy Type and Applications
2.7.2.1 Product A
2.7.2.2 Product B
2.7.3 uniQure Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
2.8 Freeline Therapeutics
2.8.1 Business Overview
2.8.2 Hemophilia Gene Therapy Type and Applications
2.8.2.1 Product A
2.8.2.2 Product B
2.8.3 Freeline Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
3 Global Hemophilia Gene Therapy Market Competition, by Players
3.1 Global Hemophilia Gene Therapy Revenue and Share by Players (2014-2019)
3.2 Market Concentration Rate
3.2.1 Top 5 Hemophilia Gene Therapy Players Market Share
3.2.2 Top 10 Hemophilia Gene Therapy Players Market Share
3.3 Market Competition Trend
4 Global Hemophilia Gene Therapy Market Size by Regions
4.1 Global Hemophilia Gene Therapy Revenue and Market Share by Regions
4.2 North America Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
4.3 Europe Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
4.4 Asia-Pacific Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
4.5 South America Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
4.6 Middle East and Africa Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
5 North America Hemophilia Gene Therapy Revenue by Countries
5.1 North America Hemophilia Gene Therapy Revenue by Countries (2014-2019)
5.2 USA Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
5.3 Canada Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
5.4 Mexico Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
6 Europe Hemophilia Gene Therapy Revenue by Countries
6.1 Europe Hemophilia Gene Therapy Revenue by Countries (2014-2019)
6.2 Germany Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
6.3 UK Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
6.4 France Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
6.5 Russia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
6.6 Italy Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
7 Asia-Pacific Hemophilia Gene Therapy Revenue by Countries
7.1 Asia-Pacific Hemophilia Gene Therapy Revenue by Countries (2014-2019)
7.2 China Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
7.3 Japan Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
7.4 Korea Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
7.5 India Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
7.6 Southeast Asia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
8 South America Hemophilia Gene Therapy Revenue by Countries
8.1 South America Hemophilia Gene Therapy Revenue by Countries (2014-2019)
8.2 Brazil Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
8.3 Argentina Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
8.4 Colombia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
9 Middle East and Africa Revenue Hemophilia Gene Therapy by Countries
9.1 Middle East and Africa Hemophilia Gene Therapy Revenue by Countries (2014-2019)
9.2 Saudi Arabia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
9.3 UAE Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
9.4 Egypt Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
9.5 Nigeria Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
9.6 South Africa Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
10 Global Hemophilia Gene Therapy Market Segment by Type
10.1 Global Hemophilia Gene Therapy Revenue and Market Share by Type (2014-2019)
10.2 Global Hemophilia Gene Therapy Market Forecast by Type (2019-2024)
10.3 Hemophilia A Revenue Growth Rate (2014-2024)
10.4 Hemophilia B Revenue Growth Rate (2014-2024)
11 Global Hemophilia Gene Therapy Market Segment by Application
11.1 Global Hemophilia Gene Therapy Revenue Market Share by Application (2014-2019)
11.2 Hemophilia Gene Therapy Market Forecast by Application (2019-2024)
11.3 Hemophilia A Gene Therapy Revenue Growth (2014-2019)
11.4 Hemophilia B Gene Therapy Revenue Growth (2014-2019)
12 Global Hemophilia Gene Therapy Market Size Forecast (2019-2024)
12.1 Global Hemophilia Gene Therapy Market Size Forecast (2019-2024)
12.2 Global Hemophilia Gene Therapy Market Forecast by Regions (2019-2024)
12.3 North America Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
12.4 Europe Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
12.5 Asia-Pacific Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
12.6 South America Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
12.7 Middle East and Africa Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
13 Research Findings and Conclusion
14 Appendix
14.1 Methodology
14.2 Data Source
List of Tables and Figures
Figure Hemophilia Gene Therapy Picture
Table Product Specifications of Hemophilia Gene Therapy
Table Global Hemophilia Gene Therapy and Revenue (Million USD) Market Split by Product Type
Figure Global Hemophilia Gene Therapy Revenue Market Share by Types in 2018
Figure Hemophilia A Picture
Figure Hemophilia B Picture
Table Global Hemophilia Gene Therapy Revenue (Million USD) by Application (2014-2024)
Figure Hemophilia Gene Therapy Revenue Market Share by Applications in 2018
Figure Hemophilia A Gene Therapy Picture
Figure Hemophilia B Gene Therapy Picture
Table Global Market Hemophilia Gene Therapy Revenue (Million USD) Comparison by Regions 2014-2024
Figure North America Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (2014-2024)
Figure Europe Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (2014-2024)
Figure Asia-Pacific Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (2014-2024)
Figure South America Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (2014-2024)
Figure Middle East and Africa Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (2014-2024)
Figure Global Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (2014-2024)
Table Spark Therapeutics Basic Information, Manufacturing Base and Competitors
Table Spark Therapeutics Hemophilia Gene Therapy Type and Applications
Table Spark Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table Ultragenyx Basic Information, Manufacturing Base and Competitors
Table Ultragenyx Hemophilia Gene Therapy Type and Applications
Table Ultragenyx Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table Shire PLC Basic Information, Manufacturing Base and Competitors
Table Shire PLC Hemophilia Gene Therapy Type and Applications
Table Shire PLC Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table Sangamo Therapeutics Basic Information, Manufacturing Base and Competitors
Table Sangamo Therapeutics Hemophilia Gene Therapy Type and Applications
Table Sangamo Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table Bioverativ Basic Information, Manufacturing Base and Competitors
Table Bioverativ Hemophilia Gene Therapy Type and Applications
Table Bioverativ Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table BioMarin Basic Information, Manufacturing Base and Competitors
Table BioMarin Hemophilia Gene Therapy Type and Applications
Table BioMarin Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table uniQure Basic Information, Manufacturing Base and Competitors
Table uniQure Hemophilia Gene Therapy Type and Applications
Table uniQure Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table Freeline Therapeutics Basic Information, Manufacturing Base and Competitors
Table Freeline Therapeutics Hemophilia Gene Therapy Type and Applications
Table Freeline Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2018)
Table Global Hemophilia Gene Therapy Revenue (Million USD) by Players (2014-2019)
Table Global Hemophilia Gene Therapy Revenue Share by Players (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Share by Players in 2017
Figure Global Hemophilia Gene Therapy Revenue Share by Players in 2018
Figure Global Top 5 Players Hemophilia Gene Therapy Revenue Market Share in 2018
Figure Global Top 10 Players Hemophilia Gene Therapy Revenue Market Share in 2018
Figure Global Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate (%) (2014-2019)
Table Global Hemophilia Gene Therapy Revenue (Million USD) by Regions (2014-2019)
Table Global Hemophilia Gene Therapy Revenue Market Share by Regions (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Market Share by Regions (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Market Share by Regions in 2018
Figure North America Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Europe Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Asia-Pacific Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure South America Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Middle East and Africa Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Table North America Hemophilia Gene Therapy Revenue by Countries (2014-2019)
Table North America Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure North America Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure North America Hemophilia Gene Therapy Revenue Market Share by Countries in 2018
Figure USA Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Canada Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Mexico Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Table Europe Hemophilia Gene Therapy Revenue (Million USD) by Countries (2014-2019)
Figure Europe Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure Europe Hemophilia Gene Therapy Revenue Market Share by Countries in 2018
Figure Germany Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure UK Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure France Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Russia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Italy Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Table Asia-Pacific Hemophilia Gene Therapy Revenue (Million USD) by Countries (2014-2019)
Figure Asia-Pacific Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure Asia-Pacific Hemophilia Gene Therapy Revenue Market Share by Countries in 2018
Figure China Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Japan Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Korea Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure India Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Southeast Asia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Table South America Hemophilia Gene Therapy Revenue by Countries (2014-2019)
Table South America Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure South America Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure South America Hemophilia Gene Therapy Revenue Market Share by Countries in 2018
Figure Brazil Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Argentina Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Colombia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Table Middle East and Africa Hemophilia Gene Therapy Revenue (Million USD) by Countries (2014-2019)
Table Middle East and Africa Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure Middle East and Africa Hemophilia Gene Therapy Revenue Market Share by Countries (2014-2019)
Figure Middle East and Africa Hemophilia Gene Therapy Revenue Market Share by Countries in 2018
Figure Saudi Arabia Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure UAE Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Egypt Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure Nigeria Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Figure South Africa Hemophilia Gene Therapy Revenue and Growth Rate (2014-2019)
Table Global Hemophilia Gene Therapy Revenue (Million USD) by Type (2014-2019)
Table Global Hemophilia Gene Therapy Revenue Share by Type (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Share by Type (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Share by Type in 2019
Table Global Hemophilia Gene Therapy Revenue Forecast by Type (2019-2024)
Figure Global Hemophilia Gene Therapy Market Share Forecast by Type (2019-2024)
Figure Global Hemophilia A Revenue Growth Rate (2014-2019)
Figure Global Hemophilia B Revenue Growth Rate (2014-2019)
Table Global Hemophilia Gene Therapy Revenue by Application (2014-2019)
Table Global Hemophilia Gene Therapy Revenue Share by Application (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Share by Application (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Share by Application in 2019
Table Global Hemophilia Gene Therapy Revenue Forecast by Application (2019-2024)
Figure Global Hemophilia Gene Therapy Market Share Forecast by Application (2019-2024)
Figure Global Hemophilia A Gene Therapy Revenue Growth Rate (2014-2019)
Figure Global Hemophilia B Gene Therapy Revenue Growth Rate (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue (Million USD) and Growth Rate Forecast (2019-2024)
Table Global Hemophilia Gene Therapy Revenue (Million USD) Forecast by Regions (2019-2024)
Figure Global Hemophilia Gene Therapy Revenue Market Share Forecast by Regions (2019-2024)
Figure North America Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
Figure Europe Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
Figure Asia-Pacific Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
Figure South America Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)
Figure Middle East and Africa Hemophilia Gene Therapy Revenue Market Forecast (2019-2024)

Download our eBo