Report Detail
Global Hemophilia Gene Therapy Market 2019 by Company, Regions, Type and Application, Forecast to 2024
- RnM2940199 |
- 14 February, 2019 |
- Global |
- 116 Pages |
- GIR (Global Info Research) |
- Other
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
Table of Contents
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
Summary:
Get latest Market Research Reports on Hemophilia Gene Therapy . Industry analysis & Market Report on Hemophilia Gene Therapy is a syndicated market report, published as Global Hemophilia Gene Therapy Market 2019 by Company, Regions, Type and Application, Forecast to 2024. It is complete Research Study and Industry Analysis of Hemophilia Gene Therapy market, to understand, Market Demand, Growth, trends analysis and Factor Influencing market.
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