Hemophilia Gene Therapy Market Report – Research, Industry Analysis Reports and Market Demands

Global Hemophilia Gene Therapy Market Growth (Status and Outlook) 2019-2024

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Table of Content

2019-2024 Global Hemophilia Gene Therapy Market Report (Status and Outlook)

1 Scope of the Report

2 Executive Summary

2.1 World Market Overview
- 2.1.1 Global Hemophilia Gene Therapy Market Size 2014-2024
- 2.1.2 Hemophilia Gene Therapy Market Size CAGR by Region
2.2 Hemophilia Gene Therapy Segment by Type
- 2.2.1 Hemophilia A
- 2.2.2 Hemophilia B
2.3 Hemophilia Gene Therapy Market Size by Type
- 2.3.1 Global Hemophilia Gene Therapy Market Size Market Share by Type (2014-2019)
- 2.3.2 Global Hemophilia Gene Therapy Market Size Growth Rate by Type (2014-2019)
2.4 Hemophilia Gene Therapy Segment by Application
- 2.4.1 Hemophilia A Gene Therapy
- 2.4.2 Hemophilia B Gene Therapy
2.5 Hemophilia Gene Therapy Market Size by Application
- 2.5.1 Global Hemophilia Gene Therapy Market Size Market Share by Application (2014-2019)
- 2.5.2 Global Hemophilia Gene Therapy Market Size Growth Rate by Application (2014-2019)

3 Global Hemophilia Gene Therapy by Players

3.1 Global Hemophilia Gene Therapy Market Size Market Share by Players
- 3.1.1 Global Hemophilia Gene Therapy Market Size by Players (2017-2019)
- 3.1.2 Global Hemophilia Gene Therapy Market Size Market Share by Players (2017-2019)
3.2 Global Hemophilia Gene Therapy Key Players Head office and Products Offered
3.3 Market Concentration Rate Analysis
- 3.3.1 Competition Landscape Analysis
- 3.3.2 Concentration Ratio (CR3, CR5 and CR10) (2017-2019)
3.4 New Products and Potential Entrants
3.5 Mergers & Acquisitions, Expansion

4 Hemophilia Gene Therapy by Regions

4.1 Hemophilia Gene Therapy Market Size by Regions
4.2 Americas Hemophilia Gene Therapy Market Size Growth
4.3 APAC Hemophilia Gene Therapy Market Size Growth
4.4 Europe Hemophilia Gene Therapy Market Size Growth
4.5 Middle East & Africa Hemophilia Gene Therapy Market Size Growth

5 Americas

5.1 Americas Hemophilia Gene Therapy Market Size by Countries
5.2 Americas Hemophilia Gene Therapy Market Size by Type
5.3 Americas Hemophilia Gene Therapy Market Size by Application
5.4 United States
5.5 Canada
5.6 Mexico
5.7 Key Economic Indicators of Few Americas Countries

6 APAC

6.1 APAC Hemophilia Gene Therapy Market Size by Countries
6.2 APAC Hemophilia Gene Therapy Market Size by Type
6.3 APAC Hemophilia Gene Therapy Market Size by Application
6.4 China
6.5 Japan
6.6 Korea
6.7 Southeast Asia
6.8 India
6.9 Australia
6.10 Key Economic Indicators of Few APAC Countries

7 Europe

7.1 Europe Hemophilia Gene Therapy by Countries
7.2 Europe Hemophilia Gene Therapy Market Size by Type
7.3 Europe Hemophilia Gene Therapy Market Size by Application
7.4 Germany
7.5 France
7.6 UK
7.7 Italy
7.8 Russia
7.9 Spain
7.10 Key Economic Indicators of Few Europe Countries

8 Middle East & Africa

8.1 Middle East & Africa Hemophilia Gene Therapy by Countries
8.2 Middle East & Africa Hemophilia Gene Therapy Market Size by Type
8.3 Middle East & Africa Hemophilia Gene Therapy Market Size by Application
8.4 Egypt
8.5 South Africa
8.6 Israel
8.7 Turkey
8.8 GCC Countries

9 Market Drivers, Challenges and Trends

9.1 Market Drivers and Impact
- 9.1.1 Growing Demand from Key Regions
- 9.1.2 Growing Demand from Key Applications and Potential Industries
9.2 Market Challenges and Impact
9.3 Market Trends

10 Global Hemophilia Gene Therapy Market Forecast

10.1 Global Hemophilia Gene Therapy Market Size Forecast (2019-2024)
10.2 Global Hemophilia Gene Therapy Forecast by Regions
- 10.2.1 Global Hemophilia Gene Therapy Forecast by Regions (2019-2024)
- 10.2.2 Americas Market Forecast
- 10.2.3 APAC Market Forecast
- 10.2.4 Europe Market Forecast
- 10.2.5 Middle East & Africa Market Forecast
10.3 Americas Forecast by Countries
- 10.3.1 United States Market Forecast
- 10.3.2 Canada Market Forecast
- 10.3.3 Mexico Market Forecast
- 10.3.4 Brazil Market Forecast
10.4 APAC Forecast by Countries
- 10.4.1 China Market Forecast
- 10.4.2 Japan Market Forecast
- 10.4.3 Korea Market Forecast
- 10.4.4 Southeast Asia Market Forecast
- 10.4.5 India Market Forecast
- 10.4.6 Australia Market Forecast
10.5 Europe Forecast by Countries
- 10.5.1 Germany Market Forecast
- 10.5.2 France Market Forecast
- 10.5.3 UK Market Forecast
- 10.5.4 Italy Market Forecast
- 10.5.5 Russia Market Forecast
- 10.5.6 Spain Market Forecast
10.6 Middle East & Africa Forecast by Countries
- 10.6.1 Egypt Market Forecast
- 10.6.2 South Africa Market Forecast
- 10.6.3 Israel Market Forecast
- 10.6.4 Turkey Market Forecast
- 10.6.5 GCC Countries Market Forecast
10.7 Global Hemophilia Gene Therapy Forecast by Type
10.8 Global Hemophilia Gene Therapy Forecast by Application

11 Key Players Analysis

11.1 Spark Therapeutics
- 11.1.1 Company Details
- 11.1.2 Hemophilia Gene Therapy Product Offered
- 11.1.3 Spark Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.1.4 Main Business Overview
- 11.1.5 Spark Therapeutics News
11.2 Ultragenyx
- 11.2.1 Company Details
- 11.2.2 Hemophilia Gene Therapy Product Offered
- 11.2.3 Ultragenyx Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.2.4 Main Business Overview
- 11.2.5 Ultragenyx News
11.3 Shire PLC
- 11.3.1 Company Details
- 11.3.2 Hemophilia Gene Therapy Product Offered
- 11.3.3 Shire PLC Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.3.4 Main Business Overview
- 11.3.5 Shire PLC News
11.4 Sangamo Therapeutics
- 11.4.1 Company Details
- 11.4.2 Hemophilia Gene Therapy Product Offered
- 11.4.3 Sangamo Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.4.4 Main Business Overview
- 11.4.5 Sangamo Therapeutics News
11.5 Bioverativ
- 11.5.1 Company Details
- 11.5.2 Hemophilia Gene Therapy Product Offered
- 11.5.3 Bioverativ Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.5.4 Main Business Overview
- 11.5.5 Bioverativ News
11.6 BioMarin
- 11.6.1 Company Details
- 11.6.2 Hemophilia Gene Therapy Product Offered
- 11.6.3 BioMarin Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.6.4 Main Business Overview
- 11.6.5 BioMarin News
11.7 uniQure
- 11.7.1 Company Details
- 11.7.2 Hemophilia Gene Therapy Product Offered
- 11.7.3 uniQure Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.7.4 Main Business Overview
- 11.7.5 uniQure News
11.8 Freeline Therapeutics
- 11.8.1 Company Details
- 11.8.2 Hemophilia Gene Therapy Product Offered
- 11.8.3 Freeline Therapeutics Hemophilia Gene Therapy Revenue, Gross Margin and Market Share (2017-2019)
- 11.8.4 Main Business Overview
- 11.8.5 Freeline Therapeutics News

12 Research Findings and Conclusion

Description

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.
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.

According to this study, over the next five years the Hemophilia Gene Therapy market will register a xx% CAGR in terms of revenue, the global market size will reach US$ xx million by 2024, from US$ xx million in 2019. In particular, this report presents the global revenue market share of key companies in Hemophilia Gene Therapy business, shared in Chapter 3.

This report presents a comprehensive overview, market shares and growth opportunities of Hemophilia Gene Therapy market by product type, application, key companies and key regions.

This study considers the Hemophilia Gene Therapy value generated from the sales of the following segments:

Segmentation by product type: breakdown data from 2014 to 2019 in Section 2.3; and forecast to 2024 in section 10.7.
Hemophilia A
Hemophilia B
Segmentation by application: breakdown data from 2014 to 2019, in Section 2.4; and forecast to 2024 in section 10.8.
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy

This report also splits the market by region: Breakdown data in Chapter 4, 5, 6, 7 and 8.
Americas
United States
Canada
Mexico
Brazil
APAC
China
Japan
Korea
Southeast Asia
India
Australia
Europe
Germany
France
UK
Italy
Russia
Spain
Middle East & Africa
Egypt
South Africa
Israel
Turkey
GCC Countries

The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report: Breakdown data in in Chapter 3.
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

In addition, this report discusses the key drivers influencing market growth, opportunities, the challenges and the risks faced by key players and the market as a whole. It also analyzes key emerging trends and their impact on present and future development.

Research objectives
To study and analyze the global Hemophilia Gene Therapy market size by key regions/countries, product type and application, history data from 2014 to 2018, and forecast to 2024.
To understand the structure of Hemophilia Gene Therapy market by identifying its various subsegments.
Focuses on the key global Hemophilia Gene Therapy players, to define, describe and analyze the value, market share, market competition landscape, SWOT analysis and development plans in next few years.
To analyze the Hemophilia Gene Therapy with respect to individual growth trends, future prospects, and their contribution to the total market.
To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).
To project the size of Hemophilia Gene Therapy submarkets, with respect to key regions (along with their respective key countries).
To analyze competitive developments such as expansions, agreements, new product launches and acquisitions in the market.
To strategically profile the key players and comprehensively analyze their growth strategies.