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Global Hemophilia Gene Therapy Market Size, Status and Forecast 2019-2025
Published Date: March 2019
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Report Code: QYRE-Othe-283
Home | Market Reports | Health | Health Conditions | Genetic Disorders
Global Hemophilia Gene Therapy Market Size Status and Forecast 2019 to 2025

Global Hemophilia Gene Therapy Market Size, Status and Forecast 2019-2025

Code: QYRE-Othe-283
Report
March 2019
99 Pages
QYResearch
Region: Global,
Description
Table of Content
Tables & Figures

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.
In 2018, the global Hemophilia Gene Therapy market size was xx million US$ and it is expected to reach xx million US$ by the end of 2025, with a CAGR of xx% during 2019-2025.

This report focuses on the global Hemophilia Gene Therapy status, future forecast, growth opportunity, key market and key players. The study objectives are to present the Hemophilia Gene Therapy development in United States, Europe and China.

The key players covered in this study
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

Market segment by Type, the product can be split into
Hemophilia A
Hemophilia B

Market segment by Application, split into
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy

Market segment by Regions/Countries, this report covers
United States
Europe
China
Japan
Southeast Asia
India
Central & South America

The study objectives of this report are:
To analyze global Hemophilia Gene Therapy status, future forecast, growth opportunity, key market and key players.
To present the Hemophilia Gene Therapy development in United States, Europe and China.
To strategically profile the key players and comprehensively analyze their development plan and strategies.
To define, describe and forecast the market by product type, market and key regions.

In this study, the years considered to estimate the market size of Hemophilia Gene Therapy are as follows:
History Year: 2014-2018
Base Year: 2018
Estimated Year: 2019
Forecast Year 2019 to 2025
For the data information by region, company, type and application, 2018 is considered as the base year. Whenever data information was unavailable for the base year, the prior year has been considered.

Table of Contents

1 Report Overview
1.1 Study Scope
1.2 Key Market Segments
1.3 Players Covered
1.4 Market Analysis by Type
1.4.1 Global Hemophilia Gene Therapy Market Size Growth Rate by Type (2014-2025)
1.4.2 Hemophilia A
1.4.3 Hemophilia B
1.5 Market by Application
1.5.1 Global Hemophilia Gene Therapy Market Share by Application (2014-2025)
1.5.2 Hemophilia A Gene Therapy
1.5.3 Hemophilia B Gene Therapy
1.6 Study Objectives
1.7 Years Considered

2 Global Growth Trends
2.1 Hemophilia Gene Therapy Market Size
2.2 Hemophilia Gene Therapy Growth Trends by Regions
2.2.1 Hemophilia Gene Therapy Market Size by Regions (2014-2025)
2.2.2 Hemophilia Gene Therapy Market Share by Regions (2014-2019)
2.3 Industry Trends
2.3.1 Market Top Trends
2.3.2 Market Drivers
2.3.3 Market Opportunities

3 Market Share by Key Players
3.1 Hemophilia Gene Therapy Market Size by Manufacturers
3.1.1 Global Hemophilia Gene Therapy Revenue by Manufacturers (2014-2019)
3.1.2 Global Hemophilia Gene Therapy Revenue Market Share by Manufacturers (2014-2019)
3.1.3 Global Hemophilia Gene Therapy Market Concentration Ratio (CR5 and HHI)
3.2 Hemophilia Gene Therapy Key Players Head office and Area Served
3.3 Key Players Hemophilia Gene Therapy Product/Solution/Service
3.4 Date of Enter into Hemophilia Gene Therapy Market
3.5 Mergers & Acquisitions, Expansion Plans

4 Breakdown Data by Type and Application
4.1 Global Hemophilia Gene Therapy Market Size by Type (2014-2019)
4.2 Global Hemophilia Gene Therapy Market Size by Application (2014-2019)

5 United States
5.1 United States Hemophilia Gene Therapy Market Size (2014-2019)
5.2 Hemophilia Gene Therapy Key Players in United States
5.3 United States Hemophilia Gene Therapy Market Size by Type
5.4 United States Hemophilia Gene Therapy Market Size by Application

6 Europe
6.1 Europe Hemophilia Gene Therapy Market Size (2014-2019)
6.2 Hemophilia Gene Therapy Key Players in Europe
6.3 Europe Hemophilia Gene Therapy Market Size by Type
6.4 Europe Hemophilia Gene Therapy Market Size by Application

7 China
7.1 China Hemophilia Gene Therapy Market Size (2014-2019)
7.2 Hemophilia Gene Therapy Key Players in China
7.3 China Hemophilia Gene Therapy Market Size by Type
7.4 China Hemophilia Gene Therapy Market Size by Application

8 Japan
8.1 Japan Hemophilia Gene Therapy Market Size (2014-2019)
8.2 Hemophilia Gene Therapy Key Players in Japan
8.3 Japan Hemophilia Gene Therapy Market Size by Type
8.4 Japan Hemophilia Gene Therapy Market Size by Application

9 Southeast Asia
9.1 Southeast Asia Hemophilia Gene Therapy Market Size (2014-2019)
9.2 Hemophilia Gene Therapy Key Players in Southeast Asia
9.3 Southeast Asia Hemophilia Gene Therapy Market Size by Type
9.4 Southeast Asia Hemophilia Gene Therapy Market Size by Application

10 India
10.1 India Hemophilia Gene Therapy Market Size (2014-2019)
10.2 Hemophilia Gene Therapy Key Players in India
10.3 India Hemophilia Gene Therapy Market Size by Type
10.4 India Hemophilia Gene Therapy Market Size by Application

11 Central & South America
11.1 Central & South America Hemophilia Gene Therapy Market Size (2014-2019)
11.2 Hemophilia Gene Therapy Key Players in Central & South America
11.3 Central & South America Hemophilia Gene Therapy Market Size by Type
11.4 Central & South America Hemophilia Gene Therapy Market Size by Application

12 International Players Profiles
12.1 Spark Therapeutics
12.1.1 Spark Therapeutics Company Details
12.1.2 Company Description and Business Overview
12.1.3 Hemophilia Gene Therapy Introduction
12.1.4 Spark Therapeutics Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.1.5 Spark Therapeutics Recent Development
12.2 Ultragenyx
12.2.1 Ultragenyx Company Details
12.2.2 Company Description and Business Overview
12.2.3 Hemophilia Gene Therapy Introduction
12.2.4 Ultragenyx Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.2.5 Ultragenyx Recent Development
12.3 Shire PLC
12.3.1 Shire PLC Company Details
12.3.2 Company Description and Business Overview
12.3.3 Hemophilia Gene Therapy Introduction
12.3.4 Shire PLC Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.3.5 Shire PLC Recent Development
12.4 Sangamo Therapeutics
12.4.1 Sangamo Therapeutics Company Details
12.4.2 Company Description and Business Overview
12.4.3 Hemophilia Gene Therapy Introduction
12.4.4 Sangamo Therapeutics Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.4.5 Sangamo Therapeutics Recent Development
12.5 Bioverativ
12.5.1 Bioverativ Company Details
12.5.2 Company Description and Business Overview
12.5.3 Hemophilia Gene Therapy Introduction
12.5.4 Bioverativ Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.5.5 Bioverativ Recent Development
12.6 BioMarin
12.6.1 BioMarin Company Details
12.6.2 Company Description and Business Overview
12.6.3 Hemophilia Gene Therapy Introduction
12.6.4 BioMarin Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.6.5 BioMarin Recent Development
12.7 uniQure
12.7.1 uniQure Company Details
12.7.2 Company Description and Business Overview
12.7.3 Hemophilia Gene Therapy Introduction
12.7.4 uniQure Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.7.5 uniQure Recent Development
12.8 Freeline Therapeutics
12.8.1 Freeline Therapeutics Company Details
12.8.2 Company Description and Business Overview
12.8.3 Hemophilia Gene Therapy Introduction
12.8.4 Freeline Therapeutics Revenue in Hemophilia Gene Therapy Business (2014-2019)
12.8.5 Freeline Therapeutics Recent Development

13 Market Forecast 2019-2025
13.1 Market Size Forecast by Regions
13.2 United States
13.3 Europe
13.4 China
13.5 Japan
13.6 Southeast Asia
13.7 India
13.8 Central & South America
13.9 Market Size Forecast by Product (2019-2025)
13.10 Market Size Forecast by Application (2019-2025)

14 Analyst's Viewpoints/Conclusions

15 Appendix
15.1 Research Methodology
15.1.1 Methodology/Research Approach
15.1.1.1 Research Programs/Design
15.1.1.2 Market Size Estimation
12.1.1.3 Market Breakdown and Data Triangulation
15.1.2 Data Source
15.1.2.1 Secondary Sources
15.1.2.2 Primary Sources
15.2 Disclaimer
15.3 Author Details

List of Tables and Figures

Table Hemophilia Gene Therapy Key Market Segments
Table Key Players Hemophilia Gene Therapy Covered
Table Global Hemophilia Gene Therapy Market Size Growth Rate by Type 2014-2025 (Million US$)
Figure Global Hemophilia Gene Therapy Market Size Market Share by Type 2014-2025
Figure Hemophilia A Figures
Table Key Players of Hemophilia A
Figure Hemophilia B Figures
Table Key Players of Hemophilia B
Table Global Hemophilia Gene Therapy Market Size Growth by Application 2014-2025 (Million US$)
Figure Hemophilia A Gene Therapy Case Studies
Figure Hemophilia B Gene Therapy Case Studies
Figure Hemophilia Gene Therapy Report Years Considered
Table Global Hemophilia Gene Therapy Market Size 2014-2025 (Million US$)
Figure Global Hemophilia Gene Therapy Market Size and Growth Rate 2014-2025 (Million US$)
Table Global Hemophilia Gene Therapy Market Size by Regions 2014-2025 (Million US$)
Table Global Hemophilia Gene Therapy Market Size by Regions 2014-2019 (Million US$)
Table Global Hemophilia Gene Therapy Market Share by Regions 2014-2019
Figure Global Hemophilia Gene Therapy Market Share by Regions 2014-2019
Figure Global Hemophilia Gene Therapy Market Share by Regions 2019
Table Market Top Trends
Table Global Hemophilia Gene Therapy Revenue by Manufacturers (2014-2019) (Million US$)
Table Global Hemophilia Gene Therapy Market Share by Manufacturers (2014-2019)
Figure Global Hemophilia Gene Therapy Market Share by Manufacturers in 2018
Table Global Hemophilia Gene Therapy Manufacturers Market Concentration Ratio (CR5 and HHI)
Table Key Players Head office and Area Served
Table Key Players Hemophilia Gene Therapy Product/Solution/Service
Table Date of Enter into Hemophilia Gene Therapy Market
Table Mergers & Acquisitions, Expansion Plans
Table Global Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table Global Hemophilia Gene Therapy Market Size Share by Type (2014-2019)
Figure Global Hemophilia Gene Therapy Market Size Market Share by Type (2014-2019)
Table Global Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table Global Hemophilia Gene Therapy Market Size Share by Application (2014-2019)
Figure Global Hemophilia Gene Therapy Market Size Market Share by Application (2014-2019)
Figure Global Hemophilia Gene Therapy Revenue Market Share by Application in 2018
Figure United States Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table United States Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table United States Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table United States Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table United States Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table United States Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table United States Hemophilia Gene Therapy Market Share by Application (2014-2019)
Figure Europe Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table Europe Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table Europe Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table Europe Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table Europe Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table Europe Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table Europe Hemophilia Gene Therapy Market Share by Application (2014-2019)
Figure China Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table China Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table China Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table China Hemophilia Gene Therapy Market Size by Type (2014-20189) (Million US$)
Table China Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table China Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table China Hemophilia Gene Therapy Market Share by Application (2014-2019)
Figure Japan Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table Japan Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table Japan Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table Japan Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table Japan Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table Japan Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table Japan Hemophilia Gene Therapy Market Share by Application (2014-2019)
Figure Southeast Asia Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table Southeast Asia Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table Southeast Asia Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table Southeast Asia Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table Southeast Asia Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table Southeast Asia Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table Southeast Asia Hemophilia Gene Therapy Market Share by Application (2014-2019)
Figure India Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table India Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table India Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table India Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table India Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table India Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table India Hemophilia Gene Therapy Market Share by Application (2014-2019)
Figure Central & South America Hemophilia Gene Therapy Market Size 2014-2019 (Million US$)
Table Central & South America Key Players Hemophilia Gene Therapy Revenue (2018-2019) (Million US$)
Table Central & South America Key Players Hemophilia Gene Therapy Market Share (2018-2019)
Table Central & South America Hemophilia Gene Therapy Market Size by Type (2014-2019) (Million US$)
Table Central & South America Hemophilia Gene Therapy Market Share by Type (2014-2019)
Table Central & South America Hemophilia Gene Therapy Market Size by Application (2014-2019) (Million US$)
Table Central & South America Hemophilia Gene Therapy Market Share by Application (2014-2019)
Table Spark Therapeutics Company Details
Table Spark Therapeutics Revenue in Hemophilia Gene Therapy Business (2014-2019) (Million US$)
Figure Spark Therapeutics Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table Spark Therapeutics Recent Development
Table Ultragenyx Company Details
Table Ultragenyx Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure Ultragenyx Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table Ultragenyx Recent Development
Table Shire PLC Company Details
Table Shire PLC Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure Shire PLC Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table Shire PLC Recent Development
Table Sangamo Therapeutics Company Details
Table Sangamo Therapeutics Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure Sangamo Therapeutics Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table Sangamo Therapeutics Recent Development
Table Bioverativ Company Details
Table Bioverativ Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure Bioverativ Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table Bioverativ Recent Development
Table BioMarin Company Details
Table BioMarin Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure BioMarin Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table BioMarin Recent Development
Table uniQure Company Details
Table uniQure Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure uniQure Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table uniQure Recent Development
Table Freeline Therapeutics Company Details
Table Freeline Therapeutics Revenue in Hemophilia Gene Therapy Business (2014-2019)(Million US$)
Figure Freeline Therapeutics Revenue Growth Rate in Hemophilia Gene Therapy Business (2014-2019)
Table Freeline Therapeutics Recent Development
Table Global Hemophilia Gene Therapy Market Size by Regions (Million US$) 2019-2025
Figure Global Hemophilia Gene Therapy Market Size Share by Regions (2019-2025)
Figure Global Hemophilia Gene Therapy Market Size Share by Regions in 2025
Figure United States Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Figure Europe Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Figure China Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Figure Japan Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Figure Southeast Asia Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Figure India Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Figure Central & South America Hemophilia Gene Therapy Market Size Forecast (2019-2025)(Million USD)
Table Global Hemophilia Gene Therapy Market Size by Product (2019-2025) (Million US$)
Figure Global Hemophilia Gene Therapy Market Size by Product (2019-2025)
Figure Global Hemophilia Gene Therapy Market Size by Product in 2025
Table Global Hemophilia Gene Therapy Market Size by Application (2019-2025) (Million US$)
Figure Global Hemophilia Gene Therapy Market Size by Application (2019-2025)
Figure Global Hemophilia Gene Therapy Market Size by Application in 2025
Table Research Programs/Design for This Report
Figure Bottom-up and Top-down Approaches for This Report
Figure Data Triangulation
Table Key Data Information from Secondary Sources
Table Key Data Information from Primary Sources

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