Report Detail
Global Hemophilia Gene Therapy Market Size, Status and Forecast 2019-2025
- RnM2717051 |
- 20 March, 2019 |
- Global |
- 99 Pages |
- QYResearch |
- 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.
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
- 15.1.1 Methodology/Research Approach
- 15.2 Disclaimer
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 Size, Status and Forecast 2019-2025. 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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