Hemophilia Gene Therapy Market (Updated Version Available)

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.

The report offers detailed coverage of Hemophilia Gene Therapy industry and main market trends. The market research includes historical and forecast market data, demand, application details, price trends, and company shares of the leading Hemophilia Gene Therapy by geography. The report splits the market size, by volume and value, on the basis of application type and geography.

The report forecast global Hemophilia Gene Therapy market to grow to reach xxx Million USD in 2020 with a CAGR of xx% during the period 2021-2025.

First, this report covers the present status and the future prospects of the global Hemophilia Gene Therapy market for 2015-2025.
And in this report, we analyze global market from 5 geographies: Asia-Pacific[China, Southeast Asia, India, Japan, Korea, Western Asia], Europe[Germany, UK, France, Italy, Russia, Spain, Netherlands, Turkey, Switzerland], North America[United States, Canada, Mexico], Middle East & Africa[GCC, North Africa, South Africa], South America[Brazil, Argentina, Columbia, Chile, Peru].

Key Companies
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

At the same time, we classify Hemophilia Gene Therapy according to the type, application by geography. More importantly, the report includes major countries market based on the type and application.

Market Segment as follows:
Market by Order Type
Hemophilia A
Hemophilia B
Market by Application
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy

By Region
Asia-Pacific[China, Southeast Asia, India, Japan, Korea, Western Asia]
Europe[Germany, UK, France, Italy, Russia, Spain, Netherlands, Turkey, Switzerland]
North America[United States, Canada, Mexico]
Middle East & Africa[GCC, North Africa, South Africa]
South America[Brazil, Argentina, Columbia, Chile, Peru]

The research provides answers to the following key questions:
• What is the estimated growth rate and market share and size of the Hemophilia Gene Therapy market for the forecast period 2021 - 2025?
• What are the driving forces in the Hemophilia Gene Therapy market for the forecast period 2021 - 2025?
• Who are the prominent market players and how have they gained a competitive edge over other competitors?
• What are the market trends influencing the progress of the Hemophilia Gene Therapy industry worldwide?
• What are the major challenges and threats restricting the progress of the industry?
• What opportunities does the market hold for the prominent market players?