Global Hemophilia Gene Therapy Market Size, Status and Forecast 2021-2027

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.

Market Analysis and Insights: Global Hemophilia Gene Therapy Market
The global Hemophilia Gene Therapy market size is projected to reach US$ XX million by 2027, from US$ XX million in 2020, at a CAGR of XX% during 2021-2027.
With industry-standard accuracy in analysis and high data integrity, the report makes a brilliant attempt to unveil key opportunities available in the global Hemophilia Gene Therapy market to help players in achieving a strong market position. Buyers of the report can access verified and reliable market forecasts, including those for the overall size of the global Hemophilia Gene Therapy market in terms of revenue.
On the whole, the report proves to be an effective tool that players can use to gain a competitive edge over their competitors and ensure lasting success in the global Hemophilia Gene Therapy market. All of the findings, data, and information provided in the report are validated and revalidated with the help of trustworthy sources. The analysts who have authored the report took a unique and industry-best research and analysis approach for an in-depth study of the global Hemophilia Gene Therapy market.

Global Hemophilia Gene Therapy Scope and Market Size
Hemophilia Gene Therapy market is segmented by company, region (country), by Type, and by Application. Players, stakeholders, and other participants in the global Hemophilia Gene Therapy market will be able to gain the upper hand as they use the report as a powerful resource. The segmental analysis focuses on revenue and forecast by Type and by Application in terms of revenue and forecast for the period 2016-2027.

Segment by Type
Hemophilia A
Hemophilia B

Segment by Application
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy

By Region
North America
U.S.
Canada
Europe
Germany
France
U.K.
Italy
Russia
Nordic
Rest of Europe
Asia-Pacific
China
Japan
South Korea
Southeast Asia
India
Australia
Rest of Asia
Latin America
Mexico
Brazil
Rest of Latin America
Middle East & Africa
Turkey
Saudi Arabia
UAE
Rest of MEA

By Company
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

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