Hemophilia Gene Therapy Market KKey Procurement Criteria, Drivers and Trends Analysis by 2025- QY Research, Inc

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.

This report studies the global Hemophilia Gene Therapy market size, industry status and forecast, competition landscape and growth opportunity. This research report categorizes the global Hemophilia Gene Therapy market by companies, region, type and end-use industry.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.

This report focuses on the global top players, covered

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

With the given market data, QYResearch offers customizations according to the company's specific needs. The following customization options are available for the report:

Regional and country-level analysis of the Hemophilia Gene Therapy market, by end-use.

Detailed analysis and profiles of additional market players.

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Market size split by Region

North America

Asia-Pacific

Europe

Central & South America

Middle East & Africa

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