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.
Scope of the Report:
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 hemophi ...
Table of Contents
1 Hemophilia Gene Therapy Market Overview
1.1 Product Overview and Scope of Hemophilia Gene Therapy
1.2 Classification of Hemophilia Gene Therapy by Types
1.2.1 Global Hemophilia Gene Therapy Revenue Comparison by Types (2019-2024)
1.2.2 Global Hemophilia Gene Therapy Revenue Market Share by Types in 2018
1.2.3 Hemophilia A
1.2.4 Hemophilia B
1.3 Global Hemophilia Gene Therapy Market by Application
1.3.1 Global Hemophilia Gene Therapy Market Size and Market Share Comparison by Applications (2014-2024)
1.3.2 Hemophilia A Gene Therapy
1.3.3 Hemophilia B Gene Therapy
1.4 Global Hemophilia Gene Therapy Market by Regions
1.4.1 Global Hemophilia Gene Therapy Market Size (Million USD) Comparison by Regions (2014-2024)
1.4.1 North America (USA, Canada and Mexico) Hemophilia Gene Therapy Status and Prospect (2014-2024)
1.4.2 Europe (Germany, France, UK, Russia and Italy) Hemophilia ...