Global Hemophilia Gene Therapy Market Size, Status and Forecast 2025

Published: June 2018
No. of Pages: 91
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Hemophilia is a genetic disorder that impairs blood clotting. When one of the genes that encode for blood clotting factors is absent or deficient, the smallest injury can trigger serious bleeding episodes. Since these genes are located on the X chromosome, the disease is much more common in males than females.

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 major players in global Hemophilia Gene Therapy market include
Spark Therapeutics
Ultragenyx
Shire PLC
Sangamo Therapeutics
Bioverativ
BioMarin
uniQure
Freeline Therapeutics

On the basis on the end users/applications, this report covers
Hemophilia A Gene Therapy
Hemophilia B Gene Therapy

Published By: QYResearch Group
Product Code: QYResearch Group258642


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