Search4Research.com adds latest research report on “Global Hemophilia Gene Therapy Market”, it include and classifies the Global Hemophilia Gene Therapy Market in a precise manner to offer detailed insights about the aspects responsible for augmenting as well as future trends and growth prospect details for business development.
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.
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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.
This study considers the Hemophilia Gene Therapy value and volume generated from the sales of the following segments:
Segmentation by product type:
- Hemophilia A
- Hemophilia B
Segmentation by application:
- Hemophilia A Gene Therapy
- Hemophilia B Gene Therapy
This report also splits the market by region:
- Middle East & Africa
- GCC Countries
The report also presents the market competition landscape and a corresponding detailed analysis of the major vendor/manufacturers in the market. The key manufacturers covered in this report:
- Spark Therapeutics
- Shire PLC
- Sangamo Therapeutics
- Freeline Therapeutics
Benefits of leading players
- Enhance productivity and optimizing back end manufacturing processes
- Product enhancement through integrating new strategies involving big data, advanced analytics into traditional manufacturing processes
- Growing businesses through serving into new application areas and identifying pockets of growth in emerging markets
- Focusing on cost effective production of devices with stability and robustness
- Strategies for Product differentiation and adjusting to the life cycle changes
- Strengthening collaboration with suppliers and distributors
- More focused strategies are found in the report
- To study and analyze the global Hemophilia Gene Therapy consumption (value & volume) by key regions/countries, product type and application, history data from 2014 to 2018, and forecast to 2024.
- To understand the structure of Hemophilia Gene Therapy market by identifying its various subsegments.
- Focuses on the key global Hemophilia Gene Therapy manufacturers, to define, describe and analyze the sales volume, value, market share, market competition landscape, SWOT analysis and development plans in next few years.
- To analyze the Hemophilia Gene Therapy with respect to individual growth trends, future prospects, and their contribution to the total market.
- To share detailed information about the key factors influencing the growth of the market (growth potential, opportunities, drivers, industry-specific challenges and risks).
- To project the consumption of Hemophilia Gene Therapy submarkets, with respect to key regions (along with their respective key countries).
- To analyze competitive developments such as expansions, agreements, new product launches, and acquisitions in the market.
- To strategically profile the key players and comprehensively analyze their growth strategies.
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