Hemophilia Gene Therapy Market Size Projected to be Resilient by 2026

Hemophilia Gene Therapy Market Size,
Share, Trend, Outlook, Future Growth
Analysis And Synthesis 2018 - 2026
Hemophilia is a genetic bleeding disorder in which an individual lacks or has low levels of proteins
called clotting factors. There are around 13 types of clotting factors that work with blood platelets,
which are necessary for clotting process to initiate. There are three forms of hemophilia - A, B, and C.
Hemophilia A is the most common form and is caused due to deficiency in clotting factor VIII.
Hemophilia B occurs due to deficiency of clotting factor IX and Hemophilia C is caused due to clotting
factor XI deficiency. Hemophilia is incurable with current therapeutic options, which only reduces
symptoms such as spontaneous bleeding in muscles and joints as well as increased risk for intracranial
hemorrhage. These treatment options lasts only for a day and is costly. As against conventional clotting
factor replacement therapy, gene therapy is expected to offer sustainable cure for hemophilia by
correcting defective gene sequence (F8 or F9 gene) that codes for clotting factor VIII or IX in the
patient’s body.
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The global hemophilia gene therapy market was valued at US$ 0.00 million in 2017 and is expected to
witness a CAGR of 56.0% over the forecast period (2018 – 2026).
Increasing hemophilia patient base worldwide especially in developed countries is expected to drive
growth of the hemophilia gene therapy market
High prevalence of hemophilia is increasing demand for sustainable treatment of the disease, which is
projected to drive the market growth over the forecast period. According to the National Hemophilia
Foundation factsheet 2018, hemophilia affected over 400,000 individuals worldwide. Moreover, according
to same source, an estimated 75% of patients suffering from hemophilia have no access to treatment or
receive inadequate treatment. According to the Centers for Disease Control and Prevention (CDC) report,
Hemophilia A occurs in 1 out of 5,000 live male births. It is around four times more common than
Hemophilia B. Few effective treatments available for hemophilia require lifetime infusion of expensive
drugs manufactured through recombinant biotechnology or from human plasma.
Strong gene-based product pipeline for hemophilia treatment is expected to drive the hemophilia gene
therapy market growth
Gene therapy for hemophilia has strong product pipeline, which is expected to enter the market in the
near future. This is expected to drive the market growth, as it would result in availability of new
treatment options for management of hemophilia. By Januarys 2019, over 15 investigational gene
therapy are under the clinical development stage. In December 2018, Spark Therapeutics announced
updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in
hemophilia A.
Current therapies for treating hemophilia are only symptomatic management of the disease by
administering clotting factors XIII or IX. Furthermore, there are serious adverse effects associated with
plasma derived products, and limited access to treatment. Gene therapy may address these issues to
certain level. BioMarin Pharmaceutical’s gene therapy product Valoctocogene roxaparvovec for
hemophilia A is in phase III of clinical development in 2019.
Regulatory bodies such as the U.S. Food and Drug Administration (FDA) are supporting the
product development for gene therapy by allotting breakthrough designation status to
investigational gene therapy. For instance, in 2017, the U.S. FDA granted breakthrough therapy
designation to valoctocogene roxaparvovec, which is an Adeno -associated virus 5 factor VIII
vector designed to restore factor VIII plasma concentrations in patients with hemophilia A.
Report includes chapters which deeply display the following deliverable about industry :
• Hemophilia Gene Therapy Market Research Objective and Assumption
• Hemophilia Gene Therapy Market Purview - Report Description, Executive Summary, and
Coherent Opportunity Map (COM)
• Hemophilia Gene Therapy Market Dynamics, Regulations, and Trends Analysis - Market
Dynamics, Regulatory Scenario, Industry Trend, Merger and Acquisitions, New system
Launch/Approvals, Value Chain Analysis, Porter’s Analysis, and PEST Analysis
• Global Hemophilia Gene Therapy Market, By Regions
• Hemophilia Gene Therapy Market Competition by Manufacturers including Production, Share,
Revenue, Average Price, Manufacturing Base Distribution, Sales Area and Product Type.
• Hemophilia Gene Therapy Market Manufacturers Profiles/Analysis including Company Basic
Information, Manufacturing Base and Its Competitors.
• Hemophilia Gene Therapy Market Manufacturing Cost Analysis including Key Raw Materials
and Key Suppliers of Raw Materials.
• Industrial Chain, Sourcing Strategy and Downstream Buyers including Upstream Raw Materials
Sourcing and Downstream Buyers
• Marketing Strategy Analysis, Distributors/Traders including Marketing Channel, Market
Positioning and Distributors/Traders List.
• Market Effect Factors Analysis including Technology Progress/Risk, Consumer Needs/Customer
Preference Change and Economic/Political Environmental Change.
• Hemophilia Gene Therapy Market Forecast including Production, Consumption, Import and
Export Forecast by Type, Applications and Region.
• Research Findings and Conclusion
Key players operating in the global hemophilia gene therapy market include BioMarin Pharmaceuticals,
Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire PLC Sangamo
Therapeutics, Inc., and Freeline Therapeutics.
Detailed Segmentation:
Global Hemophilia Gene Therapy Market, By Type:
•
Hemophilia A
•
Hemophilia B
Global Hemophilia Gene Therapy Market, By Region:
•
North America
•
Latin America
•
Europe
•
Asia Pacific
•
Middle East
•
Africa
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The global hemophilia gene therapy market was valued at US$ 0.00 million in 2017 and is expected to witness a CAGR of 56.0% over 2018 – 2026.