Coherent Market Insights published a business research report on “Hemophilia Gene Therapy Market: Global Industry Analysis, Size, Share, Growth, Trends, and Forecasts 2021–2027”. Hemophilia Gene Therapy with 100+ market data Tables, Pie Chat, Graphs & Figures spread through Pages and easy to understand detailed analysis. The information is gathered based on modern floats and requests identified with the administrations and items.
Hemophilia Gene Therapy Market report includes historic data, present market trends, environment, technological innovation, upcoming technologies and the technical progress in the related industry. Hemophilia Gene Therapy Market offers complete, proficient report delivering market research data that is relevant for new market entrants or set up players. Key strategies of the companies operating in the market and their impact analysis have been included in the report.
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Hemophilia is a bleeding disorder caused by a lack of or low level of clotting factors in a person’s blood. There are approximately 13 different types of coagulation factors that function with blood platelets and are required for the clotting process to begin. There are three forms of hemophilia – A, B, and C. Hemophilia A is the most common form and is produced 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 existing treatment options, which mainly alleviate symptoms like spontaneous bleeding in muscles and joints and a higher risk of cerebral haemorrhage. These treatments are expensive and only last a day. As against conventional clotting factor replacement therapy, gene therapy is estimated to deal sustainable therapy for hemophilia by improving defective gene sequence (F8 or F9 gene) that codes for clotting factor VIII or IX in the patient’s body.
In this treatment, recombinant Adeno – allied virus is most commonly used to deliver a codon optimized version of the clotting factors genes (VIII or IX) to patients exaggerated by hemophilia. To better fit the coding sequence into the viral vector, this gene is shortened by removing a distinct section of the gene. There is a dosage-dependent gene expression level, which indicates that the genetic expression of clotting factor increases as the dose of gene therapy is raised.
The global hemophilia gene therapy market was valued at US$ 0.00 million in 2017 and is estimated 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
The high prevalence of haemophilia is driving need for long-term therapy, which is expected to fuel market expansion throughout the forecast period. According to the National Hemophilia Foundation factsheet 2018, hemophilia affected over 400,000 people globally. Furthermore, according to the same source, an estimated 75% of haemophilia patients do not have access to treatment or receive poor treatment. Hemophilia A is seen in one out of every 5,000 live male births, according to the Centers for Disease Control and Prevention (CDC). Hemophilia A is almost four times as frequent as Hemophilia B. Few effective haemophilia treatments exist, and most of them involve a lifetime of pricey medications made from recombinant biotechnology or human plasma infusions.
Gene therapy may demonstrate to be panacea in this scenario of growing prevalence rate of hemophilia worldwide.
Strong gene-based product pipeline for hemophilia treatment is expected to drive the hemophilia gene therapy market growth
Hemophilia gene therapy has a solid product pipeline, and it is likely to hit the market soon. This is predicted to fuel market expansion since it will result in the availability of novel haemophilia treatment alternatives over 15 experimental gene therapies were in the clinical development stage as of January 2019. Spark Therapeutics released updated preliminary findings for the ongoing phase 1/2 clinical trial of SPK-8011 in haemophilia A in December 2018.
Currently, the sole treatment for haemophilia is symptomatic management with the use of clotting factors XIII or IX. Furthermore, plasma-derived products are associated with substantial side effects and restricted therapeutic options. Gene therapy may be able to help with these concerns to some extent. Valoctocogene roxaparvovec, a gene treatment for haemophilia A developed by BioMarin Pharmaceuticals, is now in phase III clinical trials.
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.
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.
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Main points in Hemophilia Gene Therapy Market Report Table of Content
Chapter 1 Industry Overview
1.3 Research Scope
1.4 Market Analysis by Regions
1.5 Hemophilia Gene Therapy Market Size Analysis from 2021 to 2027
11.6 COVID-19 Outbreak: Hemophilia Gene Therapy Industry Impact
Chapter 2 Hemophilia Gene Therapy Competition by Types, Applications, and Top Regions and Countries
2.1 Hemophilia Gene Therapy (Volume and Value) by Type
2.3 Hemophilia Gene Therapy (Volume and Value) by Regions
Chapter 3 Production Market Analysis
3.1 Production Market Analysis
3.2 Regional Production Market Analysis
Chapter 4 Hemophilia Gene Therapy Sales, Consumption, Export, Import by Regions (2016-2021)
Chapter 5 North America Hemophilia Gene Therapy Market Analysis
Chapter 6 East Asia Hemophilia Gene Therapy Market Analysis
Chapter 7 Europe Hemophilia Gene Therapy Market Analysis
Chapter 8 South Asia Hemophilia Gene Therapy Market Analysis
Chapter 9 Southeast Asia Hemophilia Gene Therapy Market Analysis
Chapter 10 Middle East Hemophilia Gene Therapy Market Analysis
Chapter 11 Africa Hemophilia Gene Therapy Market Analysis
Chapter 12 Oceania Hemophilia Gene Therapy Market Analysis
Chapter 13 South America Hemophilia Gene Therapy Market Analysis
Chapter 14 Company Profiles and Key Figures in Hemophilia Gene Therapy Business
Chapter 15 Hemophilia Gene Therapy Market Forecast (2021-2027)
Chapter 16 Conclusions
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