HEMOPHILIA GENE THERAPY MARKET ANALYSIS INDUSTRY INSIGHTS, TRENDS, OUTLOOK, AND OPPORTUNITY ANALYSIS, 2020–2027
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REPORT DESCRIPTION Hemophilia Gene Therapy Market - Overview The global Hemophilia Gene Therapy Market, by Hemophilia Type (Hemophilia A and Hemophilia B), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa), was valued at US$ 00.0 million in 2017 and is projected to exhibit a CAGR of 56.0% over the forecast period (2018 – 2026). Hemophilia is one of the prominent genetic disorders that is characterized by the absence of clotting factors of specific type resulting in lack of coagulation after blood loss. This may result in serious health complications. Bleeding may be due to injury to external tissue or spontaneous bleeding in muscles and joints. Existing therapy offers symptomatic approach where clotting factors that are absent are provided externally on regular basis. However, there are many limitations of this approach such as toxicity, short term efficacy, high cost, and accessibility. Gene therapy offers solutions to all these limitations. Most important is the ability of the hemophilia gene therapy to address the root cause of hemophilia, which is caused due to genetic mutations of the gene that codes for the clotting factors VIII (Hemophilia A) and clotting factors IX (Hemophilia B). In gene therapy, specific gene is inserted into viral vectors (rAAV is most commonly used as vectors) and this vector-based gene is injected intravenously into hemophilic patients, which then codes for formation of coagulation factors. Growing patient base for hemophilia Increasing prevalence of hemophilia worldwide is expected to drive growth of the hemophilia gene therapy market.
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REPORT DESCRIPTION Strong product pipeline for hemophilia gene therapy There are numerous pharma and biotech companies that are engaged in developing hemophilia gene therapy. Most of the companies are targeting hemophilia A, which has approximately 80% share in overall hemophilia patients. BioMarin Pharmaceuticals’ investigational gene therapy, Valoctocogene roxaparvovec for treating hemophilia A, is currently in phase III and Sangamo therapeutics’ investigational gene therapy SB – 525 is in phase I/II, which gave positive preliminary data in August 2018. In December 2018, Spark Therapeutics announced updated preliminary data for the ongoing phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A. Shire PLC’s gene therapy SHP654 for hemophilia A is currently in phase I of clinical development. It is expected that the first gene therapy would enter the market by 2022. Overall, as per database of clinicaltrial.gov, there are around 17 investigational gene therapies under development. Although hemophilia gene therapy may be accessible to only limited number of patients initially due to uniqueness of therapy. In 2016, World Federation of Hemophilia stated that 1 in 5,000 people are affected by hemophilia A and 1 in 50,000 are affected by hemophilia B. According to the National Hemophilia Foundation factsheet 2018, over 400,000 individuals were affected by hemophilia worldwide. Browse 16 Market Data Tables and 21 Figures spread through 110 Pages and indepth TOC on “Hemophilia Gene Therapy Market - by Hemophilia Type (Hemophilia A and Hemophilia B) and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa)- Global forecast to 2026” Request a Sample copy of this reports: https://www.coherentmarketinsights.com/insight/request-sample/2480 © Coherent market Insights. All Rights Reserved
REPORT DESCRIPTION Key Takeaways of the Hemophilia Gene Therapy Market: The global hemophilia gene therapy market is expected to exhibit a CAGR of 56.0% over the forecast period, owing to increasing prevalence of hemophilia worldwide, high cost and limitations with the current clotting factor replacement therapy, and high number of investigational gene therapy under clinical development stage Among hemophilia type, hemophilia A segment is expected to hold dominant position in the market due to high prevalence rate amongst population worldwide North America is expected to hold dominant position in the market, followed by Europe and Asia Pacific. The U.S. has high annual cost of hemophilia treatment. Current medication regimen to treat hemophilia costs around US$ 270,000 annually per patient, according to a 2015 Express Scripts report. This cost may be with gene therapy, which is comparatively sustainable. Key players operating in the global hemophilia gene therapy market include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc. There are various steps taken by the U.S. FDA to speed up the clinical development of hemophilia gene therapy. The U.S. FDA has allotted breakthrough therapy designation to several investigational gene therapy. For instance, in 2017, the U.S. Food and Drug Administration granted breakthrough therapy designation to valoctocogene roxaparvovec of BioMarin Pharmaceuticals.
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