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DUBLIN--(BUSINESS WIRE)--Sep 21, 2022--
The "Hemophilia Gene Therapy Market, by Hemophilia Type, and by Region - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2030" report has been added to ResearchAndMarkets.com's offering.
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.
In this therapy, recombinant Adeno - associated virus is most commonly utilized to deliver a codon optimized version of the clotting factors genes (VIII or IX) to patients affected by hemophilia. This gene is shortened by deleting a discrete portion of the gene to better fit the coding sequence into the viral vector. There is dose dependent gene expression level observed which means genetic expression for clotting factor increases with increased dose of gene therapy.
Market Dynamics
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 features of the study:
Detailed Segmentation: Global Hemophilia Gene Therapy Market, By Hemophilia Type:
Global Hemophilia Gene Therapy Market, By Region:
Company Profiles
Key Topics Covered:
1. Research Objectives and Assumptions
2. Market Purview
3. Market Dynamics, Regulations, and Trends Analysis
4. Global Hemophilia Gene Therapy Market - Impact of Coronavirus (Covid-19) Pandemic
5. Global Hemophilia Gene Therapy Market, By Hemophilia Type, 2017 - 2030, (US$ Million)
6. Global Hemophilia Gene Therapy Market, By Region, 2017 - 2030, (US$ Million)
7. Competitive Landscape
8. Section
For more information about this report visit https://www.researchandmarkets.com/r/dodq7x
View source version on businesswire.com:https://www.businesswire.com/news/home/20220921005694/en/
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KEYWORD:
INDUSTRY KEYWORD: GENETICS PHARMACEUTICAL HEALTH
SOURCE: Research and Markets
Copyright Business Wire 2022.
PUB: 09/21/2022 09:27 AM/DISC: 09/21/2022 09:27 AM
http://www.businesswire.com/news/home/20220921005694/en