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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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.
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.
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).
Market
Dynamics
Major
factors that are driving growth of the hemophilia gene therapy market
include increasing prevalence of hemophilia, issue of high cost,
access to current hemophilia treatment, which includes coagulation
factor VIII and IX, and increasing number of companies engaged in
research and development of gene therapy products. According to the
Centers for Disease Control and Prevention, hemophilia A is a rare,
X-linked bleeding disorder that affects around 1 in 5,000 to 10,000
live-born males. Hemophilia B is less common than hemophilia A, with
an incidence of around 1 in 25,000 births. According to 2016 global
survey by the World Federation of Hemophilia, the total number of
individuals with hemophilia worldwide was 184,723, including 16,949
within the U.S.
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.
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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.
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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