Hemophilia gene therapy market analysis | Coherent Market Insights
Title: Hemophilia gene therapy market analysis | Coherent Market Insights
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- HEMOPHILIA GENE THERAPY MARKET ANALYSIS
- 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) - Size,
Share, Trends, and Forecast to 2026 - 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.
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- 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 patients body.
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- Figure 1. Global Hemophilia Gene Therapy Market
Value (US Mn), by Region, 2017
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- 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. - Gene therapy may prove to be panacea in this
scenario of increasing prevalence rate of
hemophilia worldwide.
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- 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
Pharmaceuticals gene therapy product
Valoctocogene roxaparvovec for hemophilia A is in
phase III of clinical development in 2019.
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- 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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