Hemophilia Gene Therapy Market Growth Analysis
By
BioMarin
Pharmaceuticals,
Inc.,
Spark
Therapeutics, Pfizer, Inc.
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.
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