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 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.

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