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Hemophilia gene therapy market analysis | Coherent Market Insights


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Title: Hemophilia gene therapy market analysis | Coherent Market Insights

Coherent Marketing Insights
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  • 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

  • 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.

  • Figure 1. Global Hemophilia Gene Therapy Market
    Value (US Mn), by Region, 2017

  • Increasing hemophilia patient base worldwide
    especially in developed countries is expected to
    drive growth of the hemophilia gene therapy
  • 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.

  • 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.

  • 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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