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Hemophilia Gene Therapy - A Pipeline Analysis Report

Hemophilia Gene Therapy - A Pipeline Analysis Report



Hemophilia Gene Therapy: An insight

Hemophilia is an inherited bleeding disorder. The gene responsible for hemophilia is present on the X chromosome and is inherited in a recessive manner. Hemophilia A, also called as factor VIII (FVIII) deficiency or classic hemophilia, is caused by missing or defective factor VIII. Most individuals, roughly 85%, have hemophilia A and produce too little or no factor VIII. On the other hand, hemophilia B, which is also called Christmas disease, is caused by a deficiency of factor IX.

Shire became the biggest hemophilia player after it bought Baxalta in June 2016, and the company reported total hemophilia revenue of $870.9 million in the FY2016.

Covered in this report

The report covers the present scenario and the growth prospects of the hemophilia gene therapy. The report presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources.

Technavio's Hemophilia Gene Therapy - A Pipeline Analysis Report, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects during the forecast period. The report also includes a discussion of the key companies operating in this market.

Major companies

  • uniQure
  • Alnylam Pharmaceuticals
  • BioMarin Pharmaceutical
  • Spark Therapeutics
  • Shire
  • Key questions answered in this report
    • How will the market evolve during the forecast period?
    • What are the major parameters impacting the market?
    • What are the key market trends?
    • What are the challenges to market growth?
    • Who are the key companies in this market space?


    Press Release

    Technavio Announces the Publication of its Research Report - Hemophilia Gene Therapy - A Pipeline Analysis Report

    Technavio recognizes the following companies in the hemophilia gene therapy: uniQure, Alnylam Pharmaceuticals, BioMarin Pharmaceutical, Spark Therapeutics, and Shire.

    Hemophilia is an inherited bleeding disorder. The gene responsible for hemophilia is present on the X chromosome and is inherited in a recessive manner. Hemophilia A, also called as factor VIII (FVIII) deficiency or classic hemophilia, is caused by missing or defective factor VIII. Most individuals, roughly 85%, have hemophilia A and produce too little or no factor VIII. On the other hand, hemophilia B, which is also called Christmas disease, is caused by a deficiency of factor IX. Due to the deficiency of these factors, the blood does not clot properly, and this condition creates complicated health issues.

    Using a gene with the capacity to cure or prevent the progression of a disease is the basic concept of gene therapy technique. Different approaches are being tested by the researchers, including replacing a mutated gene that causes disease with a healthy copy of the gene, inactivating or knocking out a mutated gene that is malfunctioning, and introducing a new gene into the body to help fight a disease.

    The use of gene therapy for the treatment of hemophilia is in many ways a promising option. Hemophilia is an ideal genetic disorder to treat by gene therapy, as it occurs due to a single genetic defect. The drawbacks associated with the majority of the marketed drugs (standard protein substitution therapy) intensified the interest in alternative treatments by gene therapy. However, gene therapy also has some drawbacks such as the risk of developing antibodies to the viral vectors used to deliver the genetic material to the cells in the individuals and inconsistent results in clinical trials with human test remains a matter of concern for the researchers.

    Companies Mentioned

    uniQure, Alnylam Pharmaceuticals, BioMarin Pharmaceutical, Spark Therapeutics, and Shire.

    • Executive summary
    • Scope of the report
    • Research methodology
    • Hemophilia gene therapy: An insight
      • Introduction
    • Major regulatory authorities
      • US
        • Table Drug approval process by US FDA
      • Europe
      • Japan
      • Australia
      • China
    • Pipeline landscape
      • Table Pipeline landscape
      • Table Pipeline molecules by vendors
    • Comparative analysis
      • Table Pipeline molecules in different developmental stages 2017
      • Table Pipeline molecules in different developmental stages (%) 2017
      • Late-stage molecules (Phase III)
        • Table Overview: Fitusiran
        • Table Clinical trials description of Fitusiran
      • Mid-stage molecules (Phase I/II)
        • Table Overview: SB 525
        • Table Clinical trial description of SB-525
        • Table Overview: SB FIX
        • Table Clinical trials description of SB FIX
        • Table Overview: AMT 060
        • Table Clinical trials description of AMT-060 (AAV5-hFIX)
        • Table Overview: BMN 270
        • Table Clinical trials description of BMN 270
        • Table Overview: SPK 9001
        • Table Clinical trials description of SPK 9001
        • Table Overview: SPK-8011
        • Table Clinical trials description of SPK-8011
      • Early-stage molecules (Phase I and IND)
        • Table Overview: Gene therapy for hemophilia B
        • Table Clinical trials description of gene therapy for hemophilia B
        • Table Overview: DTX201
        • Table Overview: SHP654
      • Pre-clinical stage molecules
        • Table Overview: SVFVIIa
        • Table Overview: Ex-vivo stem cell-LV-FVIII gene therapy
        • Table Overview: In-vivo AAV-FIX gene therapy
        • Table Overview: Research program for hemophilia A
        • Table Overview: Gene therapy for hemophilia
        • Table Overview: Factor VIII (rhFVIII)
        • Table Overview: Gene therapy for hemophilia A
        • Table Overview: SHP648
        • Table Overview: HepaStem
      • Discontinued molecules
        • Table Discontinued hemophilia gene therapy pipeline molecules, 2017
    • Indication analysis
      • Table Segmentation of hemophilia gene therapy pipeline molecules by indication 2017
      • Table An overview of hemophilia indication
    • Therapeutic assessment by therapy
      • Therapeutic assessment based on therapy
        • Table Assessment of hemophilia gene therapy pipeline molecules by monotherapy products
    • Therapeutic assessment by RoA
      • Therapeutic assessment based on RoA
        • Table Assessment of hemophilia gene therapy pipeline molecules by RoA
    • Therapeutic assessment by stage and RoA
      • Therapeutic assessment based on RoA in various stages of development
        • Table Assessment of hemophilia gene therapy pipeline molecules by stage and RoA
    • Key companies
      • Active companies: Category and parameters
        • Table Active companies: Category and parameters 2017
        • Table Segmentation of companies 2017
        • Table Overview: Alnylam Pharmaceuticals 2017
        • Table Overview: Amarna Therapeutics 2017
        • Table Overview: BioMarin Pharmaceutical 2017
        • Table Overview: Bioverativ 2017
        • Table Overview: Dimension Therapeutics 2017
        • Table Overview: Expression Therapeutics 2017
        • Table Overview: Freeline Therapeutics 2017
        • Table Overview: Immusoft 2017
        • Table Overview: Pharming Group 2017
        • Table Overview: Promethera 2017
        • Table Overview: Sangamo Therapeutics 2017
        • Table Overview: Shire 2017
        • Table Overview: Spark Therapeutics 2017
        • Table Overview: uniQure 2017
    • Appendix
      • List of abbreviations
    • Explore Technavio

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