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. 

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.

Market Dynamics

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 features of the study:

• This report provides an in-depth analysis of the global hemophilia gene therapy market, and provides market size (US$ Million) and compound annual growth rate (CAGR%) for the forecast period (2022–2030), considering 2021 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the global hemophilia gene therapy market  based on the following parameters – company highlights, products portfolio, key highlights, financial performance, and strategies
• Key companies covered as a part of this study include BioMarin Pharmaceuticals, Inc., Spark Therapeutics, Pfizer, Inc., UniQure NV, Ultragenyx Pharmaceutical, Shire,  Sangamo Therapeutics, Inc., and Freeline Therapeutics.
• Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• The global hemophilia gene therapy market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
• Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global hemophilia gene therapy market

Detailed Segmentation:

• Global Hemophilia Gene Therapy Market, By Hemophilia Type:
  • Hemophilia A
  • Hemophilia B
• Global Hemophilia Gene Therapy Market, By Region:
  • North America
    • By Country
      • U.S.
      • Canada
  • Latin America
    • By Country
      • Brazil
      • Mexico
      • Argentina
      • Rest of Latin America
  • Europe
    • By Country
      • Germany
      • U.K.
      • France
      • Italy
      • Spain
      • Russia
      • Rest of Europe
  • Asia Pacific
    • By Country
      • China
      • India
      • Japan
      • Australia
      • South Korea
      • ASEAN
      • Rest of Asia Pacific
  • Middle East
    • By Country
      • GCC
      • Israel
      • Rest of Middle East
  • Africa
    • By Region
      • South Africa
      • Central Africa
      • North Africa
• Company Profiles
  • BioMarin Pharmaceuticals, Inc. *
    • Company Highlights
    • Products Portfolio
    • Key Highlights
    • Financial Performance
    • Strategies
  • Spark Therapeutics
  • Pfizer, Inc.
  • UniQure NV
  • Ultragenyx Pharmaceutical
  • Shire PLC
  • Sangamo Therapeutics
  • Freeline Therapeutics

“*” marked represents similar segmentation in other categories in the respective section.