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Global Gene Therapy for Blood Disorders Market | BioMarin Pharmaceutical Inc., Spark Therapeutics, Shire , bluebird bio, Inc., CRISPR Therapeutics , Medgenics, OrphageniX, Freeline Therapeutics , Errant Gene Therapeutics, Sangamo Therapeutics , Sigilon Th
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According to the latest research by InsightAce Analytic, the global gene therapy for blood disorders market is expected to growwith a CAGR of 28.4% during a forecast period. By region, North America dominated the market with a share of XX% in 2020.
Gene therapy technique is used to modify a person's genes to treat or cure disease. It has considerable potential to treat rare blood disorders such as sickle cell disease, hemophilia. Several biopharma companies are shifting toward the gene-editing technique, CRISPR-Cas9 as a tool for fighting rare diseases. CRISPR Therapeutics company is conducting a phase I/II trial of its CRISPR/Cas9-based gene-editing therapy, CTX001, to treat sickle cell disease in collaboration with Vertex Pharmaceuticals.
The high prevalence of genetic and blood disorders, increasing demand for personalized medicine, growing healthcare expenditure, rising R&D activities for developing promising gene therapies to treat blood diseases are projected to fuel the market growth in the next few years. According to the study published in the PLOS Medicine Journal, sickle cell anemia cases are estimated to increase about 30% globally by the year 2050. Hence, the rising prevalence of rare hematology disorders is the key driving factor of this market.
However, the high cost of gene therapy for blood disorders and unwanted side effects are expected to hinder the market growth during the forecast period.
Geographically, North America is estimated to account for the highest share of the market in the coming years, followed by Europe owing to the high prevalence of various blood disorders, well-established healthcare systems, and increasing R&D initiatives for developing effective gene therapies to treat rare disorders.
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Major market players operating in the gene therapy for blood disorders market include BioMarin Pharmaceutical Inc. (US), Spark Therapeutics (US), Shire (US), bluebird bio, Inc. (US), CRISPR Therapeutics (US), Medgenics (US), OrphageniX (US), Freeline Therapeutics (UK), Errant Gene Therapeutics (US), Sangamo Therapeutics (US), Sigilon Therapeutics, Inc. (US), uniQure (Netherlands), Angiocrine Bioscience (US), and Sanofi Genzyme (US) among others.
Some of the key developments in the market are as follows:
• In March 2021, the FDA approved the first test of CRISPR to correct genetic defects causing sickle cell disease. This CRISPR-based therapy can directly correct the mutation in the beta-globin gene responsible for sickle cell disease. Beta-globin is the protein in the hemoglobin complex accountable for carrying oxygen throughout the body.
• In December 2020, bluebird bio, Inc. (US) presented the treatment results with investigational LentiGlobin gene therapy for sickle cell disease (bb1111), at the 62nd Annual ASH Meeting. This treatment eliminated SCD-related severe vaso-occlusive events in group C of phase 1/2 HGB-206 clinical study.
• In May 2018, Bioverativ Inc. (US), a Sanofi company working on rare blood disorders, and Sangamo Therapeutics, Inc. announced the U.S. FDA acceptance for Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the sickle cell disease treatment. Bioverativ and Sangamo collaborated to develop and commercialize gene-edited cell therapies for sickle cell disease and beta-thalassemia.
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Market Segments
Global Gene Therapy for Blood Disorders Market, by Indication, 2020-2030 (Value US$ Mn)
• Sickle Cell Anemia
• β-thalassemia
• Hemophilia B
• Hemophilia A
Global Gene Therapy for Blood Disorders Market, by Mechanism of Action, 2020-2030 (Value US$ Mn)
• Gene Therapy
• Gene Editing
Global Gene Therapy for Blood Disorders Market, by Region, 2020-2030 (Value US$ Mn)
• North America
• Europe
• Asia Pacific
• Latin America
• Middle East & Africa
North America Gene Therapy for Blood Disorders Market, by Country, 2020-2030 (Value US$ Mn)
• U.S.
• Canada
Europe Gene Therapy for Blood Disorders Market, by Country, 2020-2030 (Value US$ Mn)
• Germany
• France
• Italy
• Spain
• Russia
• Rest of Europe
Asia Pacific Gene Therapy for Blood Disorders Market, by Country, 2020-2030 (Value US$ Mn)
• India
• China
• Japan
• South Korea
• Australia & New Zealand
Latin America Gene Therapy for Blood Disorders Market, by Country, 2020-2030 (Value US$ Mn)
• Brazil
• Mexico
• Rest of Latin America
Middle East & Africa Gene Therapy for Blood Disorders Market, by Country, 2020-2030 (Value US$ Mn)
• GCC Countries
• South Africa
• Rest of Middle East & Africa
Why should buy this report:
To receive a comprehensive analysis of the prospects for global gene therapy for blood disorders market
To receive an industry overview and future trends in gene therapy for blood disorders market
To analyze the gene therapy for blood disorders market drivers and challenges
To get information on the gene therapy for blood disorders market value (US$ Mn) and forecast to 2030
Major investments, mergers & acquisitions in the gene therapy for blood disorders market industry
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InsightAce Analytic is a market research and consulting firm that enables clients to make strategic decisions. Our qualitative and quantitative market intelligence solutions inform the need for market and competitive intelligence to expand businesses. We help clients gain competitive advantage by identifying untapped markets, exploring new and competing technologies, segmenting potential markets and repositioning products. Our expertise is in providing syndicated and custom market intelligence reports with an in-depth analysis with key market insights in a timely and cost-effective manner.
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