The Arena of Gene Therapy is determined to outline a path to the clinic and the market. In the gene therapy market, over 2000 human gene therapy clinical trials have been reported globally, and about 20 gene therapy products are approved to treat various disorders. Hence, the demand for gene therapy to treat blood disorders is increasing. Gene therapy accounts great to cure blood disorders such as sickle cell disease and hemophilia, which have inadequate treatment choices. The gene therapy helps to understand the detailed pathomechanisms of conditions as well as the advancement of specific and efficient gene targeting and delivery tools that are transforming the global market. The approval of gene therapy for the treatment of β-thalassemia in Europe is propelling the research activities for the approval of the drug in different regions and new drug development. The various global biopharmaceutical companies are strategically taking initiatives to develop advanced therapy such as gene therapy for severe blood disorders. This is expected to boost market growth significantly in the coming years.
Globally around 35 companies are working on regenerative medicine therapies for hereditary blood disorders, counting 18 clinical-stage companies. Additionally, the presence of strong pipeline drugs for the treatment of blood disorders with gene therapy is estimated to drive the growth of the market in the coming years. The global Gene Therapy for Blood Disorders market size is expected to reach US$ xx million by 2028, growing at a CAGR of xx% over the forecast period. Additionally, growing strategic partnership, acquisitions among the key players operating in the market is expected to propel the market growth significantly. However, the high cost of treatment is the major factor expected to restrain the growth of gene therapy in the blood disorders market. However, increasing demand for personalized medicine and growing healthcare expenditure globally is estimated to drive the market growth substantially during the estimated time frame.
The Global Gene Therapy for Blood Disorders market is segmented on the basis of indication, mechanism of action, and region. Based on the indication, the market is divided into Sickle Cell Anemia, β-thalassemia, Hemophilia B, and Hemophilia A.
Based on the mechanism of action, the market is segmented into gene therapy and gene editing. Based on the region, the market is studied across North America, Asia-Pacific, Europe, and LAMEA. Among them, North America accounted for the largest share of the market, followed by Europe. The well-established healthcare system in the U.S. and the presence of a large number of companies operating in the market are propelling the growth of the U.S. gene therapy for blood disorders market.
The key players of this market include Biomarin Therapeutics, Spark Therapeutics, Shire, bluebird bio, Inc., CRISPR Therapeutics, Medgenics, OrphageniX, Freeline Therapeutics, Errant Gene Therapeutics, Sangamo Therapeutics, Sigilon Therapeutics, Inc., uniQure, Angiocrine Bioscience, and Sanofi Genzyme among others. The growing collaboration among various research centers and companies to develop a gene therapy to treat severe blood disorders. Gene therapy has enormous potential for blood disorders such as hemophilia and is becoming a large market. Some corporations, such as CRISPR Therapeutics, are revolving to the gene-editing technique CRISPR-Cas9 as a tool for fighting blood disorders. The company is conducting a phase I/II trial for its CRISPR tool to cure sickle cell disease in partnership with the German company Vertex Pharmaceuticals.