Beta Thalassemia (B Thal) Market
Thalassemia is a term for a group of disorders in which there is reduced levels of hemoglobin, decreased red blood cell production and anemia. There are two main forms – alpha thalassemia and beta thalassemia, each with various subtypes. Beta thalassemia minor, also known as beta thalassemia trait, is a common condition. The symptoms and severity of beta thalassemia varies greatly from one person to another. Individuals with beta thalassemia minor do not develop symptoms of the disorder but may have a mild anemia. Several individuals with beta thalassemia minor go through life never knowing they carry an altered gene for the disorder. A beta thalassemia major diagnosis is usually made during the first two years of life and individuals require regular blood transfusions and lifelong medical care to survive. When the disorder develops later during life, a diagnosis of beta thalassemia intermedia is given; individuals may only require blood transfusions on rare, specific instances. Most beta thalassemia cases are caused by a mutation in the HBB gene. In extremely rare cases, a loss of genetic material (deletion) that includes the HBB gene causes the disorder. Genes provide instructions for creating proteins that play a critical role in many body functions. When a gene mutation occurs, the protein product may be faulty, inefficient, or absent. Depending upon the functions of the particular protein, this can affect several organ systems of the body. Individuals with beta thalassemia minor have a mutation in one HBB gene and are carriers for the disorder. Individuals with beta thalassemia intermedia or major have mutations in both HBB genes.
Market Dynamics
The increasing incidence of beta thalassemia, rising research and development activities for the development of novel therapeutics for the treatment of beta thalassemia, and increasing adoption of inorganic growth strategies such as acquisitions and collaborations by key players operating in the market, are the major factors that are expected to drive growth of the global beta-thalassemia (B-thal) market over the forecast period.
For instance, in April 2018, GSK, a global pharmaceutical company and Orchard Therapeutics, a pharmaceutical company, entered into a strategic agreement under which GSK will transfer its portfolio of approved and investigational rare disease gene therapies to Orchard. This acquisition strengthens Orchard’s position as a global leader in gene therapy for rare diseases. GSK will continue to invest in the development of its platform capabilities in cell and gene therapies, with a focus on oncology.
Moreover, the portfolio of gene therapy programs Orchard had acquired includes, Strimvelis, the first autologous ex vivo gene therapy for children with adenosine deaminase severe combined immunodeficiency (ADA-SCID), approved by the EMA in 2016, two late-stage clinical programs in ongoing registration studies for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical program for beta thalassemia.
Key features of the study
Market Segmentation
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