Beta-thalassemia (B-thal) Market, by Treatment (Iron Chelating Drugs, Erythroid Maturation Agents, Stem Cell Therapy, and Others), by Disease Indication (Beta Thalassemia Minor, Beta Thalassemia Intermedia, and Beta Thalassemia Major), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Size, Share, Outlook, and Opportunity Analysis, 2022 - 2028
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:
This report provides in-depth analysis of the global beta-thalassemia (B-thal) market, and provides market size (US$ Mn) and compound annual growth rate (CAGR%) for the forecast period (2022-2028), 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 beta-thalassemia (B-thal) market based on the following parameters – company overview, financial performance, product portfolio, market presence, distribution strategies, key developments, strategies, and future plans
Key companies covered as a part of this study include Celgene Corp., BlueBird Bio, Protagonist Therapeutics, Agios Pharmaceuticals, Ionis Pharmaceuticals, Vifor Pharma, Orchard Therapeutics, Sangamo Therapeutics, CRISPR Therapeutics, DisperSol Technologies, Kiadis Pharma, Incyte Corporation, Editas Medicine, Global Blood Therapeutics, and Syros Pharmaceuticals
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 beta-thalassemia (B-thal) 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 beta-thalassemia (B-thal) market
Detailed Segmentation:
Global Beta-thalassemia (B-thal) Market, By Treatment:
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Global Polycythemia Vera Therapeutics Market, By Disease Indication:
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
Global Polycythemia Vera Therapeutics Market, By Region:
North America
By Treatment
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Disease Indication
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
By Country
U.S.
Canada
Latin America
By Treatment
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Disease Indication
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
By Country
Brazil
Mexico
Argentina
Rest of Latin America
Europe
By Treatment
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Disease Indication
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
By Country
Germany
U.K.
France
Italy
Spain
Russia
Rest of Europe
Asia Pacific
By Treatment
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Disease Indication
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
By Country
China
India
Japan
Australia
South Korea
ASEAN
Rest of Asia Pacific
Middle East
By Treatment
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Disease Indication
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
By Country
GCC
Israel
Rest of Middle East
Africa
By Treatment
Iron Chelating Drugs
Erythroid Maturation Agents
Stem Cell Therapy
Others
Disease Indication
Beta Thalassemia Minor
Beta Thalassemia Intermedia
Beta Thalassemia Major
By Region/Country
South Africa
Central Africa
North Africa
Company Profiles
Celgene Corp. *
Company Overview
Material Portfolio
Financial Performance
Key Highlights
Market Strategies
BlueBird Bio
Protagonist Therapeutics
Agios Pharmaceuticals
Ionis Pharmaceuticals
Vifor Pharma
Orchard Therapeutics
Sangamo Therapeutics
CRISPR Therapeutics
DisperSol Technologies
Kiadis Pharma
Incyte Corporation
Editas Medicine
Global Blood Therapeutics
Syros Pharmaceuticals
“*” marked represents similar segmentation in other categories in the respective section.
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