
Beta-thalassemia- Pipeline Insight, 2025
Description
DelveInsight’s, “Beta-thalassaemia- Pipeline Insight, 2025” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in Beta-thalassaemia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Beta-thalassaemia: Overview
Beta thalassemia is an inherited blood disorder that limits your body’s ability to make beta-globin. Beta-globin is an important protein needed to make hemoglobin and red blood cells. Beta thalassemia can cause the patient to experience anemia symptoms.
There are several types of thalassemia. The types include beta thalassemia major, beta thalassemia intermedia and beta thalassemia minor. The signs and symptoms you have depend on the type and severity of your condition. Thalassemia signs and symptoms may include fatigue, weakness, pale or yellowish skin, facial bone deformities, slow growth, abdominal swelling, and dark urine.
Thalassemia is caused by mutations in the DNA of cells that make hemoglobin — the substance in red blood cells that carries oxygen throughout your body. The mutations associated with thalassemia are passed from parents to children. Hemoglobin molecules are made of chains called alpha and beta chains that can be affected by mutations. In thalassemia, the production of either the alpha or beta chains are reduced, resulting in either alpha-thalassemia or beta-thalassemia. In alpha-thalassemia, the severity of thalassemia you have depends on the number of gene mutations you inherit from your parents. The more mutated genes, the more severe your thalassemia and in beta-thalassemia, the severity of thalassemia you have depends on which part of the hemoglobin molecule is affected.
Beta thalassemia is often diagnosed in childhood. Beta thalassemia major, the most severe form, is diagnosed early in childhood, by age 2. The Healthcare professional diagnose beta thalassemia based on your symptoms and blood test results. The diagnosis may include the following tests.
Report Highlights
This segment of the Beta-thalassaemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Beta-thalassaemia Emerging Drugs
Vamifeport is currently in phase II development for beta-thalassemia, an inherited rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body and potentially cause anaemia.
Further product details are provided in the report……..
Beta-thalassaemia: Therapeutic Assessment
This segment of the report provides insights about the different Beta-thalassaemia drugs segregated based on following parameters that define the scope of the report, such as:
Beta-thalassaemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Beta-thalassaemia therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Beta-thalassaemia drugs.
Beta-thalassaemia Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Beta-thalassaemia: Overview
Beta thalassemia is an inherited blood disorder that limits your body’s ability to make beta-globin. Beta-globin is an important protein needed to make hemoglobin and red blood cells. Beta thalassemia can cause the patient to experience anemia symptoms.
There are several types of thalassemia. The types include beta thalassemia major, beta thalassemia intermedia and beta thalassemia minor. The signs and symptoms you have depend on the type and severity of your condition. Thalassemia signs and symptoms may include fatigue, weakness, pale or yellowish skin, facial bone deformities, slow growth, abdominal swelling, and dark urine.
Thalassemia is caused by mutations in the DNA of cells that make hemoglobin — the substance in red blood cells that carries oxygen throughout your body. The mutations associated with thalassemia are passed from parents to children. Hemoglobin molecules are made of chains called alpha and beta chains that can be affected by mutations. In thalassemia, the production of either the alpha or beta chains are reduced, resulting in either alpha-thalassemia or beta-thalassemia. In alpha-thalassemia, the severity of thalassemia you have depends on the number of gene mutations you inherit from your parents. The more mutated genes, the more severe your thalassemia and in beta-thalassemia, the severity of thalassemia you have depends on which part of the hemoglobin molecule is affected.
Beta thalassemia is often diagnosed in childhood. Beta thalassemia major, the most severe form, is diagnosed early in childhood, by age 2. The Healthcare professional diagnose beta thalassemia based on your symptoms and blood test results. The diagnosis may include the following tests.
- A complete blood count (CBC): A CBC provides information about blood cells, including red blood cells. It can show whether the red blood cells are less than normal count, oddly shaped or pale (light red). These characteristics may be signs of thalassemia.
- Reticulocyte count: Immature red blood cells are called reticulocytes. A low reticulocyte count indicates that the body isn’t producing enough red blood cells. Severe thalassemia is usually accompanied by an elevated reticulocyte count. This is because the body is trying to make more red cells to make up for the destruction of the red cells containing the abnormal hemoglobin.
- Molecular genetic testing: Molecular genetic testing allows doctors to study hemoglobin closely and identify the mutation associated with beta thalassemia.
- Hemoglobin electrophoresis: A hemoglobin electrophoresis test measures different types of hemoglobin proteins in blood. Certain types of hemoglobin proteins are increased with beta thalassemia, while other types are decreased.
- Blood transfusions: Frequent blood transfusions (as much as every two weeks) with beta thalassemia major. During the procedure, blood is received from a donor. The influx of blood from a transfusion supplies red blood cells needed to carry oxygen to tissues throughout your body.
- Iron chelation therapy: Iron is an important part of the hemoglobin protein allowing it to carry oxygen. Too much iron, however, can be harmful. Iron chelation therapy can help prevent iron overload.
- Folic acid supplements: Folic acid can help boost your body’s ability to make red blood cells. Doctor may recommend supplements if the patient has beta thalassemia minor or folic acid in addition to receiving regular blood transfusions if the condition is more severe.
- Luspatercept: In case of severe thalassemia, an injection (shot) of luspatercept is received every three weeks to help the body make more red blood cells. Luspatercept improves anemia in people diagnosed with beta thalassemia who are receiving blood transfusions.
- Bone marrow and stem cell transplant: Replacement of bone marrow stem cells with the stem cells of a healthy donor can cure beta thalassemia. Unfortunately, finding a compatible donor can be challenging. Also, this type of transplant is considered a high-risk procedure.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Beta-thalassaemia R&D. The therapies under development are focused on novel approaches to treat/improve Beta-thalassaemia.
This segment of the Beta-thalassaemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Beta-thalassaemia Emerging Drugs
- CTX001: CRISPR Therapeutics
- VIT-2763: CSL Vifor
Vamifeport is currently in phase II development for beta-thalassemia, an inherited rare blood disorder that reduces the production of functional haemoglobin in red blood cells, which can lead to a lack of oxygen in many parts of the body and potentially cause anaemia.
- Emeramide: EmeraMed
Further product details are provided in the report……..
Beta-thalassaemia: Therapeutic Assessment
This segment of the report provides insights about the different Beta-thalassaemia drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Beta-thalassaemia
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Intravenous
- Subcutaneous
- Oral
- Intramuscular
- Molecule Type
- Monoclonal antibody
- Small molecule
- Peptide
- Product Type
Beta-thalassaemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Beta-thalassaemia therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Beta-thalassaemia drugs.
Beta-thalassaemia Report Insights
- Beta-thalassaemia Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Beta-thalassaemia drugs?
- How many Beta-thalassaemia drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Beta-thalassaemia?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Beta-thalassaemia therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Beta-thalassaemia and their status?
- What are the key designations that have been granted to the emerging drugs?
- CRISPR Therapeutics
- CSL Vifor
- Beam Therapeutics
- EmeraMed
- Fulcrum Therapeutics
- Editas Medicine
- EdiGene Inc
- Silence Therapeutics
- Phoenicia Biosciences
- Shanghai BDgene
- Disc Medicine
- Exa-cel
- VIT-2763
- BEAM 101
- Emeramide
- FTX 6058
- EDIT 301
- ET-01
- SLN124
- PB-04
- BD 211
- DISC a
Key Players
Table of Contents
80 Pages
- Introduction
- Executive Summary
- Beta-thalassaemia: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Beta-thalassaemia– DelveInsight’s Analytical Perspective
- Late Stage Products (Preregistration)
- Comparative Analysis
- Exa-cel : CRISPR Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- VIT-2763: CSL Vifor
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I/II)
- Comparative Analysis
- EDIT 301: Editas Medicine
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- ET-01: EdiGene Inc
- Product Description
- Research and Development
- Product Development Activities
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Beta-thalassaemia Key Companies
- Beta-thalassaemia Key Products
- Beta-thalassaemia- Unmet Needs
- Beta-thalassaemia- Market Drivers and Barriers
- Beta-thalassaemia- Future Perspectives and Conclusion
- Beta-thalassaemia Analyst Views
- Beta-thalassaemia Key Companies
- Appendix
Pricing
Currency Rates
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