Thalassemia- Pipeline Insight, 2025
Description
DelveInsight’s, “Thalassemia- Pipeline Insight, 2025,”report provides comprehensive insights about 30+ companies and 30+ pipeline drugs in Thalassemia 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
Thalassemia: Overview
Thalassemia is a group of inherited blood disorders that affect the body’s ability to produce hemoglobin and red blood cells. Thalassemia is a blood disorder that is inherited. This means it is passed down from one or both parents through their genes. When a person has thalassemia, the body makes less hemoglobin than normal. Hemoglobin is an iron-rich protein in red blood cells. It carries oxygen to all parts of the body. There are 2 main types of thalassemia: alpha and beta. Different genes are affected for each type. Thalassemia can cause mild or severe anemia. Anemia occurs when the body does not have enough red blood cells or hemoglobin. The severity and type of anemia depends on how many genes are affected.
Alpha thalassemia occurs when some or all of the 4 genes that make hemoglobin (the alpha-globin genes) are missing or damaged. There are 4 types of alpha thalassemia: Alpha thalassemia silent carrier: One gene is missing or damaged, and the other 3 are normal. Alpha thalassemia carrier: Two genes are missing. Hemoglobin H disease: Three genes are missing. This leaves just 1 working gene. Alpha thalassemia major. All 4 genes are missing. This causes severe anemia. In most cases, a baby with this condition will die before birth. This is a genetic disease inherited from one or both parents.
The only risk factor is having a family history of the disease. Beta thalassemia is caused by damaged or missing genes. Two specific genes are involved. There are several types of this disorder: Beta thalassemia major (Cooley’s anemia). There are two damaged genes. This is the most severe form of this disorder. Beta thalassemia minor or thalassemia trait. Only one gene is damaged. This causes less severe anemia. People with this type have a 50% chance of passing the gene to their children. If the other parent is not affected, their children will also have this form of the disorder. Many people with this disorder are given iron replacement by mistake. This happens when a lack of iron is believed to cause their anemia. Too much iron can be harmful. So it is important to get the right diagnosis.
Thalassemia Symptoms include: Slow growth in children, wide or brittle bones, enlarged spleen (an organ in your abdomen that filters blood and fights disease), fatigue, weakness, Pale or yellow skin, dark urine and poor appetite. The following tests may help to tell if the person is a carrier, and can pass the disorder on to children: Complete blood count (CBC). This test checks the size, number, and maturity of different blood cells in a set volume of blood, Hemoglobin electrophoresis with A2 and F quantitation. A lab test that tells what type of hemoglobin is present, FEP (free-erythrocyte protoporphyrin) and ferritin. This test is done to rule out iron-deficiency anemia. All of these tests can be done using a single blood sample. In a pregnant woman, the baby is diagnosed using CVS (chorionic villus sampling) or amniocentesis. A DNA test is needed to make a diagnosis of alpha thalassemia. There is no cure. Treatment can reduce symptoms.
""Thalassemia- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Thalassemia pipeline landscape is provided which includes the disease overview and Thalassemia treatment guidelines. The assessment part of the report embraces, in depth Thalassemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Thalassemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Thalassemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.
Thalassemia Emerging Drugs
HBB gene stimulants (Hemoglobin beta chain gene stimulants) mechanism of action that targets
β-globin (Hemoglobin beta chain) developed for the treatment of patients suffering with thalassemia. On August 16, 2022, the clinical trial application (IND) of BRL Medicine's BRL-101 was officially approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, entering the registration clinical stage. The results of the IIT and IND Phase 1 clinical studies of BRL-101 indicated a significant increase in overall Hb and HbF levels after all patients received gene-edited HSC transplantation. Throughout the treatment process, most adverse events were consistent with known adverse events for hematopoietic stem cell mobilization/apheresis, busulfan myeloablative conditioning, and autologous hematopoietic stem cell transplantation. Currently it is in Phase I/II stage of development for the treatment of Thalassemia.
Further product details are provided in the report……..
Thalassemia: Therapeutic Assessment
This segment of the report provides insights about the different Thalassemia drugs segregated based on following parameters that define the scope of the report, such as:
Thalassemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III II, I, preclinical and discovery stage. It also analyses Thalassemia 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 Thalassemia drugs.
Thalassemia Report Insights
Current Treatment Scenario and Emerging Therapies:
Geography Covered
- Global coverage
Thalassemia: Overview
Thalassemia is a group of inherited blood disorders that affect the body’s ability to produce hemoglobin and red blood cells. Thalassemia is a blood disorder that is inherited. This means it is passed down from one or both parents through their genes. When a person has thalassemia, the body makes less hemoglobin than normal. Hemoglobin is an iron-rich protein in red blood cells. It carries oxygen to all parts of the body. There are 2 main types of thalassemia: alpha and beta. Different genes are affected for each type. Thalassemia can cause mild or severe anemia. Anemia occurs when the body does not have enough red blood cells or hemoglobin. The severity and type of anemia depends on how many genes are affected.
Alpha thalassemia occurs when some or all of the 4 genes that make hemoglobin (the alpha-globin genes) are missing or damaged. There are 4 types of alpha thalassemia: Alpha thalassemia silent carrier: One gene is missing or damaged, and the other 3 are normal. Alpha thalassemia carrier: Two genes are missing. Hemoglobin H disease: Three genes are missing. This leaves just 1 working gene. Alpha thalassemia major. All 4 genes are missing. This causes severe anemia. In most cases, a baby with this condition will die before birth. This is a genetic disease inherited from one or both parents.
The only risk factor is having a family history of the disease. Beta thalassemia is caused by damaged or missing genes. Two specific genes are involved. There are several types of this disorder: Beta thalassemia major (Cooley’s anemia). There are two damaged genes. This is the most severe form of this disorder. Beta thalassemia minor or thalassemia trait. Only one gene is damaged. This causes less severe anemia. People with this type have a 50% chance of passing the gene to their children. If the other parent is not affected, their children will also have this form of the disorder. Many people with this disorder are given iron replacement by mistake. This happens when a lack of iron is believed to cause their anemia. Too much iron can be harmful. So it is important to get the right diagnosis.
Thalassemia Symptoms include: Slow growth in children, wide or brittle bones, enlarged spleen (an organ in your abdomen that filters blood and fights disease), fatigue, weakness, Pale or yellow skin, dark urine and poor appetite. The following tests may help to tell if the person is a carrier, and can pass the disorder on to children: Complete blood count (CBC). This test checks the size, number, and maturity of different blood cells in a set volume of blood, Hemoglobin electrophoresis with A2 and F quantitation. A lab test that tells what type of hemoglobin is present, FEP (free-erythrocyte protoporphyrin) and ferritin. This test is done to rule out iron-deficiency anemia. All of these tests can be done using a single blood sample. In a pregnant woman, the baby is diagnosed using CVS (chorionic villus sampling) or amniocentesis. A DNA test is needed to make a diagnosis of alpha thalassemia. There is no cure. Treatment can reduce symptoms.
""Thalassemia- Pipeline Insight, 2025"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Thalassemia pipeline landscape is provided which includes the disease overview and Thalassemia treatment guidelines. The assessment part of the report embraces, in depth Thalassemia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Thalassemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Thalassemia R&D. The therapies under development are focused on novel approaches to treat/improve Thalassemia.
This segment of the Thalassemia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.
Thalassemia Emerging Drugs
- Mitapivat: Agios Pharmaceuticals, Inc
- Vamifeport: CSL Vifor
- BRL-101: BRL MEDICINE
HBB gene stimulants (Hemoglobin beta chain gene stimulants) mechanism of action that targets
β-globin (Hemoglobin beta chain) developed for the treatment of patients suffering with thalassemia. On August 16, 2022, the clinical trial application (IND) of BRL Medicine's BRL-101 was officially approved by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration, entering the registration clinical stage. The results of the IIT and IND Phase 1 clinical studies of BRL-101 indicated a significant increase in overall Hb and HbF levels after all patients received gene-edited HSC transplantation. Throughout the treatment process, most adverse events were consistent with known adverse events for hematopoietic stem cell mobilization/apheresis, busulfan myeloablative conditioning, and autologous hematopoietic stem cell transplantation. Currently it is in Phase I/II stage of development for the treatment of Thalassemia.
Further product details are provided in the report……..
Thalassemia: Therapeutic Assessment
This segment of the report provides insights about the different Thalassemia drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Thalassemia
- Phases
- Late stage products (Phase II)
- 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
- Oral
- Parenteral
- intravenous
- Subcutaneous
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Thalassemia: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III II, I, preclinical and discovery stage. It also analyses Thalassemia 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 Thalassemia drugs.
Thalassemia Report Insights
- Thalassemia 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 Thalassemia drugs?
- How many Thalassemia drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Thalassemia?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Thalassemia 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 Thalassemia and their status?
- What are the key designations that have been granted to the emerging drugs?
- Agios Pharmaceuticals, Inc
- Vifor Pharma Group
- BRL MEDICINE
- Sangamo Therapeutics
- Vifor (International) Inc
- Celgene
- Pharmacosmos A/S
- Bioray Laboratories
- DisperSol Technologies, LLC
- CorrectSequence Therapeutics
- Editas Medicine
- Mitapivat
- Vamifeport
- BRL-101
- ST-400
- VIT-2763
- Luspatercept
- SP-420
- BRL-101
- DST-0509
- CS-101
- Renizgamglogene autogedtemcel
Table of Contents
80 Pages
- Introduction
- Executive Summary
- Thalassemia: 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
- Late Stage Products (Phase III)
- Comparative Analysis
- Mitapivat: Agios Pharmaceuticals, Inc
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- VIT-2763: Vifor Pharma Group
- Product Description
- Research and Development
- Product Development Activities
- Early Stage Products (Phase I/II)
- Comparative Analysis
- BRL-101: BRL MEDICINE
- Product Description
- Research and Development
- Product Development Activities
- Inactive Products
- Comparative Analysis
- Thalassemia Key Companies
- Thalassemia Key Products
- Thalassemia- Unmet Needs
- Thalassemia- Market Drivers and Barriers
- Thalassemia- Future Perspectives and Conclusion
- Thalassemia Analyst Views
- Thalassemia Key Companies
- Appendix
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