Thalassemia - Pipeline Insight, 2025

DelveInsight's, "Thalassemia - Pipeline Insight, 2025" report provides comprehensive insights about 25+ companies and 28+ 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:

• Global coverage

Thalassemia: Understanding

Thalassemia: Overview

Thalassemia is a group of inherited blood disorders characterized by reduced or absent synthesis of either alpha or beta chains of hemoglobin, the protein responsible for carrying oxygen in red blood cells. This imbalance leads to ineffective red blood cell production, chronic anemia, and varying clinical severity depending on the type and number of gene mutations or deletions involved. Alpha thalassemia results from deletions in the alpha-globin genes, while beta thalassemia arises from point mutations in the beta-globin gene. The disease ranges from silent carriers to severe transfusion-dependent forms like beta-thalassemia major. Classification now often includes transfusion-requiring and non-transfusion-requiring types, reflecting the clinical burden more accurately.

Thalassemia presents with a range of symptoms depending on its severity, commonly including fatigue, weakness, pale or yellowish skin, and dark urine due to reduced hemoglobin and increased red blood cell destruction. In more severe forms, patients may experience facial bone deformities, slow growth, jaundice, shortness of breath, and abdominal swelling from liver or spleen enlargement. Complications can extend to heart problems, bone disorders, and, in rare cases, developmental delays, particularly in those with long-standing untreated or poorly managed disease. Regular monitoring and timely treatment, such as blood transfusions and iron chelation therapy, are essential to manage symptoms and prevent complications. Early diagnosis can significantly improve outcomes and quality of life. Genetic counseling is recommended for families with a history of the condition.

Thalassemia is diagnosed through a combination of blood and genetic tests. A complete blood count (CBC) typically reveals low hemoglobin levels and smaller-than-normal red blood cells, while hemoglobin electrophoresis and high-performance liquid chromatography (HPLC) help identify abnormal hemoglobin variants. Iron studies are used to distinguish thalassemia from iron deficiency anemia. Definitive diagnosis is achieved through molecular genetic testing, which detects mutations in globin genes. Prenatal screening methods like chorionic villus sampling (CVS) and amniocentesis can identify thalassemia in fetuses, and newborn screening programs in some regions enable early detection and management.

Treatment and management of thalassemia depend on its type and severity, ranging from minimal intervention in mild cases to intensive therapy in severe forms. Mild thalassemia may require occasional transfusions, while moderate to severe cases often need regular blood transfusions to maintain adequate hemoglobin levels, along with iron chelation therapy to prevent iron overload. Advanced options include stem cell transplantation, gene therapy, and genome editing techniques like CRISPR, offering potential long-term cures in select cases. Supportive measures such as splenectomy, cholecystectomy, appropriate vaccinations, and careful dietary management also play a crucial role in improving quality of life and minimizing complications.

"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.

Thalassemia Emerging Drugs Chapters

This segment of the Thalassemia 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.

Thalassemia Emerging Drugs

• Etavopivat: Novo Nordisk A/S

Etavopivat is an investigational, oral, small-molecule activator of erythrocyte pyruvate kinase (PKR) activator in development for the treatment of sickle cell disease and other haemoglobinopathies10. Etavopivat-mediated activation of PKR lowers levels of 2,3- diphosphoglycerate (2,3-DPG) and raises adenosine triphosphate (ATP) levels, which has the potential to improve oxygen bind to haemoglobin (i.e. increase oxygen affinity), reduce haemolysis and decrease VOCs by improving red blood cell health. Currently, the drug is in Phase III stage of its development for the treatment of Thalassemia.

• AND017: Kind Pharmaceuticals LLC

AND017 is a first-in-class hemoglobin elevating agent (HbEA) that targets multiple stages of the red blood cell (RBC) life cycle and is being developed to treat anemia associated with dialysis-dependent chronic kidney disease (DD-CKD), non-dialysis dependent chronic kidney disease (NDD-CKD), cancer-related anemia (CRA), myelodysplastic syndromes (MDS) anemia, sickle cell disease (SCD), and B-thalassemia. Currently, the drug is in Phase II stage of its development for the treatment of Thalassemia.

• CS-101: CorrectSequence Therapeutics Co., Ltd

The underlying principle of CS-101 involves the collection of the patient's own hematopoietic stem cells, followed by the treatment of the transformer Base Editor (tBE). By generating a naturally occurring beneficial single-nucleotide variant found in individuals with hereditary persistence of fetal hemoglobin, the tBE reactivates Y-globin expression, resulting in the production of functional HbF. The edited stem cells are then reintroduced into the patient's body, enabling continuous production of blood cells with functional hemoglobin. Compared to traditional blood transfusion therapy and allogeneic hematopoietic stem cell transplantation, CS-101 offers several compelling advantages. One of the key benefits is its short preparation period. Another is the elimination of the long waiting time for a matching donor, as it utilizes the patient's own hematopoietic stem cells. CS-101 has the potential to achieve a ""single treatment, lifetime cure"" in just a few weeks. Currently, the drug is in Phase I stage of its development for the treatment of Thalassemia.

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
• There are approx. 25+ key companies which are developing the therapies for Thalassemia. The companies which have their Thalassemia drug candidates in the most advanced stage, i.e. Phase II include, Novo Nordisk A/S.
• Phases

DelveInsight's report covers around 28+ products under different phases of clinical development like

• 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

Thalassemia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

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

Thalassemia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

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?

Key Players

• Novo Nordisk A/S
• Kind Pharmaceuticals LLC
• CorrectSequence Therapeutics Co., Ltd
• Editas Medicine
• San Rocco Therapeutics
• Lantu Biopharma
• Regeneron Pharmaceuticals
• Kanglin Biotechnology (Hangzhou) Co., Ltd.
• Shanghai BDgene Co., Ltd.
• Bioray Laboratories
• Celgene
• Mabwell (Shanghai) Bioscience Co., Ltd.

Key Products

• Etavopivat
• AND017
• CS-101
• In Vivo Gene Edited Medicines
• TNS9
• Vebeglogene autotemcel
• REGN7999
• KL003
• BD211
• BRL-101
• ACE-536
• 9MW3011