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DelveInsight’s “Beta Thalassemia Pipeline Insight 2025” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in the Beta Thalassemia pipeline landscape. It covers the Beta Thalassemia Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Beta Thalassemia Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Curious about the latest updates in the Beta Thalassemia Pipeline? Click here to explore the therapies and trials making headlines @ Beta Thalassemia Pipeline Outlook Report

Key Takeaways from the Beta Thalassemia Pipeline Report

• On 14 October 2025, Vertex Pharmaceuticals Incorporated conducted a study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
• On 14 October 2025, Regeneron Pharmaceuticals announced a study is researching an experimental drug called REGN7999 (called “study drug”). The study is focused on patients with non-transfusion dependent beta-thalassemia. The aim of the study is to see how safe and effective the study drug is.
• On 02 October 2025, Celgene initiated a study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to
• DelveInsight’s Beta Thalassemia Pipeline report depicts a robust space with 22+ active players working to develop 22+ pipeline therapies for Beta Thalassemia treatment.
• The leading Beta Thalassemia Companies such as CRISPR Therapeutics, CSL Vifor, Beam Therapeutics, EmeraMed, Fulcrum Therapeutics, Editas Medicine, EdiGene Inc, Silence Therapeutics, Phoenicia Biosciences, Shanghai BDgene, Disc Medicine and others.
• Promising Beta Thalassemia Therapies such as Vebeglogene Autotemcel, ACE-536, Luspatercept, HQK-1001, Deferitrin (GT56-252), Desferoxamine (DFO), Bitopertin, ICL670 and others.

Want to know which companies are leading innovation in Beta Thalassemia? Dive into the full pipeline insights @ Beta Thalassemia Clinical Trials Assessment

The Beta Thalassemia Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Beta Thalassemia Pipeline Report also highlights the unmet needs with respect to the Beta Thalassemia.

Beta Thalassemia 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.

Beta Thalassemia Emerging Drugs Profile

• CTX001: CRISPR Therapeutics

CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy in development for patients suffering from β-thalassemia and sickle cell disease. The drug is in Phase I/II clinical evaluation for the treatment of β-thalassemia. CTX001 has been designated an orphan drug in the U.S. and Europe, and given fast track, rare pediatric disease, and regenerative medicine advanced therapy designations in the U.S. for SCD and transfusion-dependent Beta thalassemia (B-thal) (TDT).

• VIT-2763: CSL Vifor

VIT-2763 is an orally administered small molecule developed by Vifor Pharma. Intended for daily administration, VIT-2763 has the potential for treating diseases with impaired iron metabolism. Ferroportin is an iron transporter that plays a key role in regulating iron uptake and distribution in the body and thus in controlling iron levels in the blood. At the molecular level, VIT-2763 binds to ferroportin and blocks it to prevent excessive iron release into the blood. Pre-clinical evidence serving as the basis for the clinical development of VIT-2763 revolves around its efficacy for reducing elevated blood and tissue iron levels and for restricting iron uptake in patients suffering from conditions in which iron metabolism is altered. 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

Emeramide is an antioxidant heavy metal chelator. It prevents methylmercury-induced glutathione (GSH) loss, and cytotoxicity to, isolated mouse aortic endothelial cells when used at a concentration of 50 µM. Emeramide is a lipophilic, di-thiol antioxidant, anti-viral, and metal chelator. Orphan Drug Designations were received for the treatment of mercury toxicity in the EU and US. Pre-clinical safety studies, a Phase I trial, and four Phase II trials have been performed targeting multiple indications. Currently the drug is in phase II for the treatment of beta-thalassaemia.

If you’re tracking ongoing Beta Thalassemia Clinical trials, this press release is a must-read. Tap to see the breakthroughs @ Beta Thalassemia Treatment Drugs

The Beta Thalassemia Pipeline report provides insights into:-

• The report provides detailed insights about companies that are developing therapies for the treatment of Beta Thalassemia with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Beta Thalassemia Treatment.
• Beta Thalassemia Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Beta Thalassemia Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Beta Thalassemia market.

Beta Thalassemia Companies

CRISPR Therapeutics, CSL Vifor, Beam Therapeutics, EmeraMed, Fulcrum Therapeutics, Editas Medicine, EdiGene Inc, Silence Therapeutics, Phoenicia Biosciences, Shanghai BDgene, Disc Medicine and others.

Beta 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,

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Beta Thalassemia Products have been categorized under various Molecule types such as,

• Monoclonal antibody
• Small molecule
• Peptide

From emerging drug candidates to competitive intelligence, the Beta Thalassemia Pipeline Report covers it all – check it out now @ Beta Thalassemia Market Drivers and Barriers, and Future Perspectives

Scope of the Beta Thalassemia Pipeline Report

• Coverage- Global
• Beta Thalassemia Companies- CRISPR Therapeutics, CSL Vifor, Beam Therapeutics, EmeraMed, Fulcrum Therapeutics, Editas Medicine, EdiGene Inc, Silence Therapeutics, Phoenicia Biosciences, Shanghai BDgene, Disc Medicine and others.
• Promising Beta Thalassemia Therapies such as Vebeglogene Autotemcel, ACE-536, Luspatercept, HQK-1001, Deferitrin (GT56-252), Desferoxamine (DFO), Bitopertin, ICL670 and others.
• Beta Thalassemia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Beta Thalassemia Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

Stay ahead in Healthcare Research – discover what’s next for the Beta Thalassemia Treatment landscape in this detailed analysis @ Beta Thalassemia Emerging Drugs and Major Players

Table of Contents

1. Introduction
2. Executive Summary
3. Beta-thalassaemia: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Beta-thalassaemia– DelveInsight’s Analytical Perspective
7. Late Stage Products (Preregistration)
8. Exa-cel: CRISPR Therapeutics
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II)
11. VIT-2763: CSL Vifor
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I/II)
14. EDIT 301: Editas Medicine
15. Drug profiles in the detailed report…..
16. Early Stage Products (Phase I)
17. ET-01: EdiGene Inc
18. Preclinical and Discovery Stage Products
19. Drug name: Company name
20. Drug profiles in the detailed report…..
21. Inactive Products
22. Beta-thalassaemia Key Companies
23. Beta-thalassaemia Key Products
24. Beta-thalassaemia- Unmet Needs
25. Beta-thalassaemia- Market Drivers and Barriers
26. Beta-thalassaemia- Future Perspectives and Conclusion
27. Beta-thalassaemia Analyst Views
28. Beta-thalassaemia Key Companies
29. Appendix

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