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DelveInsight’s 2025 report on the Beta Thalassemia Pipeline offers detailed analysis of over 22 companies and more than 22 drugs in development. It includes profiles of pipeline therapies at clinical and non-clinical stages, evaluations by product type, development phase, administration route, and molecular category. The report also covers inactive projects in this area.
Interested in recent developments in the Beta Thalassemia Pipeline? Explore the therapies and studies gaining attention in the Beta Thalassemia Pipeline Outlook Report.
Key Highlights from the Beta Thalassemia Pipeline Report
On October 14, 2025, Vertex Pharmaceuticals Incorporated initiated a study in pediatric patients with transfusion-dependent thalassemia (TDT), assessing the safety and effectiveness of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) known as CTX001.
On October 14, 2025, Regeneron Pharmaceuticals announced a study on an investigational medication called REGN7999, targeting patients with non-transfusion-dependent beta-thalassemia to evaluate its safety and efficacy.
On October 2, 2025, Celgene launched a two-part study for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) beta-thalassemia patients: TD Part A focuses on adolescents aged 12.
DelveInsight’s Beta Thalassemia Pipeline analysis reveals a dynamic field with over 22 active entities developing more than 22 therapies for Beta Thalassemia management.
Leading companies in Beta Thalassemia include CRISPR Therapeutics, CSL Vifor, Beam Therapeutics, EmeraMed, Fulcrum Therapeutics, Editas Medicine, EdiGene Inc, Silence Therapeutics, Phoenicia Biosciences, Shanghai BDgene, Disc Medicine, and others.
Promising therapies encompass Vebeglogene Autotemcel, ACE-536, Luspatercept, HQK-1001, Deferitrin (GT56-252), Desferoxamine (DFO), Bitopertin, ICL670, and more.
Want to identify which organizations are driving innovation in Beta Thalassemia? Explore the complete pipeline details in the Beta Thalassemia Clinical Trials Assessment.
Understanding Beta Thalassemia
Beta Thalassemia is a genetic blood disorder that impairs the body's production of beta-globin, a crucial protein for hemoglobin and red blood cell formation. This can lead to anemia symptoms. Variants include beta thalassemia major, intermedia, and minor. Symptoms vary by type and severity, potentially involving fatigue, weakness, pale or yellowish skin, facial bone changes, delayed growth, abdominal swelling, and dark urine.
Profiles of Emerging Beta Thalassemia Drugs
CTX001: CRISPR Therapeutics
CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell treatment for beta-thalassemia and sickle cell disease patients. It is in Phase I/II trials for beta-thalassemia. The therapy holds orphan drug status in the U.S. and Europe, plus fast track, rare pediatric disease, and regenerative medicine advanced therapy designations in the U.S. for sickle cell disease and transfusion-dependent beta-thalassemia (TDT).VIT-2763: CSL Vifor
VIT-2763 is an oral small molecule from Vifor Pharma, designed for daily use to address iron metabolism disorders. It targets ferroportin, an iron transporter, by binding and inhibiting it to curb excessive iron release into the bloodstream. Preclinical data supports its role in lowering elevated iron levels in blood and tissues, and limiting iron absorption in conditions with altered metabolism. It is currently in Phase II for beta-thalassemia, a rare inherited disorder causing reduced functional hemoglobin and potential anemia.Emeramide: EmeraMed
Emeramide acts as an antioxidant and heavy metal chelator, protecting against methylmercury-induced glutathione loss and cell damage in mouse aortic endothelial cells at 50 µM. As a lipophilic di-thiol compound, it offers antioxidant, antiviral, and metal-chelating properties. It has received orphan drug designations for mercury toxicity in the EU and U.S. Safety studies, including Phase I and four Phase II trials for various conditions, have been completed. The drug is now in Phase II for beta-thalassemia.
If you're following active Beta Thalassemia Clinical Trials, this update is essential. Discover the advancements in Beta Thalassemia Treatment Drugs.
The Beta Thalassemia Pipeline report offers insights into:
Detailed profiles of companies developing Beta Thalassemia therapies, including the number of treatments per company.
Categorization of therapeutic candidates into early, mid, and late development stages for Beta Thalassemia care.
Companies engaged in targeted drug development, with information on active and inactive (dormant or discontinued) projects.
Drugs in development, analyzed by stage, administration route, target receptor, monotherapy or combination, mechanism of action, and molecular type.
Comprehensive examination of partnerships (company-to-company and company-academia), licensing deals, and funding to advance the Beta Thalassemia market.
Beta Thalassemia Companies
Key players include 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 Therapeutic Assessment by Route of Administration
The report classifies pipeline drugs by delivery methods, including:
Intravenous
Subcutaneous
Oral
Intramuscular
Beta Thalassemia Products by Molecule Type
Products are grouped by molecular categories, such as:
Monoclonal antibody
Small molecule
Peptide
From promising new candidates to market intelligence, the Beta Thalassemia Pipeline Report covers everything—review it at Beta Thalassemia Market Drivers and Barriers, and Future Perspectives.
Scope of the Beta Thalassemia Pipeline Report
Coverage: Worldwide.
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 Therapies: Vebeglogene Autotemcel, ACE-536, Luspatercept, HQK-1001, Deferitrin (GT56-252), Desferoxamine (DFO), Bitopertin, ICL670, and others.
Therapeutic Assessment by Product Type: Monotherapy, Combination Therapy, or Both.
Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III.
Stay informed in healthcare research—learn about future trends in the Beta Thalassemia Treatment landscape through this comprehensive analysis at Beta Thalassemia Emerging Drugs and Major Players.
Table of Contents
Introduction
Executive Summary
Beta-thalassaemia: Overview
Pipeline Therapeutics
Therapeutic Assessment
Beta-thalassaemia – DelveInsight’s Analytical Perspective
Late Stage Products (Preregistration)
Exa-cel: CRISPR Therapeutics
(Detailed profiles in the full report...)
Mid Stage Products (Phase II)
VIT-2763: CSL Vifor
(Detailed profiles in the full report...)
Early Stage Products (Phase I/II)
EDIT 301: Editas Medicine
(Detailed profiles in the full report...)
Early Stage Products (Phase I)
ET-01: EdiGene Inc
Preclinical and Discovery Stage Products
Drug Name: Company Name
(Detailed profiles in the full report...)
Inactive Products
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
About DelveInsight
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