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DelveInsight’s, “Beta thalassemia (B-thal) Pipeline Insight 2023” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in Beta thalassemia (B-thal) pipeline landscape. It covers the Beta thalassemia pipeline drug profiles, including Beta thalassemia clinical trials and nonclinical stage products. It also covers the Beta thalassemia therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

 

Key Takeaways from the Beta thalassemia Pipeline Report

 

• 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 includes 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 Pipeline Therapies include CTX001, VIT-2763, β-globin restored autologous HSC, BEAM 101, Emeramide, FTX 6058, EDIT 301, CRISPR/Cas9 modified human haematopoietic stem cell therapy, Research programme: HDAC1/2 inhibitors, ET-01, IONIS TMPRSS6-LRx, Mitapivat, SLN124, PB 04, Beta globin transduced bone marrow cells, BD 211, DISC a, BRL 101, ACE-011,  and others.

 

• The Beta Thalassemia companies and academics are working to assess challenges and seek opportunities that could influence Beta-thalassaemia R&D. The Beta Thalassemia therapies under development are focused on novel approaches to treat/improve Beta-thalassaemia.

 

Request a sample and discover the recent breakthroughs happening in the Beta Thalassemia Pipeline landscape @ Beta Thalassemia Pipeline Outlook Report

 

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.

 

Recent Developmental Activities in the Beta thalassemia Treatment Landscape

 

• In December 2022, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced an oral, encore presentation of clinical data from patients with sickle cell disease (SCD) or transfusion-dependent beta-thalassemia (TDT) treated with the investigational therapy exagamglogene autotemcel (exa-cel) in CLIMB-111 or CLIMB-121 and followed in CLIMB-131, a long-term follow-up study. Vertex will also present new health economics and outcomes research from multiple studies in patients with SCD and TDT.

 

• In December 2022, Agios Pharmaceuticals, Inc. reported new data from the ongoing long-term extension period of Phase II open-label study of PYRUKYND® (mitapivat), a first-in-class, oral, small molecule allosteric activator of wild-type and a variety of mutated pyruvate kinase (PK) enzymes, in adults with non-transfusion dependent α- or β-thalassemia. Data from the study showed, consistent with previously reported data, durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients. Additionally, markers of iron homeostasis remained stable or improved through Week 72. PYRUKYND was well tolerated, and the safety profile was consistent with previous studies.

 

• In September 2022, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that Vertex had concluded discussions with the U.S. Food and Drug Administration (FDA), and the FDA granted exagamglogene autotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

 

• In August 2022, Following the FDA approval of ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, for the treatment of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions, bluebird bio released details of its U.S. commercial infrastructure to support rapid access to ZYNTEGLO, including an innovative, outcomes-based contract offering and a comprehensive patient support program.

 

• In August 2022, The U.S. Food and Drug Administration (USFDA) approved bluebird bio’s gene therapy, ZYNTEGLO, for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million. According to a report, the approval sent the company’s shares 8 percent higher and is for the treatment of beta-thalassemia, which causes an oxygen shortage in the body and often leads to liver and heart issues.

 

For further information, refer to the detailed Beta Thalassemia Drugs Launch, Beta Thalassemia Developmental Activities, and Beta Thalassemia News, click here for Beta Thalassemia Ongoing Clinical Trial Analysis

 

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.

 

Beta Thalassemia Pipeline Therapeutics Assessment

There are approx. 22+ key companies which are developing the therapies for Beta-thalassaemia. The companies which have their Beta-thalassaemia drug candidates in the most advanced stage, i.e. pre-registration include, CRISPR Therapeutics.

 

Find out more about the Beta Thalassemia Diagnosis and Treatment of patients @ Beta Thalassemia Ongoing Clinical Trials Analysis

 

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

 

• Beta Thalassemia Pipeline Therapies- CTX001, VIT-2763, β-globin restored autologous HSC, BEAM 101, Emeramide, FTX 6058, EDIT 301, CRISPR/Cas9 modified human haematopoietic stem cell therapy, Research programme: HDAC1/2 inhibitors, ET-01, IONIS TMPRSS6-LRx, Mitapivat, SLN124, PB 04, Beta globin transduced bone marrow cells, BD 211, DISC a, BRL 101, ACE-011,  and others.

 

• Beta Thalassemia Pipeline Segmentation: Product Type, Molecule Type, Route of Administration

 

Discover more about the list of FDA-approved drugs for Beta Thalassemia @ Beta Thalassemia Treatment Landscape

 

Table of Content

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. 29.   Appendix

 

For further information on the Beta Thalassemia Pipeline therapeutics, reach out @ Beta Thalassemia Market Drivers and Barriers

 

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