Beta Thalassemia Clinical Trials Pipeline Analysis: 22+ Companies are Working to Improve the Treatment Space | DelveInsight

The prevalence of beta thalassemia has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of Beta Thalassemia and the growing research and development activities to develop novel therapies to treat Beta Thalassemia drive the market. The companies developing the potential therapies in the last stage of development include CRISPR Therapeutics, CSL Vifor, EmeraMed, and several others.

New York, USA, Jan. 09, 2023 (GLOBE NEWSWIRE) — Beta Thalassemia Clinical Trials Pipeline Analysis: 22+ Companies are Working to Improve the Treatment Space | DelveInsight

The prevalence of beta thalassemia has been rising over the past few years, which prompts the growing demand for treatment options. The increasing prevalence of Beta Thalassemia and the growing research and development activities to develop novel therapies to treat Beta Thalassemia drive the market. The companies developing the potential therapies in the last stage of development include CRISPR Therapeutics, CSL Vifor, EmeraMed, and several others.

DelveInsight’s Beta Thalassemia Pipeline Insight – 2023 report provides comprehensive global coverage of available, marketed, and pipeline beta thalassemia therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the beta thalassemia pipeline domain.

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 25+ pipeline therapies for beta-thalassemia treatment. 
  • Key beta thalassemia companies such as CRISPR Therapeutics, CSL Vifor, EdiGene (GuangZhou) Inc., Ionis Pharmaceuticals, Inc., Agios Pharmaceuticals, Inc., Silence Therapeutics plc, Phoenicia Biosciences, Shanghai BDgene, Beam Therapeutics, EmeraMed, Regenacy Pharmaceuticals, Editas Medicine, Fulcrum Therapeutics, Allife Medical Science and Technology, Global Blood Therapeutics, Inc, Orchard Therapeutics, Acceleron Pharma, Disc Medicine, BRL Medicine, Celgene, and others are evaluating new drugs for beta thalassemia to improve the treatment landscape.
  • Promising beta thalassemia pipeline therapies in various stages of development 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.
  • 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.

Request a sample and discover the recent advances in beta-thalassemia treatment drugs @ Beta Thalassemia Pipeline Report

The beta-thalassemia pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage beta thalassemia drugs, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the beta thalassemia clinical trial landscape.

Beta Thalassemia Overview

Beta thalassemia (B-thal) is an inherited blood disorder that causes a decrease in hemoglobin production. Affected people also lack red blood cells (anemia), resulting in pale skin, weakness, fatigue, and other serious complications. People with beta thalassemia are more likely to develop abnormal blood clots. Anemia is a defining feature of beta thalassemia, which occurs when red blood cells are abnormally small (microcytic), are not produced in sufficient quantities, and do not contain enough functional hemoglobin. The severity of beta thalassemia symptoms distinguishes two types of beta thalassemia: thalassemia major (also known as Cooley’s anemia) and thalassemia intermedia. Thalassemia major is the more severe of the two types.

Blood tests for beta thalassemia can reveal the number of red blood cells present and abnormalities in size, shape, or color. Blood tests can also be used to analyze DNA for mutated genes. Mild cases of thalassemia trait do not require treatment. Treatments for moderate to severe thalassemia may include frequent blood transfusions, chelation therapy, and stem cell transplant.

Find out more about beta thalassemia treatment drugs @ Drugs for Beta Thalassemia Treatment 

A snapshot of the Beta Thalassemia Pipeline Drugs mentioned in the report:

Drugs Company Phase  MoA RoA
CTX001 CRISPR Therapeutics Phase III Cell replacements; Fetal hemoglobin expression stimulants Intravenous
Vamifeport  CSL Vifor Phase II Metal-transporting-protein-1-inhibitors Oral
Emeramide  EmeraMed Phase II Antioxidants; Chelating agents Oral
BEAM 101 Beam Therapeutics Preclinical Gene transference NA
FTX 6058 Fulcrum Therapeutics Preclinical Fetal hemoglobin expression stimulants; Polycomb repressive complex 2 inhibitors NA

Learn more about the emerging beta thalassemia pipeline therapies @ Beta Thalassemia Clinical Trials

Beta Thalassemia Therapeutics Assessment

The beta-thalassemia pipeline report proffers an integral view of beta thalassemia emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration.

Scope of the Beta Thalassemia Pipeline Report 

  • Coverage: Global 
  • Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
  • Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
  • Therapeutics Assessment By Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical
  • Therapeutics Assessment By Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy
  • Therapeutics Assessment By Mechanism of Action: Cell replacements, Fetal hemoglobin expression stimulants, Metal-transporting-protein-1-inhibitors, Gene transference, Antioxidants, Chelating agents, Fetal hemoglobin expression stimulants, Polycomb repressive complex 2 inhibitors
  • Key Beta Thalassemia Companies: CRISPR Therapeutics, CSL Vifor, EdiGene (GuangZhou) Inc., Ionis Pharmaceuticals, Inc., Agios Pharmaceuticals, Inc., Silence Therapeutics plc, Phoenicia Biosciences, Shanghai BDgene, Beam Therapeutics, EmeraMed, Regenacy Pharmaceuticals, Editas Medicine, Fulcrum Therapeutics, Allife Medical Science and Technology, Global Blood Therapeutics, Inc, Orchard Therapeutics, Acceleron Pharma, Disc Medicine, bluebird bio, BRL Medicine, Celgene, and others.
  • Key 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.

Dive deep into rich insights for new drugs for beta-thalassemia treatment; visit @ Beta Thalassemia Drugs

Table of Contents

1. Beta Thalassemia Pipeline Report Introduction
2. Beta Thalassemia Pipeline Report Executive Summary
3. Beta Thalassemia Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Beta Thalassemia Clinical Trial Therapeutics
6. Beta Thalassemia Pipeline: Late Stage Products (Pre-registration)
7. Beta Thalassemia Pipeline: Late Stage Products (Phase III)
7.1. CTX001: CRISPR Therapeutics
8. Beta Thalassemia Pipeline: Mid Stage Products (Phase II)
8.1. Vamifeport: CSL Vifor
9. Beta Thalassemia Pipeline: Early Stage Products (Phase I)
9.1. SLN124: Silence Therapeutics plc
10. Beta Thalassemia Pipeline Therapeutics Assessment
11. Inactive Products in the Beta Thalassemia Pipeline
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Key Companies
14. Key Products in the Beta Thalassemia Pipeline
15. Unmet Needs
16. Market Drivers and Barriers
17. Future Perspectives and Conclusion
18. Analyst Views
19. Appendix

For further information on the beta thalassemia pipeline therapeutics, reach out @ Beta Thalassemia Medications

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