Beta Thalassemia Pipeline Landscape Analysis of 22+ Companies by DelveInsight | Leading Players – CRISPR Therapeutics, and Others

Beta Thalassemia Pipeline Landscape Analysis of 22+ Companies by DelveInsight | Leading Players - CRISPR Therapeutics, and Others

DelveInsight’s, “Beta Thalassemia Pipeline Insight, 2022,” report provides comprehensive insights about 22+ companies and 22+ pipeline drugs in Beta thalassemia pipeline landscape.

 

“Beta thalassemia Pipeline Insight, 2022” report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Beta thalassemia (B-thal) pipeline landscape is provided which includes the disease overview and Beta thalassemia treatment guidelines. The assessment part of the report embraces, in depth Beta thalassemia (B-thal) 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, Beta thalassemia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

 

Key takeaways from 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.
  • Leading Beta Thalassemia Companies such as CRISPR Therapeutics, Imara, Inc., Sangamo Therapeutics, Vifor Pharma, Bioray Laboratories, EdiGene (GuangZhou) Inc., Ionis Pharmaceuticals Inc., Agios Pharmaceuticals, Inc., Silence Therapeutics PLC, Aruvant Sciences, Phoenicia Biosciences, San Rocco Therapeutics, 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, and others are evaluating Beta Thalassemia drugs candidate to improve the treatment landscape.
  • The promising Beta Thalassemia therapies in the pipeline such as VIT-2763, ST-400, ET-01, EPEG, Emeramide, ACE-536, LentiGlobin BB305, and others

 

Latest Breakthroughs and Developments in the Beta Thalassemia Treatment Landscape

  • The U.S. Food and Drug Administration (FDA) has granted Fast Track designation, Rare Pediatric Disease designation, and Orphan Drug Designation for IMR-687 for the treatment of patients with beta-thalassaemia. IMR-687, which is an oral, once-a-day, potentially disease-modifying treatment for sickle cell disease (SCD) and β-thalassemia. IMR-687 is a highly selective, potent small molecule inhibitor of PDE9 (Phosphodiesterase 9A). Imara has evaluating the drug in Phase 2b clinical trial for patients with beta-thalassemia.
  • 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).

 

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Beta Thalassemia Overview

Beta thalassemia (B-thal) is an inherited blood disorder that reduces the production of hemoglobin. Affected individuals also have a shortage of red blood cells (anemia), which can cause pale skin, weakness, fatigue, and more serious complications. People with Beta thalassemia (B-thal) are at an increased risk of developing abnormal blood clots. The characteristic finding of Beta thalassemia (B-thal) is anemia, which is caused because red blood cells are abnormally small (microcytic), are not produced at the normal amounts, and do not contain enough functional hemoglobin. Beta thalassemia (B-thal) can be classified into two types depending on the severity of symptoms: thalassemia major (also known as Cooley’s anemia) and thalassemia intermedia. Of the two types, thalassemia major is more severe. Blood tests for diagnosis of Beta thalassemia (B-thal) can reveal the number of red blood cells and abnormalities in size, shape or color. Blood tests can also be used for DNA analysis to look for mutated genes. Mild forms of thalassemia trait don’t need treatment. For moderate to severe thalassemia, treatments might include: frequent blood transfusions, chelation therapy, and stem cell transplant.

 

Beta Thalassemia Emerging Drugs

  • CTX001: CRISPR Therapeutics
  • IMR-687: Imara

 

Beta Thalassemia Pipeline Therapeutics Analysis

There are approx. 22+ key companies which are developing the therapies for Beta thalassemia (B-thal). The companies which have their Beta thalassemia (B-thal) drug candidates in the most advanced stage, i.e. Phase IIb include, Imara.

 

Beta Thalassemia Pipeline Insight Report

  • The report provides detailed insights about emerging therapies for Beta Thalassemia treatment and the aggregate therapies developed by major pharma companies.
  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Beta Thalassemia Treatment.
  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
  • The Beta Thalassemia Pipeline Report evaluates the drugs that are 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.
  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in the Beta Thalassemia market.

The Beta Thalassemia Pipeline Report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third party sources etc.

 

Learn more about the novel and emerging Beta Thalassemia Pipeline therapies @ Beta Thalassemia Pipeline Analysis

 

Scope of the Beta Thalassemia Pipeline Report

  • Coverage- Global
  • Beta Thalassemia Companies– CRISPR Therapeutics, Imara, Inc., Sangamo Therapeutics, Vifor Pharma, Bioray Laboratories, EdiGene (GuangZhou) Inc., Ionis Pharmaceuticals Inc., Agios Pharmaceuticals, Inc., Silence Therapeutics PLC, Aruvant Sciences, Phoenicia Biosciences, San Rocco Therapeutics, 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, and others
  • Beta Thalassemia Therapies– VIT-2763, ST-400, ET-01, EPEG, Emeramide, ACE-536, LentiGlobin BB305, and others
  • Beta Thalassemia Therapeutic Assessment by Product Type
  • Beta Thalassemia Therapeutic Assessment by Stage and Product Type
  • Beta Thalassemia Therapeutic Assessment by Route of Administration
  • Beta Thalassemia Therapeutic Assessment by Stage and Route of Administration
  • Beta Thalassemia Therapeutic Assessment by Molecule Type
  • Beta Thalassemia Therapeutic Assessment by Stage and Molecule Type
  • Beta Thalassemia companies’ collaborations, Licensing, Acquisition -Deal Value Trends
  • Beta Thalassemia – Market Drivers and Barriers
  • Beta Thalassemia – Future Perspectives and Conclusion

 

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. Mid Stage Products (Phase II)
  8. IMR-687: Imara, Inc.
  9. Early Stage Products (Phase I)
  10. BD 211: Shanghai BDgene
  11. Preclinical and Discovery Stage Products
  12. DISC a: Disc Medicine
  13. Inactive Products
  14. Beta Thalassemia Key Companies
  15. Beta Thalassemia Key Products
  16. Beta Thalassemia – Unmet Needs
  17. Beta Thalassemia – Market Drivers and Barriers
  18. Beta Thalassemia – Future Perspectives and Conclusion
  19. Beta Thalassemia Analyst Views
  20. Beta Thalassemia Key Companies
  21. Appendix

 

For further information on the Beta Thalassemia Pipeline therapeutics, reach out to Beta Thalassemia Drugs

 

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