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Sickle Cell Disease Pipeline Landscape Analysis: 40+ Key Players & 50+ Pipeline Drugs

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Sickle Cell Disease Pipeline Landscape Analysis: 40+ Key Players & 50+ Pipeline Drugs

November 15
01:31 2022
Sickle Cell Disease Pipeline Landscape Analysis: 40+ Key Players & 50+ Pipeline Drugs

DelveInsight’s, “Sickle Cell Disease Pipeline Insights, 2022” report provides comprehensive insights about 40+ companies and 50+ pipeline drugs in Sickle cell disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the 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 Sickle Cell Disease Pipeline Insight Report

  • DelveInsight’s Sickle Cell Disease Pipeline analysis depicts a robust space with 40+ active players working to develop 50+ pipeline treatment therapies.
  • The leading Sickle Cell Disease Companies such as Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others.
  • Promising Sickle Cell Disease Pipeline therapies such as BPX-501 T cells, Canakinumab, EPI01, CTX001, and others.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Sickle Cell Disease (SCD) R&D. The therapies under development are focused on novel approaches to treat/improve Sickle Cell Disease (SCD).
  • This segment of the Sickle Cell Disease (SCD) report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

 

Recent Breakthroughs of Sickle Cell Disease Treatment Landscape

  • CTX001 has been granted Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. Food and Drug Administration (FDA) for both TDT and SCD. CTX001 has also been granted Orphan Drug Designation from the European Commission for both TDT and SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD. Currently it is being investigated in Phase III stage of development for SCD.
  • EpiDestiny has been granted Rare Pediatric Disease, Fast Track and Orphan Designations by the U.S. Food and Drug Administration (FDA) for EPI01.
  • Novartis is also developing ACZ885 for preventing cardiovascular risk. Company believe that ACZ885 is the first and only agent which has shown that selectively targeting inflammation significantly reduces cardiovascular risk in patients who have had a prior heart attack and have an increased cardiovascular inflammatory burden. Canakinumab is administered subcutaneously.
  • BPX-501 T cells (also known as Rivo-cel) is being developed by Bellicum Pharmaceuticals. BPX-501 T cells are derived from non-mobilized donor leukapheresis and are expanded, transduced and selected for the iC9 gene, cryopreserved and shipped back to the clinical site. Currently it is in Phase I/II stage of development for the treatment of Hematological Disorders. Rivo-cel is a T cell therapy incorporating CaspaCIDe safety switch intended to improve HSCT outcomes in the treatment of hematological malignancies and inherited blood disorders. Rivo-cel is designed to treat immunodeficiency following allogeneic HSCT, preventing morbidity and mortality due to disease relapse and infection.

 

Request a sample and discover the recent advances in Sickle Cell Disease Medication @ Sickle Cell Disease Treatment Landscape

 

Sickle Cell Disease Overview

Sickle cell disease is the name given for a group of inherited conditions that affect the red blood cells and the most serious type is called Sickle cell anemia. Sickle cells are destroyed rapidly in the bodies of people with the disease, causing anemia which is why it is commonly known as sickle cell anemia. The sickle cells also block the flow of blood through vessels, resulting in lung tissue damage that causes acute chest syndrome, pain episodes, stroke, priapism and damage to the spleen, kidneys and liver. Sickle cell disease is a serious and lifelong condition, although long-term treatment can help manage many of the problems associated with it. The disease affects hemoglobin, the molecule in red blood cells that delivers oxygen to cells throughout the body. There is an atypical hemoglobin molecule called hemoglobin S, which can distort red blood cells into a sickle, or crescent, shape. Sickle cell disease is caused by a mutation in the hemoglobin-Beta gene found on chromosome.

 

Sickle Cell Disease Emerging Drugs

  • BPX-501 T cells: Bellicum Pharmaceuticals
  • Canakinumab: Novartis
  • EPI01: Novo Nordisk
  • CTX001: Vertex Pharmaceuticals

 

Sickle Cell Disease Pipeline Therapeutics Analysis

There are approx. 40+ key companies which are developing the therapies for Sickle Cell Disease (SCD). The companies which have their Sickle Cell Disease (SCD) drug candidates in the most advanced stage, i.e. Phase III include Vertex Pharmaceuticals.

 

DelveInsight’s Sickle Cell Disease Pipeline report covers around 50+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates

 

Learn more about the emerging Sickle Cell Disease pipeline therapies @ Sickle Cell Disease Clinical Trials

 

Scope of the Sickle Cell Disease Pipeline Insight Report

  • Coverage- Global
  • Sickle cell disease Pipeline Assessment by Product Type
  • Sickle cell disease Pipeline Assessment by Stage and Product Type
  • Sickle cell disease Pipeline Assessment by Route of Administration
  • Sickle cell disease Pipeline Assessment by Stage and Route of Administration
  • Sickle cell disease Pipeline Assessment by Molecule Type
  • Sickle cell disease Pipeline Assessment by Stage and Molecule Type
  • Sickle cell disease Companies- Cellectis, Sana Biotechnology, Global Blood Therapeutics, Inc., CSL Behring, Pfizer, Aruvant Sciences, Graphite Bio, Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharmaceuticals, Global Blood Therapeutics, Inc., Alfasigma, Novo Nordisk, bluebird bio, ExCellThera, Gamida Cell, KM Biologics/Takeda, Editas Medicine, and others
  • Sickle Cell Disease Pipeline Therapies- BPX-501 T cells, Canakinumab, EPI01, CTX001, and others

 

Table of Content

  1. Introduction
  2. Executive Summary
  3. Sickle Cell Disease (SCD): Overview
  4. Pipeline Therapeutics
  5. Therapeutic Assessment
  6. Sickle Cell Disease (SCD)– DelveInsight’s Analytical Perspective
  7. Mid Stage Products (Phase III)
  8. CTX001: Vertex Pharmaceuticals
  9. Drug profiles in the detailed report…..
  10. Mid Stage Products (Phase II/III)
  11. Mitapivat: Agios Pharmaceuticals
  12. Drug profiles in the detailed report…..
  13. Early Stage Products (Phase I/II)
  14. ARU-1801: Aruvant Sciences
  15. Drug profiles in the detailed report…..
  16. Early Stage Products (Phase I)
  17. GBT021601: Global Blood Therapeutics, Inc.
  18. Drug profiles in the detailed report…..
  19. Preclinical Stage Products
  20. TALGlobin01: Cellectis
  21. Drug profiles in the detailed report…..
  22. Inactive Products
  23. Sickle Cell Disease (SCD) Key Companies
  24. Sickle Cell Disease (SCD) Key Products
  25. Sickle Cell Disease (SCD)- Unmet Needs
  26. Sickle Cell Disease (SCD)- Market Drivers and Barriers
  27. Sickle Cell Disease (SCD)- Future Perspectives and Conclusion
  28. Sickle Cell Disease (SCD) Analyst Views
  29. Sickle Cell Disease (SCD) Key Companies
  30. Appendix

 

Key Questions

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Sickle cell disease drugs?
  • How many Sickle cell disease drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Sickle cell disease?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Sickle cell disease therapeutics? 
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Sickle cell disease and their status?
  • What are the key designations that have been granted to the emerging drugs?

 

Got Queries? Get in touch with our Business Development Executive @ Sickle Cell Disease Market Drivers and Barriers

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Contact Person: Yash Bhardwaj
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