United States, Nevada, Las Vegas, DelveInsight’s ‘Sickle Cell Disease Pipeline Insight 2023‘ report provides comprehensive global coverage of available, marketed, and pipeline Sickle Cell Disease therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Sickle Cell Disease pipeline domain.
Key Takeaways from the Sickle Cell Disease Pipeline Report
- Over 50+ Sickle Cell Disease pipeline therapies are in various stages of development, and their anticipated acceptance in the Sickle Cell Disease market would significantly increase market revenue.
- Leading Sickle Cell Disease companies developing novel drug candidates to improve the Sickle Cell Disease treatment landscape include Bellicum Pharmaceuticals, Novartis, Novo Nordisk, Vertex Pharmaceuticals, and others.
- Promising Sickle Cell Disease pipeline therapies in various stages of development include BPX-501 T cells, Canakinumab, EPI01, CTX001, and others.
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 Pipeline Analysis: Drug Profile
BPX-501 T cells: Bellicum Pharmaceuticals
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.
Discover more about the emerging Sickle Cell Disease drugs @ Sickle Cell Disease Treatment Drugs
Sickle Cell Disease Key Companies
- Bellicum Pharmaceuticals
- Novartis
- Novo Nordisk
- Vertex Pharmaceuticals
Sickle Cell Disease Pipeline Therapies
- BPX-501 T cells
- Canakinumab
- EPI01
- CTX001
Sickle Cell Disease Pipeline Therapeutics Assessment
- By development stage
- By product type
- By route of administration
- By molecule type
- By MOA type
Scope of the Sickle Cell Disease Pipeline Report
- Coverage: Global
- Key Sickle Cell Disease Companies: Bellicum Pharmaceuticals, Novartis, Novo Nordisk, Vertex Pharmaceuticals, and others
- Key Sickle Cell Disease Pipeline Therapies: BPX-501 T cells, Canakinumab, EPI01, CTX001, and others
Find out more about the Sickle Cell Disease treatment options in development @ Sickle Cell Disease Clinical Trials
Table of Contents
1. Introduction
2. Executive Summary
3. Overview
4. Pipeline Therapeutics
5. Late-Stage Products (Phase III)
6. Mid-Stage Products (Phase II)
7. Early Stage Products (Phase I/II)
8. Preclinical Stage Products
9. Discovery Stage Products
10. Therapeutic Assessment
11. Inactive Products
12. Collaborations Assessment- Licensing / Partnering / Funding
13. Unmet Needs
14. Market Drivers and Barriers
15. Appendix
16. About DelveInsight
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