Advancements in Sickle Cell Disease Therapies: Updates on FDA, EMA, and PDMA Approvals, Clinical Trials, Mechanisms, and Leading Companies by DelveInsight | Featuring Bellicum Pharmaceuticals, Novartis, Novo Nordisk, Vertex Pharmaceuticals

(Albany, USA) DelveInsight's 'Sickle Cell Disease Pipeline Insight' 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.

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Some facts of the Sickle Cell Disease Pipeline Report
• Over 50+ Sickle Cell Disease 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.
• Emerging Sickle Cell Disease pipeline therapies in various stages of development include BPX-501 T cells, Canakinumab, EPI01, CTX001, and others.
• Mozobil (plerixafor) was initially developed by Genzyme. In April 2011, Sanofi acquired Genzyme and it turned into a wholly owned subsidiary.
• In January 2020, Aruvant announced that the US Food and Drug Administration has granted Orphan Drug designation to ARU-1801, Aruvant's investigational therapy for the treatment of sickle cell disease. The US Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation to ARU-1801.

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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 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.

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Sickle Cell Disease Companies:
Leading Sickle Cell Disease companies working in the treatment market are Cellectis, Sana Biotechnology, 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:
Some of the Sickle Cell Disease therapies developed by the major players in different phases include BPX-501 T cells, Canakinumab, EPI01, CTX001, and others.

Scope of the Sickle Cell Disease Pipeline Report
• Coverage: Global
• Key Sickle Cell Disease Companies: Cellectis, Sana Biotechnology, 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
• Key Sickle Cell Disease Pipeline Therapies: BPX-501 T cells, Canakinumab, EPI01, CTX001, and others

• Sickle Cell Disease Pipeline Therapeutics Assessment (By development stage, By product type, By route of administration, By molecule type, )By MOA type

Find out more about the Sickle Cell Disease treatment options in development @ https://www.delveinsight.com/sample-request/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

Table of Contents
1. Introduction
2. Executive Summary
3. Sickle Cell Disease Overview
4. Sickle Cell Disease 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. Sickle Cell Disease Therapeutic Assessment
11. Inactive Sickle Cell Disease Products
12. Collaborations Assessment- Licensing / Partnering / Funding
13. Sickle Cell Disease Unmet Needs
14. Sickle Cell Disease Market Drivers and Barriers
15. Appendix
16. About DelveInsight

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About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

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