Sickle Cell Disease Pipeline Analysis 2024: FDA, EMA, and PDMA Approvals, Clinical Trials, Mechanisms of action, and ROA by DelveInsight | Companies - Bellicum Pharmaceuticals, Novartis, Novo Nordisk, Vertex Pharmaceuticals

(Albany, USA) DelveInsight's 'Sickle Cell Disease Pipeline Insight 2024' 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.

To know more about the Sickle Cell Disease pipeline report, click here @ https://www.delveinsight.com/report-store/sickle-cell-disease-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=apr

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 (SCD) is a genetic blood disorder characterized by abnormal hemoglobin molecules, leading to the formation of sickle-shaped red blood cells. These abnormal cells can cause blockages in blood vessels, leading to pain, organ damage, and increased risk of infections. Sickle cell disease is inherited in an autosomal recessive pattern, meaning that an individual must inherit two abnormal hemoglobin genes (one from each parent) to develop the disease.

Symptoms of Sickle cell disease can vary widely in severity and may include pain episodes (known as sickle cell crises), fatigue, jaundice, and delayed growth and development. Complications of Sickle cell disease can affect various organs and systems in the body, including the bones, lungs, kidneys, and spleen.

Treatment for Sickle cell disease focuses on managing symptoms and preventing complications. This may include pain management, hydration, blood transfusions, and medications to reduce the frequency of sickle cell crises. Bone marrow transplants may offer a cure for some individuals with severe Sickle cell disease. Early detection, regular medical care, and healthy lifestyle choices are essential for managing Sickle cell disease and improving quality of life.

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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