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Sickle Cell Disease Pipeline

(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 @ Sickle Cell Disease Pipeline Insight

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.

Find out more about Sickle Cell Disease treatment drugs @ Sickle Cell Disease Clinical Trials

Sickle Cell Disease Overview

Sickle cell disease (SCD) is a genetic blood disorder characterized by the production of abnormal hemoglobin, known as hemoglobin S. This abnormal hemoglobin causes red blood cells to become rigid, sticky, and crescent-shaped, resembling a sickle. These deformed cells can obstruct blood flow, leading to painful episodes known as sickle cell crises, and cause damage to organs and tissues.

Sickle cell disease primarily affects individuals of African, Mediterranean, Middle Eastern, and Indian ancestry. Symptoms of SCD vary but often include severe pain, anemia, fatigue, swelling in hands and feet, frequent infections, and delayed growth in children. Chronic complications can involve the lungs, kidneys, liver, and other organs, potentially leading to stroke, acute chest syndrome, and organ failure.

Sickle cell disease Diagnosis is usually confirmed through newborn screening or genetic testing. Treatment focuses on managing symptoms and preventing complications. This may involve pain relief strategies, blood transfusions, and medications like hydroxyurea to reduce the frequency of pain crises and other complications. Recently, advances in gene therapy and bone marrow transplants have shown promise as potential cures for Sickle cell disease.

Preventive measures for Sickle cell disease include regular medical care, vaccinations, and antibiotics to prevent infections, alongside lifestyle adjustments to avoid triggers of pain crises. Ongoing research aims to find more effective treatments and ultimately a universal cure for this debilitating disease.

Find out more about Sickle Cell Disease Pipeline @ Sickle Cell Disease Therapeutic Assessment

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. 

Canakinumab: Novartis

Novartis is developing Canakinumab (ACZ885), a selective, high-affinity, fully human monoclonal antibody that acts as an interleukin-1β (IL-1β) blocker, currently in Phase II development for treating sickle cell anemia. IL-1β is a key cytokine in the inflammatory pathway, driving the progression of inflammatory atherosclerosis. ACZ885 blocks IL-1β, inhibiting inflammation caused by its overproduction. Already approved under the brand name Ilaris for Cryopyrin-Associated Periodic Syndromes (CAPS), including Familial Cold Autoinflammatory Syndrome (FCAS) and Muckle-Wells Syndrome (MWS), Canakinumab is also being developed to prevent cardiovascular risk. It is the first and only agent shown to selectively target inflammation, significantly reducing cardiovascular risk in patients with a history of heart attacks and increased cardiovascular inflammatory burden. Canakinumab is administered subcutaneously.

EPI01: Novo Nordisk

EPI01 is an oral, fixed-dose formulation of a DNA methyltransferase enzyme 1 and cytidine deaminase inhibitor, decitabine, and tetrahydrouridine. It potentially increases the amount of fetal hemoglobin, which can substitute the defective hemoglobin in sickle cell disease (SCD) patients, preventing red blood cell deformation and improving oxygen levels in the blood. EpiDestiny has been granted Rare Pediatric Disease, Fast Track, and Orphan Designations by the U.S. FDA for EPI01.

CTX001: Vertex Pharmaceuticals

CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy evaluated for patients with transfusion-dependent beta-thalassemia (TDT) or severe sickle cell disease (SCD). It edits a patient’s hematopoietic stem cells to produce high levels of fetal hemoglobin (HbF), reducing transfusion requirements for TDT patients and painful sickle crises for SCD patients. CTX001 has received Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the U.S. FDA for both TDT and SCD. It also has Orphan Drug Designation from the European Commission and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA) for SCD. Currently, CTX001 is in Phase III development for SCD.

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 @ Sickle Cell Disease Drugs and Phases

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 Sickle Cell Disease Products

9. Discovery Stage Sickle Cell Disease 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

15. Sickle Cell Disease Market Barriers

16. Appendix

17. About DelveInsight

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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