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.
For Sickle Cell Disease emerging drugs, the Sickle Cell Disease pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
Key Takeaways from the Sickle Cell Disease Pipeline Report
- DelveInsight’s Sickle Cell Disease Pipeline analysis depicts a robust space with 40+ active players working to develop 50+ pipeline therapies for Sickle Cell Disease treatment.
- The leading Sickle Cell Disease companies include 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 are evaluating their lead assets to improve the Sickle Cell Disease treatment landscape.
- Key Sickle Cell Disease pipeline therapies in various stages of development include TALGlobin01, SG418, GBT021601, CSL889, PF-07209326, ARU-1801, GPH101, OTQ923, Mitapivat, Etavopivat, CTX001, Inclacumab, Rifaximin, Decitabine, BCL11a shRNA(miR), Stem cell therapeutics, Omidubicel, Apadamtase alfa, EDIT 301, and others.
- In April 2023, bluebird Bio announced the submission of a Biologics Licence Application (BLA) for lovotibeglogene autotemcel (lovo-cel), a gene therapy treatment for sickle cell disease (SCD).
- In April 2023, Vertex Pharmaceuticals and CRISPR Therapeutics submitted an application to the US Food and Drug Administration (FDA) for the approval of exagamglogene autotemcel (exa-cel) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).
Request a sample and discover the recent breakthroughs happening in the Sickle Cell Disease pipeline landscape @ Sickle Cell Disease Pipeline Outlook
Sickle Cell Disease Overview
Sickle cell disease is the name given to a series of genetic disorders that affect red blood cells, the most serious of which is known as Sickle cell anemia. Sickle cells are rapidly destroyed in the bodies of patients with the condition, resulting in anemia, which is why it is generally called sickle cell anemia. The sickle cells also impede the passage of blood via vessels, causing lung tissue damage, acute chest syndrome, pain episodes, stroke, priapism, and damage to the spleen, kidneys, and liver. Sickle cell disease is a serious and chronic condition, but long-term treatment can help manage many of the issues associated with it. The condition affects hemoglobin, a protein found in red blood cells that transports oxygen throughout the body. Haemoglobin S, an unusual hemoglobin molecule, can distort red blood cells into a sickle or crescent shape. A mutation in the hemoglobin-Beta gene on the chromosome causes sickle cell disease.
Find out more about Sickle Cell Disease medication @ New Drug for Sickle Cell Disease
Sickle Cell Disease Treatment Analysis: Drug Profile
BPX-501 T cells: Bellicum Pharmaceuticals
Bellicum Pharmaceuticals is developing BPX-501 T cells (also known as Rivo-cel). T cells from non-mobilized donor leukapheresis are grown, transduced, and chosen for the iC9 gene before being cryopreserved and transported back to the clinical site. It is now in Phase I/II development for the treatment of Haematological Disorders. Rivo-cel is a T-cell therapy that includes a CaspaCIDe safety switch that is designed to improve HSCT results in the treatment of hematological cancers and hereditary blood diseases. Rivo-cel is intended to treat immunodeficiency after allogeneic HSCT, reducing morbidity and mortality associated with disease relapse and infection.
EPI01: Novo Nordisk
EPI01 is an oral, fixed-dose formulation of a DNA methyl-transferase enzyme 1 and cytidine deaminase inhibitor, decitabine, and tetrahydrouridine that has the potential to work by increasing the amount of fetal hemoglobin that can replace the defective hemoglobin in SCD patients, thereby preventing red blood cell deformation and improving blood oxygen levels. The FDA has granted EpiDestiny Rare Pediatric Disease, Fast Track, and Orphan Designations for EPI01.
Key Sickle Cell Disease Therapies and Companies
- BPX-501 T cells: Bellicum Pharmaceuticals
- Canakinumab: Novartis
- EPI01: Novo Nordisk
- CTX001: Vertex Pharmaceuticals
- TALGlobin01: Cellectis
- SG418: Sana Biotechnology
- GBT021601: Global Blood Therapeutics
- Hemopexin: CSL Behring
- PF-07209326: Pfizer
- Mitapivat: Agios Pharmaceuticals
Learn more about the novel and emerging Sickle Cell Disease pipeline therapies @ Sickle Cell Disease Clinical Trials
Sickle Cell Disease Therapeutics Assessment
- By Product Type
- Monotherapy
- Combination Therapy
- By Stage
- Discovery
- Pre-Clinical
- Phase I
- Phase II
- Phase III
- Pre-registration
- By Route of Administration
-
- Intranasal
- Intrathecal
- Intravenous
- Oral
- Parenteral
- Subcutaneous
- Intramuscular
- Transdermal
- By Molecule Type
- Antisense oligonucleotide
- Gene therapy
- Hormones
- Neuropeptides
- Oligonucleotides
- Small Molecule
- Triglyceride
Scope of the Sickle Cell Disease Pipeline Report
-
- Coverage: Global
- Key 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.
- Key Sickle Cell Disease Pipeline Therapies: TALGlobin01, SG418, GBT021601, CSL889, PF-07209326, ARU-1801, GPH101, OTQ923, Mitapivat, Etavopivat, CTX001, Inclacumab, Rifaximin, Decitabine, BCL11a shRNA(miR), Stem cell therapeutics, Omidubicel, Apadamtase alfa, EDIT 301, and others.
Dive deep into rich insights for drugs used for Sickle Cell Disease treatment; visit @ Sickle Cell Disease Drugs
Table of Contents
| 1. | Introduction |
| 2. | Executive Summary |
| 3. | Sickle Cell Disease Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Sickle Cell Disease Pipeline Therapeutics |
| 6. | Sickle Cell Disease Pipeline: Late Stage Products (Phase III) |
| 7. | Sickle Cell Disease Pipeline: Late Stage Products (Phase III) |
| 8. | Sickle Cell Disease Pipeline: Mid Stage Products (Phase II) |
| 9. | Sickle Cell Disease Pipeline: Early Stage Products (Phase I) |
| 10. | Sickle Cell Disease Therapeutic Assessment |
| 11. | Inactive Products |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Sickle Cell Disease Companies |
| 14. | Key Products |
| 15. | Sickle Cell Disease Unmet Needs |
| 16. | Sickle Cell Disease Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the Sickle Cell Disease therapy in the pipeline, reach out @ Sickle Cell Disease Medication
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