DelveInsight’s “Sickle Cell Disease Pipeline Insights 2025” report provides comprehensive insights about 55+ companies and 60+ drugs in the Sickle Cell Disease Competitive landscape. It covers the Sickle Cell Disease Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Sickle Cell Disease Pipeline Report

• In April 2025, Kamau Therapeutics announced a study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.
• In April 2025, Novartis Pharmaceuticals conducted a phase III study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.
• In April 2025, Novo Nordisk A/S organized a study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance.
• DelveInsight’s Sickle Cell Disease pipeline report depicts a robust space with 55+ active players working to develop 60+ pipeline therapies for Sickle Cell Disease treatment.
• The leading Sickle Cell Disease Companies such as Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc, GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, Base Therapeutics and others.
• Promising Sickle Cell Disease Pipeline Therapies such as Siplizumab, Daratumumab, Alemtuzumab, Sirolimus, ITU512, BEAM-101, Crizanlizumab, Etavopivat, Epeleuton, Voxelotor and others.

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Sickle Cell Disease Marketed Therapies

• Vertex Pharmaceuticals: CASGEVY

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

CASGEVY™ is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for certain indications in multiple jurisdictions for eligible patients.

• Emmaus Medical Inc.: ENDARI

Emmaus Life Sciences, Inc. is a commercial-stage biopharmaceutical company and leader in the treatment of sickle cell disease. The company is engaged in the discovery, development and commercialization of innovative treatments and therapies primarily for rare and orphan diseases. The initial efforts have focused on treatments for Sickle Cell Disease, a genetic disorder. Emmaus’s lead commercial product is Endari®, an oral pharmaceutical grade L-glutamine treatment indicated to reduce acute complications of sickle cell disease in adult and pediatric patients five years of age and older.

ENDARI [L-glutamine oral powder] is indicated to reduce the acute complications of sickle cell disease in adults and children 5 years and older. ENDARI is an amino acid indicated to reduce the acute complication of sickle cell disease in adult. The mechanism of action of the amino acid L-glutamine in treating sickle cell disease (SCD) is not fully understood. Oxidative stress phenomena are involved in the pathophysiology of SCD. Sickle red blood cells (RBCs) are more susceptible to oxidative damage than normal RBCs, which may contribute to the chronic hemolysis and vaso-occlusive events associated with SCD. The pyridine nucleotides, NAD+ and its reduced form NADH, play roles in regulating and preventing oxidative damage in RBCs. L-glutamine may improve the NAD redox potential in sickle RBCs through increasing the availability of reduced glutathione.

The Sickle Cell Disease Pipeline Report provides insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of Sickle Cell Disease with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Sickle Cell Disease Treatment.
• Sickle Cell Disease Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Sickle Cell Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Sickle Cell Disease market.

Emerging Sickle Cell Disease Drugs Under Different Phases of Clinical Development Include:

• RL 101: BRL Medicine
• ORY-300:1 Oryzon Genomics
• GSK 4172239D: GlaxoSmithKline
• AG-946: Agios Pharmaceuticals
• BEAM-101: Beam Therapeutics Inc.
• Nula-cel: Graphite Bio
• RG 6107: Roche
• Isoquercetin: Quercis Pharma
• Renizgamglogene autogedtemcel: Editas Medicin
• Inclacumab: Pfizer
• L-glutamine: Emmaus Medical, Inc
• CASGEVY: Vertex Pharmaceuticals
• ESCAPE: Beam Therapeutic
• IHP-102: IHP Therapeutics
• HBI-002: Hillhurst Biopharmaceuticals
• BEAM101: Beam Therapeutics
• EPI01: Novo Nordisk
• VIT-2763: CSL Vifor
• Inclacumab: Pfizer
• L-glutamine: Emmaus Medical
• Oxbryta: Pfizer
• Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals
• Mitapivat: Agios Pharmaceuticals
• Canakinumab: Novartis
• ALXN1820: Alexion Pharmaceuticals
• Crovalimab: Chugai Pharmaceutical/Roche
• EDIT 301: Editas Medicine
• BIVV003: Sangamo Therapeutics
• BEAM101: Beam Therapeutics
• Hemopexin: CSL Behring

Get a detailed analysis of the latest innovations in the Sickle Cell Disease pipeline. Explore DelveInsight’s expert-driven report today! @ Sickle Cell Disease Unmet Needs

Sickle Cell Disease Companies

Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc, GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, Base Therapeutics, and others.

Sickle Cell Disease Pipeline Report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as

• Intranasal

• Intrathecal

• Intravenous

• Oral

• Oral/Intravenous

• Parenteral

• Subcutaneous

• Subcutaneous/Intramuscular

• Transdermal

Sickle Cell Disease Products have been categorized under various Molecule types, such as

• Antisense oligonucleotide

• Gene therapy

• Hormones

• Neuropeptides

• Oligonucleotides

• Small Molecule

• Triglyceride

Sickle Cell Disease Pipeline Analysis

• The report provides detailed insights about companies that are developing therapies for the treatment of Sickle Cell Disease with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Sickle Cell Disease Treatment.
• Sickle Cell Disease key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Sickle Cell Disease Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Sickle Cell Disease market.
• The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.

Discover the latest advancements in Sickle Cell Disease Treatment by visiting our website. Stay informed about how we’re transforming the future of Genetic Disorders @ Sickle Cell Disease Market Drivers and Barriers, and Future Perspectives

Scope of the Sickle Cell Disease Pipeline Report

• Coverage- Global
• Sickle Cell Disease Companies- Pfizer, Editas Medicine, Roche, Quercis Pharma, Graphite Bio, Beam Therapeutics Inc, GlaxoSmithKline, Agios Pharmaceuticals, BRL Medicine, Oryzon Genomics, CorrectSequence Therapeutics, GluBio Therapeutics, Base Therapeutics and others.
• Sickle Cell Disease Pipeline Therapies- Siplizumab, Daratumumab, Alemtuzumab, Sirolimus, ITU512, BEAM-101, Crizanlizumab, Etavopivat, Epeleuton, Voxelotor and others.
• Sickle Cell Disease Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Sickle Cell Disease Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

For a detailed overview of our latest research findings and future plans, read the full details of Sickle Cell Disease Pipeline on our website @ Sickle Cell Disease Emerging Drugs and Companies

Table of Contents

1. Introduction
2. Executive Summary
3. Sickle Cell Disease (SCD): Overview
4. Sickle Cell Disease -Analytical Perspective: In-depth Commercial Assessment
5. Competitive Landscape
6. Sickle Cell Disease: Company and Product Profiles (Marketed Therapies)
7. Vertex Pharmaceuticals
8. CASGEVY
9. Sickle Cell Disease: Company and Product Profiles (Pipeline Therapies)
10. Late Stage Products (Phase III)
11. Pfizer
12. Inclacumab
13. Drug profiles in the detailed report…..
14. Mid Stage Products (Phase II)
15. Roche
16. RG 6107
17. Drug profiles in the detailed report…..
18. Early Stage Products (Phase I/II)
19. Beam Therapeutics
20. BEAM-101
21. Drug profiles in the detailed report…..
22. Preclinical and Discovery Stage Products
23. BRL Medicine
24. BRL 101
25. Drug profiles in the detailed report…..
26. Inactive Products
27. Sickle Cell Disease- Unmet needs
28. Sickle Cell Disease – Market drivers and barriers
29. Appendix

About Us

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