The Sickle Cell Disease Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.

“Sickle Cell Disease Pipeline Insight, 2023“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Sickle Cell Disease Market.

Some of the key takeaways from the Sickle Cell Disease Pipeline Report:

• Companies across the globe are diligently working toward developing novel Sickle Cell Disease treatment therapies with a considerable amount of success over the years. 
• Sickle Cell Disease companies working in the treatment market are Afimmune, IHP Therapeutics, Hillhurst Biopharmaceuticals, Agios Pharmaceuticals, Novo Nordisk, Alexion Pharmaceuticals, Prolong Pharmaceuticals, CRISPR Therapeutics, Vertex Pharmaceuticals, Pfizer, Agios Pharmaceuticals, Novartis, Alexion Pharmaceuticals, Chugai Pharmaceutical, Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others, are developing therapies for the Sickle Cell Disease treatment 
• Emerging Sickle Cell Disease therapies in the different phases of clinical trials are- Epeleuton, IHP-102, HBI-002, AG-946, EPI01, ALXN1820, Sanguinate, Exagamglogene autotemcel, Inclacumab, Mitapivat, Canakinumab, ALXN1820, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others are expected to have a significant impact on the Sickle Cell Disease market in the coming years.   
• In May 2023, The experimental cell-based gene-editing therapy, EDIT-301, received orphan drug designation from the US Food and Drug Administration (FDA). This designation applies to a one-time infusion treatment designed for addressing sickle cell disease (SCD).
• In April 2023, Bluebird bio, Inc. has reported the submission of its Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for the gene therapy lovotibeglogene autotemcel (lovo-cel) in patients aged 12 and above with sickle cell disease (SCD) and a history of vaso-occlusive events (VOEs). The BLA includes a request for Priority Review, which, if granted, would expedite the FDA’s assessment to six months from the filing date, compared to the standard review timeline of 10 months. If approved, lovo-cel would mark bluebird bio’s third ex-vivo gene therapy endorsed by the FDA for a rare genetic disease and its second FDA approval for an inherited hemoglobin disorder, further building on the company’s over a decade-long leadership in gene therapy.
• In March 2023, BioLineRx Ltd. has disclosed a partnership with Washington University School of Medicine in St. Louis to progress a Phase I clinical trial assessing the safety and feasibility of the company’s primary clinical candidate, motixafortide. The trial aims to mobilize CD34+ hematopoietic stem cells (HSCs) for gene therapies targeting sickle cell disease (SCD).
• In October 2022, Pfizer Inc. has declared the finalization of its acquisition of GBT, a biopharmaceutical company focused on discovering, developing, and delivering transformative treatments, with a particular focus on underserved patient communities, beginning with sickle cell disease (SCD).

Sickle Cell Disease Overview

Sickle Cell Disease (SCD) is a genetic disorder characterized by the presence of abnormal hemoglobin, known as hemoglobin S (HbS), in red blood cells. Hemoglobin is a protein in red blood cells responsible for carrying oxygen from the lungs to the rest of the body.

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

• Epeleuton: Afimmune
• IHP-102: IHP Therapeutics
• HBI-002: Hillhurst Biopharmaceuticals
• AG-946: Agios Pharmaceuticals
• EPI01: Novo Nordisk
• ALXN1820: Alexion Pharmaceuticals
• Sanguinate: Prolong Pharmaceuticals
• Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals
• Inclacumab: Pfizer
• 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

Sickle Cell Disease Route of Administration

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 Molecule Type

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 Therapeutics Assessment

• Sickle Cell Disease Assessment by Product Type
• Sickle Cell Disease By Stage and Product Type
• Sickle Cell Disease Assessment by Route of Administration
• Sickle Cell Disease By Stage and Route of Administration
• Sickle Cell Disease Assessment by Molecule Type
• Sickle Cell Disease by Stage and Molecule Type

DelveInsight’s Sickle Cell Disease Report covers around 50+ products under different phases of clinical development like

• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I)
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Further Sickle Cell Disease product details are provided in the report. Download the Sickle Cell Disease pipeline report to learn more about the emerging Sickle Cell Disease therapies

Some of the key companies in the Sickle Cell Disease Therapeutics Market include:

Key companies developing therapies for Sickle Cell Disease are – Addmedica, Medunik USA, Bristol Myers Squibb Co., Sanofi SA, Bluebird Biotechnology, Pfizer Inc., Aruvant Sciences Inc., Novartis AG, Global Blood Therapeutics Inc., Emmaus Medical Inc., Glycomimetics Inc., Editas Medicine Inc., CRISPR Therapeutic, and others.

Sickle Cell Disease Pipeline Analysis:

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 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.

Download Sample PDF Report to know more about Sickle Cell Disease drugs and therapies

Sickle Cell Disease Pipeline Market Drivers

• Increasing prevalence of Sickle Cell Disease, rising initiatives by various governments and private organizations are some of the important factors that are fueling the Sickle Cell Disease Market.

Sickle Cell Disease Pipeline Market Barriers

• However, clinical heterogeneity of the disease, associated complications of the disease and other factors are creating obstacles in the Sickle Cell Disease Market growth.

Scope of Sickle Cell Disease Pipeline Drug Insight    

• Coverage: Global
• Key Sickle Cell Disease Companies: Afimmune, IHP Therapeutics, Hillhurst Biopharmaceuticals, Agios Pharmaceuticals, Novo Nordisk, Alexion Pharmaceuticals, Prolong Pharmaceuticals, CRISPR Therapeutics, Vertex Pharmaceuticals, Pfizer, Agios Pharmaceuticals, Novartis, Alexion Pharmaceuticals, Chugai Pharmaceutical, Roche, Editas Medicine, Sangamo Therapeutics, Beam Therapeutics, CSL Behring, and others
• Key Sickle Cell Disease Therapies: Epeleuton, IHP-102, HBI-002, AG-946, EPI01, ALXN1820, Sanguinate, Exagamglogene autotemcel, Inclacumab, Mitapivat, Canakinumab, ALXN1820, Crovalimab, EDIT 301, BIVV003, BEAM101, Hemopexin, and others
• Sickle Cell Disease Therapeutic Assessment: Sickle Cell Disease current marketed and Sickle Cell Disease emerging therapies
• Sickle Cell Disease Market Dynamics: Sickle Cell Disease market drivers and Sickle Cell Disease market barriers 

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Table of Contents

1. Sickle Cell Disease Report Introduction

2. Sickle Cell Disease Executive Summary

3. Sickle Cell Disease Overview

4. Sickle Cell Disease- Analytical Perspective In-depth Commercial Assessment

5. Sickle Cell Disease Pipeline Therapeutics

6. Sickle Cell Disease Late Stage Products (Phase II/III)

7. Sickle Cell Disease Mid Stage Products (Phase II)

8. Sickle Cell Disease Early Stage Products (Phase I)

9. Sickle Cell Disease Preclinical Stage Products

10. Sickle Cell Disease Therapeutics Assessment

11. Sickle Cell Disease Inactive Products

12. Company-University Collaborations (Licensing/Partnering) Analysis

13. Sickle Cell Disease Key Companies

14. Sickle Cell Disease Key Products

15. Sickle Cell Disease Unmet Needs

16 . Sickle Cell Disease Market Drivers and Barriers

17. Sickle Cell Disease Future Perspectives and Conclusion

18. Sickle Cell Disease Analyst Views

19. Appendix

20. About DelveInsight

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

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