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, Pfizer, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Novartis, 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, Inclacumab, Exagamglogene autotemcel, Mitapivat, Canakinumab, 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, which is administered as a single infusion for the treatment of sickle cell disease (SCD), has been accorded orphan drug status by the US Food and Drug Administration (FDA).
• In April 2023, The US Food and Drug Administration (FDA) has received the biologics licence application (BLA) for the gene therapy lovotibeglogene autotemcel (lovo-cel) from bluebird bio, Inc. for sickle cell disease (SCD) patients aged 12 and older with a history of vaso-occlusive episodes (VOEs). The BLA contains a request for Priority evaluation, which, if approved, would reduce the FDA’s evaluation of the application from a standard 10 months to six months from the time of filing. If approved, lovo-cel will be bluebird bio’s third ex-vivo gene therapy and its second FDA approval for an inherited haemoglobin deficiency. This builds on the company’s more than ten years of pioneering work in gene therapy.
• In March 2023, In order to advance a Phase I clinical trial that will assess the safety and viability of the Company’s lead clinical candidate motixafortide to mobilise CD34+ hematopoietic stem cells (HSCs) for gene therapies in SCD, BioLineRx Ltd. has partnered with Washington University School of Medicine in St. Louis.
• In November 2022, For its NRF2 Activator initiative, C4X Discovery Holdings plc and AstraZeneca entered into an exclusive worldwide licencing arrangement valued up to $402 million.
• In October 2022, Global Blood Therapeutics, Inc. (GBT), a biopharmaceutical business devoted to the research, development, and delivery of game-changing therapies that bring hope to underserved patient communities starting with sickle cell disease (SCD), was acquired by Pfizer, it was revealed.
• In March 2022, To assess the long-term safety of inclacumab delivered to SCD patients who had taken part in an Inclacumab clinical trial, GBT started an open-label extension research. This investigation is an open-label study to assess the security of long-term inclacumab therapy in SCD patients. Participants in this trial will have finished an inclacumab study previously.

Sickle Cell Disease Overview

Haemoglobin, the protein that delivers oxygen throughout the body, is impacted by a series of genetic red blood cell abnormalities known as sickle cell disease (SCD). Round and healthy red blood cells travel via tiny blood channels to deliver oxygen to every region of the body. When a person has SCD, their haemoglobin is defective, which makes the red blood cells hard and sticky, giving them the appearance of the farm implement known as a “sickle.”

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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
• Inclacumab: Pfizer
• Exagamglogene autotemcel: CRISPR Therapeutics/Vertex Pharmaceuticals
• Mitapivat: Agios Pharmaceuticals
• Canakinumab: Novartis
• 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 – CRISPR therapeutics, Bluebird Bio, Pfizer, Novo Nordisk, Agios Pharmaceuticals, Alexion Pharmaceuticals, Takeda, Prolong Pharmaceuticals, Roche, Beam Therapeutics, Editas Medicine, Sangamo Therapeutics, Bellicum Pharmaceuticals, Invenux, EpiDestiny, Hillhurst Biopharmaceuticals, CSL Behring, Fulcrum Therapeutics, Sana Biotechnology, 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, Pfizer, CRISPR Therapeutics/Vertex Pharmaceuticals, Agios Pharmaceuticals, Novartis, 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, Inclacumab, Exagamglogene autotemcel, Mitapivat, Canakinumab, 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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