Sickle Cell Disease Pipeline Drugs Analysis Report, 2023: FDA Approvals, Clinical Trials, Therapies, Mechanism of Action, Route of Administration by DelveInsight | Addmedica, Medunik USA, Bristol Myer

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 Hillhurst Biopharmaceuticals, Agios Pharmaceuticals, Novo Nordisk, Afimmune, IHP Therapeutics, Alexion Pharmaceuticals, Prolong Pharmaceuticals, Pfizer, and others, are developing therapies for the Sickle Cell Disease treatment 

• Emerging Sickle Cell Disease therapies in the different phases of clinical trials are- HBI-002, AG-946, EPI01, Epeleuton, IHP-102, ALXN1820, Sanguinate, Inclacumab, and others are expected to have a significant impact on the Sickle Cell Disease market in the coming years.   
• In December 2022, Bluebird Bio Inc. declared that the US FDA has removed a partial clinical hold on its gene therapy studies intended for treating sickle cell disease, allowing the studies to proceed.
• In October 2022, Pfizer Inc. has finalized its acquisition of GBT, a biopharmaceutical company committed to discovering, developing, and delivering transformative treatments for underrepresented patient communities, with a primary focus on sickle cell disease (SCD).
• In June 2022, GBT, Inc. has revealed that the US FDA awarded inclacumab both orphan drug and rare pediatric disease designations for treating sickle cell disease (SCD).
• In March 2022, GBT has commenced an open-label extension study aimed at assessing the extended safety of inclacumab in individuals diagnosed with sickle cell disease (SCD) who were part of a previous Inclacumab clinical trial. This study intends to evaluate the safety aspects associated with the prolonged use of inclacumab in participants with SCD who have already finished a prior study involving this treatment.
• In February 2022, Global Blood Therapeutics Inc. has obtained marketing authorization for Oxbryta to address hemolytic anemia resulting from sickle cell disease (SCD) in both adult and pediatric patients aged 12 years and older. This approval allows for the use of Oxbryta either as a standalone treatment or in combination with hydroxycarbamide.

Sickle Cell Disease Overview

Hemoglobin, the protein that delivers oxygen throughout the body, is impacted by a set of hereditary red blood cell abnormalities known as sickle cell disease (SCD). All regions of the body receive oxygen from healthy red blood cells, which are spherical and travel through tiny blood capillaries. A person with sickle cell disease (SCD) has aberrant hemoglobin, which makes red blood cells sticky and rigid, resembling the C-shaped “sickle” used in agriculture.

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

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

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, Novartis AG, Global Blood Therapeutics Inc., Emmaus Medical Inc., Bluebird Biotechnology, Pfizer Inc., Aruvant Sciences 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.

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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: Hillhurst Biopharmaceuticals, Agios Pharmaceuticals, Novo Nordisk, Afimmune, IHP Therapeutics, Alexion Pharmaceuticals, Prolong Pharmaceuticals, Pfizer, and others
• Key Sickle Cell Disease Therapies: HBI-002, AG-946, EPI01, Epeleuton, IHP-102, ALXN1820, Sanguinate, Inclacumab, 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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