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Sickle Cell Disease Pipeline Insights 2024 | Clinical Trials, Latest Approvals, Treatment Outlook, Competitive Landscape, Companies | Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharma

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Sickle Cell Disease Pipeline Insights 2024 | Clinical Trials, Latest Approvals, Treatment Outlook, Competitive Landscape, Companies | Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharma

June 03
10:05 2024
Sickle Cell Disease Pipeline Insights 2024 | Clinical Trials, Latest Approvals, Treatment Outlook, Competitive Landscape, Companies | Novartis, Agios Pharmaceuticals, Forma therapeutics, Vertex Pharma
Sickle Cell Disease Pipeline Insights
DelveInsight’s report, “Sickle Cell Disease (SCD) – Pipeline Insight, 2024,” offers in-depth analysis on over 40 companies and more than 50 pipeline drugs within the Sickle cell disease landscape. The report includes detailed profiles of pipeline drugs, encompassing both clinical and nonclinical stage products. Moreover, it provides a comprehensive assessment of therapeutics based on product type, stage, route of administration, and molecule type.

As per DelveInsight’s assessment, about 40+ key pharma and biotech companies are working on 50+ drugs in the Sickle Cell Disease therapeutics landscape based on different Routes of Administration (RoA), Mechanism of Action (MoA), and molecule types. Several of the therapies are in the advanced stages of clinical development and are expected to launch in the coming years.

Sickle Cell Disease Overview

Sickle cell disease encompasses a group of inherited conditions impacting red blood cells, with its most severe form known as sickle cell anemia. In individuals with this condition, sickle cells undergo rapid destruction, leading to anemia, a hallmark of sickle cell anemia. Additionally, these abnormally shaped cells obstruct blood flow in vessels, contributing to complications such as lung tissue damage, acute chest syndrome, pain crises, stroke, priapism, and organ damage to the spleen, kidneys, and liver. Despite being a lifelong and serious condition, long-term management strategies can mitigate many associated challenges. The disease primarily affects hemoglobin, the molecule responsible for oxygen delivery to cells throughout the body. Hemoglobin S, an aberrant hemoglobin variant, is pivotal in distorting red blood cells into the characteristic sickle or crescent shape. The underlying cause of sickle cell disease lies in a mutation within the hemoglobin-Beta gene located on a chromosome.

Sickle Cell Disease Pipeline Insight, 2024” report by DelveInsight outlines a comprehensive assessment of the present clinical/non-clinical development activities and growth prospects across the Sickle Cell Disease Market. 

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 Analysis

The report provides insights into:

  • The report provides detailed insights into the emerging therapies for the treatment of Sickle Cell Disease and the aggregate therapies developed by major pharma companies.

  • It accesses the different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of clinical development.

  • It outlines the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

  • The report evaluates the drugs that are 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. 

  • It navigates the major collaborations (company-company collaborations and company-academia collaborations), licensing agreements, financing details, data presentation by the pharma giants, and regulatory approval in 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.  

Analysis of Emerging Therapies by Phases

The report covers the emerging 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

Sickle Cell Disease (SCD) 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


Molecule Type

Products have been categorized under various Molecule types, such as

  • Antisense oligonucleotide

  • Gene therapy

  • Hormones

  • Neuropeptides

  • Oligonucleotides

  • Small Molecule

  • Triglyceride

Learn How the Ongoing Clinical & Commercial Activities will Affect the Sickle Cell Disease Therapeutic Segment @

Sickle Cell Disease Therapeutics Landscape

There are approx. 40+ key companies developing therapies for Sickle Cell Disease (SCD). Currently,  Vertex Pharmaceuticals is leading the therapeutics market with its Sickle Cell Disease (SCD) drug candidates in the most advanced stage of clinical development.

The Leading Companies in the Sickle Cell Disease (SCD) Therapeutics Market Include:

Agios Pharmaceuticals, Alfasigma, Aruvant Sciences, bluebird bio, Cellectis, CSL Behring, Editas Medicine, ExCellThera, Forma therapeutics, Gamida Cell, Global Blood Therapeutics, Inc., Graphite Bio, KM Biologics, Novartis, Novo Nordisk, Pfizer, Sana Biotechnology, Takeda, Vertex Pharmaceuticals, and many more.

Sickle Cell Disease (SCD) Emerging and Marketed Drugs Covered in the Report Include:

  • BPX-501 T cells: Bellicum Pharmaceuticals

  • Canakinumab: Novartis

  • EPI01: Novo Nordisk

  • CTX001: Vertex Pharmaceuticals

And Many More

Request the Sample PDF to Get a Better Understanding of the Emerging Drugs and Key Companies @

Table of Contents

1. Report Introduction

2. Executive Summary

3. Sickle Cell Disease Current Treatment Patterns

4. Sickle Cell Disease – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

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

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

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

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Sickle Cell Disease Discontinued Products

13. Sickle Cell Disease Product Profiles

14. Key Companies in the Sickle Cell Disease Market

15. Key Products in the Sickle Cell Disease Therapeutics Segment

16. Dormant and Discontinued Products

17. Sickle Cell Disease Unmet Needs

18. Sickle Cell Disease Future Perspectives

19. Sickle Cell Disease Analyst Review  

20. Appendix

21. Report Methodology

Download Sample PDF to Explore the Key Offerings of the Report @

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kritika Rehani
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States