Sickle Cell Disease Overview

Hemoglobin, the protein that delivers oxygen throughout the body, is impacted by Sickle Cell Disease, a group of genetic red blood cell abnormalities. It is frequently referred to as sickle cell anaemia because sickle cells are rapidly destroyed in the bodies of those who have the condition, leading to anaemia. Additionally, sickle cells prevent blood from flowing through capillaries, which damages lung tissue and leads to acute chest syndrome, pain episodes, stroke, priapism, and damage to the spleen, kidneys, and liver.

“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.

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.

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 Key players such as – Sana Biotechnology, Beam Therapeutics, Pfizer, CSL Behring, Novartis, Graphite Bio, Sangamo Therapeutics, Forma therapeutics, Agios Pharmaceuticals, Global Blood Therapeutics, Inc., Vertex Pharmaceuticals, and others, are developing therapies for the Sickle Cell Disease treatment 

• Sickle Cell Disease Emerging therapies such as – SG418, BEAM-102, PF-07209326, CSL889, OTQ923, GPH101, BIVV003, Etavopivat, Mitapivat, Inclacumab, CTX001, and others are expected to have a significant impact on the Sickle Cell Disease market in the coming years.   
• In September 2022, Vertex Pharmaceuticals Incorporated and CRISPR Therapeutics announced that Vertex has concluded discussions with the US Food and Drug Administration (FDA), and the FDA granted exagamglogeneautotemcel (exa-cel) a rolling review for the potential treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT)

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

Molecule Type

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

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

Key companies developing therapies for Sickle Cell Disease  are – Bristol-Myers Squibb Company, Addmedica, Novartis AG, Global Blood Therapeutics, Inc. (Pfizer Inc.), Emmaus Medical, Inc., bluebird bio Inc., Agios Pharmaceuticals, Inc.,and others

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

• Canakinumab: Novartis
• EPI01: Novo Nordisk
• SG418: Sana Biotechnology
• BEAM-102: Beam Therapeutics
• PF-07209326: Pfizer
• CSL889: CSL Behring
• OTQ923: Novartis
• GPH101: Graphite Bio
• BIVV003: Sangamo Therapeutics
• Etavopivat: Forma therapeutics
• Mitapivat: Agios Pharmaceuticals
• Inclacumab: Global Blood Therapeutics, Inc.
• CTX001: Vertex Pharmaceuticals

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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.

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

Sickle Cell Disease Pipeline Market Drivers

• Increasing prevalence
• Rising demand for better treatment
• Growing health awareness

Sickle Cell Disease Pipeline Market Barriers

• High cost of treatment
• Lack of FDA approved treatment

Scope of Sickle Cell Disease Pipeline Drug Insight    

• Coverage: Global
• Key Sickle Cell Disease Companies: Sana Biotechnology, Beam Therapeutics, Pfizer, CSL Behring, Novartis, Graphite Bio, Sangamo Therapeutics, Forma therapeutics, Agios Pharmaceuticals, Global Blood Therapeutics, Inc., Vertex Pharmaceuticals, and others
• Key Sickle Cell Disease Therapies: SG418, BEAM-102, PF-07209326, CSL889, OTQ923, GPH101, BIVV003, Etavopivat, Mitapivat, Inclacumab, CTX001, 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

*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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