Sickle Cell Disease Overview

Sickle Cell Disease is a group of inherited red blood cell disorders that affects hemoglobin, the protein that carries oxygen through the body. Sickle cells are destroyed rapidly in the bodies of people with the disease, causing anemia which is why it is commonly known as sickle cell anemia. The sickle cells also block the flow of blood through vessels, resulting in lung tissue damage that causes acute chest syndrome, pain episodes, stroke, priapism and damage to the spleen, kidneys and liver

“Sickle Cell Disease Pipeline Insight, 2022“ 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, Cellectis, Pfizer, CSL Behring, Global Blood Therapeutics, Inc., Novartis, Graphite Bio, Aruvant Sciences, 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, TALGlobin01, PF-07209326, CSL889, GBT021601, OTQ923, GPH101, ARU-1801, 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 2020, CRISPR Therapeutics and Vertex Pharmaceuticals announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to CTX001, an investigational, autologous, exvivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD).
• In May 2022, Vertex pharmaceuticals in collaboration with CRISPR therapeutics initiated a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001) 

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 

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

• SG418: Sana Biotechnology
• TALGlobin01: Cellectis
• PF-07209326: Pfizer
• CSL889: CSL Behring
• GBT021601: Global Blood Therapeutics, Inc.
• OTQ923: Novartis
• GPH101: Graphite Bio
• ARU-1801: Aruvant Sciences
• 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 Sickle Cell Disease treatment 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 Sickle Cell Disease prevalence
• Rising demand for better Sickle Cell Disease treatment
• Growing health awareness

Sickle Cell Disease Pipeline Market Barriers

• High cost of Sickle Cell Disease treatment 
• Lack of FDA approved treatment

Scope of Sickle Cell Disease Pipeline Drug Insight    

• Coverage: Global
• Key Sickle Cell Disease Companies: Sana Biotechnology, Cellectis, Pfizer, CSL Behring, Global Blood Therapeutics, Inc., Novartis, Graphite Bio, Aruvant Sciences, Forma therapeutics, Agios Pharmaceuticals, Global Blood Therapeutics, Inc., Vertex Pharmaceuticals, and others
• Key Sickle Cell Disease Therapies: SG418, TALGlobin01, PF-07209326, CSL889, GBT021601, OTQ923, GPH101, ARU-1801, 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

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