“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 companies working in the treatment market are 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
Emerging Sickle Cell Disease 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 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). The trial is currently active with 12 participants and is anticipated to get completed by May2026
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)
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, ex vivo CRISPR/Cas9 gene-edited therapy for the treatment of severe sickle cell disease (SCD)
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.
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Emerging Sickle Cell Disease Drugs Under Different Phases of Clinical Development Include:
SG418: Sana Biotechnology
BEAM-102: Beam Therapeutics
CSL889: CSL Behring
BPX-501 T cells: Bellicum Pharmaceuticals
GPH101: Graphite Bio
BIVV003: Sangamo Therapeutics
EPI01: Novo Nordisk
Etavopivat: Forma therapeutics
Mitapivat: Agios Pharmaceuticals
Inclacumab: Global Blood Therapeutics, Inc.
CTX001: Vertex Pharmaceuticals
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
Products have been categorized under various Molecule types, such as
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 – Agios Pharmaceuticals, Bristol-Myers Squibb, CRISPR Therapeutics, editas Medicine, Emmaus Life Sciences, Inc., Global Blood Therapeutics, Inc., Medunic USA Inc., Novartis AG, Novo Nordisk, Vifor Pharma, 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, rising demand for better treatment, and growing health awareness are some of the important factors that are fueling the Sickle Cell Disease Market.
Sickle Cell Disease Pipeline Market Barriers
However, high cost of treatment, lack of FDA approved treatment and other factors are creating obstacles in the Sickle Cell Disease Market growth.
Scope of Sickle Cell Disease Pipeline Drug Insight
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
Sickle Cell Disease Report Introduction
Sickle Cell Disease Executive Summary
Sickle Cell Disease Overview
Sickle Cell Disease- Analytical Perspective In-depth Commercial Assessment
Sickle Cell Disease Pipeline Therapeutics
Sickle Cell Disease Late Stage Products (Phase II/III)
Sickle Cell Disease Mid Stage Products (Phase II)
Sickle Cell Disease Early Stage Products (Phase I)
Sickle Cell Disease Preclinical Stage Products
Sickle Cell Disease Therapeutics Assessment
Sickle Cell Disease Inactive Products
Company-University Collaborations (Licensing/Partnering) Analysis
Sickle Cell Disease Key Companies
Sickle Cell Disease Key Products
Sickle Cell Disease Unmet Needs
Sickle Cell Disease Market Drivers and Barriers
Sickle Cell Disease Future Perspectives and Conclusion
Sickle Cell Disease Analyst Views
*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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