“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
• PF-07209326: Pfizer
• CSL889: CSL Behring
• OTQ923: Novartis
• BPX-501 T cells: Bellicum Pharmaceuticals
• GPH101: Graphite Bio
• Canakinumab: Novartis
• 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 

• 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

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    

• 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.

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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