Dublin, April 29, 2025 (GLOBE NEWSWIRE) -- The "Sickle Cell Disease- Pipeline Insight, 2025" has been added to ResearchAndMarkets.com's offering.
The report offers an in-depth look into the sickle cell disease (SCD) pipeline, featuring over 40 companies and more than 50 pipeline drugs across various stages of development. This comprehensive review includes profiles of clinical and nonclinical stage products, therapeutic assessments by product type, developmental stage, route of administration, molecule type, and inactive products in the pipeline.
Sickle Cell Disease (SCD) is characterized by abnormal hemoglobin causing red blood cells to become rigid and sticky, leading to blockages in blood flow and resulting in pain, infections, and organ damage. While the condition is chronic, long-term treatments can help manage its complications. Advances in prenatal screening, diagnostic tools, and therapeutic strategies, such as gene therapy, offer promising avenues for managing SCD.
The report highlights significant market players actively pursuing research and development to mitigate the challenges and harness opportunities in SCD treatment. Key players, such as Pfizer, Novo Nordisk, Agios Pharmaceuticals, and many others, are engaged in innovative approaches to improve therapies for SCD.
Among the emerging drugs, Inclacumab by Pfizer is advancing in Phase III trials. This monoclonal antibody aims to reduce vaso-occlusive crises (VOCs), a severe complication of SCD. Another promising candidate is Mitapivat by Agios Pharmaceuticals, an oral molecule in Phase II/III trials designed to reduce hemoglobin polymerization, potentially circumventing red blood cell sickling.
Additionally, EPI01 from Novo Nordisk focuses on increasing fetal hemoglobin to mitigate cell deformation. This treatment is currently in Phase II, with significant commercial collaboration milestones in place. GSK 4172239 by GlaxoSmithKline is also being investigated in Phase I as an oral drug targeting methylation mechanisms in SCD.
The report classifies pipeline products based on their clinical development phase, with insights into late-stage, mid-stage, and early-phase products, alongside preclinical findings. In terms of administration routes, drugs are explored through oral, intravenous, subcutaneous, and other methods.
The report elaborates on drug mechanisms, clinical studies, approvals, and development activities, including licensing, collaborations, mergers, and other strategic maneuvers in the SCD landscape.
It's evident that significant strides are being made to enhance therapeutic options for SCD patients, guided by the collaborative efforts of several major pharmaceutical companies. The report's insights on therapeutic advancements, unmet needs, and emerging trends solidify its value to stakeholders seeking to drive innovation and improve SCD patient outcomes.
Key strategic questions addressed in the report include the identification of companies and drugs in development, phased advancements, groundbreaking collaborations, recent drug trends, and ongoing clinical trials. The report also details pivotal regulatory designations awarded to emerging therapies.
Main key players pursuing breakthroughs in SDC treatment include CRISPR Therapeutics, Bluebird Bio, Alexion Pharmaceuticals, and others. Key products in development are CTX001, LentiGlobin BB305, Etavopivat, ALXN1820, TAK-755, and more.
Company Coverage:
- CRISPR therapeutics
- Bluebird Bio
- Pfizer
- Novo Nordisk
- Agios Pharmaceuticals
- Alexion Pharmaceuticals
- Takeda
- Prolong Pharmaceuticals
- Roche
- Beam Therapeutics
- Editas Medicine
- Sangamo Therapeutics
- Bellicum Pharmaceuticals
- Invenux
- EpiDestiny
- Hillhurst Biopharmaceuticals
- CSL Behring
- Fulcrum Therapeutics
- Sana Biotechnology
Key Topics Covered:
Sickle Cell Disease: Overview
- Introduction
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Treatment
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Sickle Cell Disease- The Publisher's Analytical Perspective
Late Stage Products (Phase III)
- Comparative Analysis
Inclacumab: Pfizer
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
EPI01 (NDec): Novo Nordisk A/S
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I)
- Comparative Analysis
Preclinical and Discovery Stage Products
- Comparative Analysis
SG418: Sana Biotechnology
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative 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
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/dcaxnq
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