Sickle Cell Disease Drug Pipeline Analysis Report 2025
Description
Sickle cell disease (SCD) is a genetic disorder characterized by abnormal hemoglobin, leading to distorted red blood cells that can cause severe pain and organ damage. The disease affects around 300,000 individuals worldwide annually. Current treatment options, including blood transfusions and hydroxyurea, are limited in their effectiveness and do not offer a cure, highlighting a critical unmet clinical need. The growing focus on gene therapy, CRISPR-based treatments, and novel drug candidates is expected to drive significant growth in the SCD drug pipeline in the coming years.
Report Coverage
The Sickle Cell Disease Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into sickle cell disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for sickle cell disease. The sickle cell disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The sickle cell disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with sickle cell disease treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to sickle cell disease.
Sickle Cell Disease Drug Pipeline Outlook
Sickle Cell Disease (SCD) is a genetic blood disorder characterized by abnormal hemoglobin, which causes red blood cells to become rigid and crescent shaped. This disrupts their ability to carry oxygen, leading to blockages in blood flow, pain, and organ damage. SCD occurs when a person inherits sickle hemoglobin from both parents, causing both inherited genes to result in defective red blood cells.
Sickle cell disease treatment focuses on managing symptoms and preventing complications. These include pain management, blood transfusions, and hydroxyurea, which increases fetal hemoglobin levels. Bone marrow or stem cell transplants offer a potential cure, though they are not suitable for all patients. Recent advancements in gene therapy aim to provide a lasting solution by correcting genetic defects.
Sickle Cell Disease Epidemiology
The global sickle cell disease drug pipeline is advancing as the disease continues to impact diverse populations. India accounts for about 14.5% of the world’s sickle cell disease newborns, with over 42,000 cases annually. In the United States, approximately 100,000 individuals are affected, with an incidence rate of 1 in 365 among Black or African American infants and 1 in 16,300 among Hispanic American infants.
Sickle Cell Disease Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of sickle cell disease drug candidates based on several segmentations, including:
By Phase
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis , phase II covers a major share of the total sickle cell disease clinical trials.
Sickle Cell Disease Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the sickle cell disease pipeline analysis include small molecules, monoclonal antibodies, and gene therapies. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for sickle cell disease.
Sickle Cell Disease Clinical Trials Therapeutic Assessment – Competitive Dynamics
The EMR report for the sickle cell disease report insights includes the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in sickle cell disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for sickle cell disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of sickle cell disease drug candidates.
Drug: Etavopivat
A clinical study, sponsored by Novo Nordisk A/S, is being conducted to evaluate the effectiveness of Etavopivat in reducing vaso-occlusive crises (VOCs) in individuals with sickle cell disease. The Phase 3 trial will assess its impact on organ damage, exercise tolerance, and fatigue. The study, involving around 400 participants, is expected to conclude by September 2028.
Drug: Voxelotor
The Phase 2 clinical study, sponsored by Inova Health Care Services, is assessing the impact of voxelotor on physical function in sickle cell disease patients. The study is designed to evaluate improvements in performance following six months of treatment and is expected to be completed by June 2025.
Drug: Biotin Labeled Red Blood Cells
Sponsored by Emory University, this Phase 1 clinical trial is examining the survival of transfused red blood cells (RBCs) in sickle cell disease (SCD) patients receiving chronic transfusion therapy (CTT). The study, which involves around 40 participants, is expected to be completed by May 2025. Its objective is to identify factors affecting RBC survival and enhance stroke prevention in SCD.
Reasons To Buy This Report
The Sickle Cell Disease Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for sickle cell disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within sickle cell disease pipeline insights.
Key Questions Answered in the Sickle Cell Disease – Pipeline Insight Report
Sickle Cell Disease Treatment Market
Global Clinical Trials Market
Report Coverage
The Sickle Cell Disease Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into sickle cell disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for sickle cell disease. The sickle cell disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The sickle cell disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with sickle cell disease treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to sickle cell disease.
Sickle Cell Disease Drug Pipeline Outlook
Sickle Cell Disease (SCD) is a genetic blood disorder characterized by abnormal hemoglobin, which causes red blood cells to become rigid and crescent shaped. This disrupts their ability to carry oxygen, leading to blockages in blood flow, pain, and organ damage. SCD occurs when a person inherits sickle hemoglobin from both parents, causing both inherited genes to result in defective red blood cells.
Sickle cell disease treatment focuses on managing symptoms and preventing complications. These include pain management, blood transfusions, and hydroxyurea, which increases fetal hemoglobin levels. Bone marrow or stem cell transplants offer a potential cure, though they are not suitable for all patients. Recent advancements in gene therapy aim to provide a lasting solution by correcting genetic defects.
Sickle Cell Disease Epidemiology
The global sickle cell disease drug pipeline is advancing as the disease continues to impact diverse populations. India accounts for about 14.5% of the world’s sickle cell disease newborns, with over 42,000 cases annually. In the United States, approximately 100,000 individuals are affected, with an incidence rate of 1 in 365 among Black or African American infants and 1 in 16,300 among Hispanic American infants.
Sickle Cell Disease Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of sickle cell disease drug candidates based on several segmentations, including:
By Phase
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
- Small Molecules
- Monoclonal Antibodies
- Gene Therapies
- Oral
- Intravenous
- Others
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis , phase II covers a major share of the total sickle cell disease clinical trials.
Sickle Cell Disease Pipeline Assessment Segmentation, By Drug Classes
The drug molecule categories covered under the sickle cell disease pipeline analysis include small molecules, monoclonal antibodies, and gene therapies. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for sickle cell disease.
Sickle Cell Disease Clinical Trials Therapeutic Assessment – Competitive Dynamics
The EMR report for the sickle cell disease report insights includes the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in sickle cell disease clinical trials:
- Pfizer
- Novo Nordisk A/S
- Novartis Pharmaceuticals
- Biogen
- Hoffmann-La Roche
- Kamau Therapeutics
- Fulcrum Therapeutics
- CorrectSequence Therapeutics Co., Ltd.
- Beam Therapeutics Inc.
- Agios Pharmaceuticals, Inc.
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for sickle cell disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of sickle cell disease drug candidates.
Drug: Etavopivat
A clinical study, sponsored by Novo Nordisk A/S, is being conducted to evaluate the effectiveness of Etavopivat in reducing vaso-occlusive crises (VOCs) in individuals with sickle cell disease. The Phase 3 trial will assess its impact on organ damage, exercise tolerance, and fatigue. The study, involving around 400 participants, is expected to conclude by September 2028.
Drug: Voxelotor
The Phase 2 clinical study, sponsored by Inova Health Care Services, is assessing the impact of voxelotor on physical function in sickle cell disease patients. The study is designed to evaluate improvements in performance following six months of treatment and is expected to be completed by June 2025.
Drug: Biotin Labeled Red Blood Cells
Sponsored by Emory University, this Phase 1 clinical trial is examining the survival of transfused red blood cells (RBCs) in sickle cell disease (SCD) patients receiving chronic transfusion therapy (CTT). The study, which involves around 40 participants, is expected to be completed by May 2025. Its objective is to identify factors affecting RBC survival and enhance stroke prevention in SCD.
Reasons To Buy This Report
The Sickle Cell Disease Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for sickle cell disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into market trends, regulatory environments, and potential growth opportunities within sickle cell disease pipeline insights.
Key Questions Answered in the Sickle Cell Disease – Pipeline Insight Report
- Which companies/institutions are leading the sickle cell disease drug development?
- What is the efficacy and safety profile of sickle cell disease pipeline drugs?
- Which company is leading the sickle cell disease pipeline development activities?
- What is the current sickle cell disease commercial assessment?
- What are the opportunities and challenges present in the sickle cell disease drug pipeline landscape?
- What is the efficacy and safety profile of sickle cell disease pipeline drugs?
- Which company is conducting major trials for sickle cell disease drugs?
- Which companies/institutions are involved in sickle cell disease collaborations aimed at providing enhanced therapeutic alternatives for patients?
- What are the geographies covered for clinical trials in sickle cell disease?
Sickle Cell Disease Treatment Market
Global Clinical Trials Market
Table of Contents
200 Pages
- 1 Preface
- 1.1 Introduction
- 1.2 Objectives of the Study
- 1.3 Research Methodology & Assumptions
- 2 Executive Summary
- 3 Overview of Sickle Cell Disease
- 3.1 Signs and Symptoms
- 3.2 Causes
- 3.3 Risk Factors
- 3.4 Diagnosis
- 3.5 Treatment
- 4 Patient Profile: Sickle Cell Disease
- 4.1 Patient Profile Overview
- 4.2 Patient Psychology and Emotional Impact Factors
- 4.3 Risk Assessment and Treatment Success Rate
- 5 Sickle Cell Disease: Epidemiology Snapshot
- 5.1 Sickle Cell Disease Incidence by Key Markets
- 5.2 Sickle Cell Disease – Patients Seeking Treatment in Key Markets
- 6 Sickle Cell Disease: Market Dynamics
- 6.1 Market Drivers and Constraints
- 6.2 SWOT Analysis
- 7 Sickle Cell Disease: Key Facts Covered
- 7.1 Top Countries Contributing to Clinical Trials in Asia-Pacific
- 7.2 Top Countries Contributing to Clinical Trials in Europe
- 7.3 Top Countries Contributing to Clinical Trials in North America
- 7.4 Top Countries Contributing to Clinical Trials in Other Regions
- 8 Sickle Cell Disease, Drug Pipeline Assessment
- 8.1 Assessment by Treatment Type
- 8.2 Assessment by Route of Administration
- 8.3 Assessment by Drug Class
- 9 EMR Drug Pipeline Comparative Analysis
- 9.1 List of Sickle Cell Disease Pipeline Drugs
- 9.1.1 By Company
- 9.1.2 By Phase
- 9.1.3 By Indication
- 9.1.4 By Trial Status
- 9.1.5 By Funder Type
- 9.2 EMR Attribute Scoring Analysis of Pipeline Drugs (Top Drugs)
- 10 Sickle Cell Disease Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
- 10.1 Comparative Analysis for Late-Stage Drugs
- 10.1.1 Study Type
- 10.1.2 Recruitment Status
- 10.1.3 Company
- 10.1.4 Funder Type
- 10.2 Product Level Analysis*
- 10.2.1 Biological: Crizanlizumab
- 10.2.1.1 Product Description
- 10.2.1.2 Trial ID
- 10.2.1.3 Sponsor Name
- 10.2.1.4 Study Type
- 10.2.1.5 Drug Class
- 10.2.1.6 Eligibility Criteria
- 10.2.1.7 Study Record Dates
- 10.2.1.7.1 First Submitted
- 10.2.1.7.2 First Posted
- 10.2.1.7.3 Last Update Posted
- 10.2.1.7.4 Last Verified
- 10.2.1.8 Indication
- 10.2.1.9 Study Design
- 10.2.1.10 Recruitment Status
- 10.2.1.11 Enrollment (Estimated)
- 10.2.1.12 Location Countries
- 10.2.1.13 Recent Results
- 10.2.2 Drug: Etavopivat
- 10.2.3 Genetic: bb1111
- 10.2.4 Other Drugs
- 11 Sickle Cell Disease Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
- 11.1 Comparative Analysis for Mid-Stage Drugs
- 11.1.1 Study Type
- 11.1.2 Recruitment Status
- 11.1.3 Company
- 11.1.4 Funder Type
- 11.2 Product Level Analysis*
- 11.2.1 Drug: Voxelotor
- 11.2.1.1 Product Description
- 11.2.1.2 Trial ID
- 11.2.1.3 Sponsor Name
- 11.2.1.4 Study Type
- 11.2.1.5 Drug Class
- 11.2.1.6 Eligibility Criteria
- 11.2.1.7 Study Record Dates
- 11.2.1.7.1 First Submitted
- 11.2.1.7.2 First Posted
- 11.2.1.7.3 Last Update Posted
- 11.2.1.7.4 Last Verified
- 11.2.1.8 Indication
- 11.2.1.9 Study Design
- 11.2.1.10 Recruitment Status
- 11.2.1.11 Enrollment (Estimated)
- 11.2.1.12 Location Countries
- 11.2.1.13 Recent Results
- 11.2.2 Biological: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- 11.2.3 Drug: Etavopivat tablets
- 11.2.4 Other Drugs
- 12 Sickle Cell Disease Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
- 12.1 Comparative Analysis for Early-Stage Drugs
- 12.1.1 Study Type
- 12.1.2 Recruitment Status
- 12.1.3 Company
- 12.1.4 Funder Type
- 12.2 Product Level Analysis*
- 12.2.1 Biological: OTQ923
- 12.2.1.1 Product Description
- 12.2.1.2 Trial ID
- 12.2.1.3 Sponsor Name
- 12.2.1.4 Study Type
- 12.2.1.5 Drug Class
- 12.2.1.6 Eligibility Criteria
- 12.2.1.7 Study Record Dates
- 12.2.1.7.1 First Submitted
- 12.2.1.7.2 First Posted
- 12.2.1.7.3 Last Update Posted
- 12.2.1.7.4 Last Verified
- 12.2.1.8 Indication
- 12.2.1.9 Study Design
- 12.2.1.10 Recruitment Status
- 12.2.1.11 Enrollment (Estimated)
- 12.2.1.12 Location Countries
- 12.2.2 Drug: Biotin Labeled Red Blood Cells
- 12.2.3 Other Drugs
- 13 Sickle Cell Disease Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
- 13.1 Comparative Analysis for Preclinical and Discovery Stage Drugs
- 13.1.1 Study Type
- 13.1.2 Recruitment Status
- 13.1.3 Company
- 13.1.4 Funder Type
- 13.2 Product Level Analysis*
- 13.2.1 Genetic: CS-206
- 13.2.1.1 Product Description
- 13.2.1.2 Trial ID
- 13.2.1.3 Sponsor Name
- 13.2.1.4 Study Type
- 13.2.1.5 Drug Class
- 13.2.1.6 Eligibility Criteria
- 13.2.1.7 Study Record Dates
- 13.2.1.7.1 First Submitted
- 13.2.1.7.2 First Posted
- 13.2.1.7.3 Last Update Posted
- 13.2.1.7.4 Last Verified
- 13.2.1.8 Indication
- 13.2.1.9 Study Design
- 13.2.1.10 Recruitment Status
- 13.2.1.11 Enrollment (Estimated)
- 13.2.1.12 Location Countries
- 13.2.2 Other Drugs
- 14 Sickle Cell Disease, Key Drug Pipeline Companies
- 14.1 Pfizer
- 14.1.1 Company Snapshot
- 14.1.2 Pipeline Product Portfolio
- 14.1.3 Financial Analysis
- 14.1.4 Recent News and Developments
- 14.2 Novo Nordisk A/S
- 14.2.1 Company Snapshot
- 14.2.2 Pipeline Product Portfolio
- 14.2.3 Financial Analysis
- 14.2.4 Recent News and Developments
- 14.3 Novartis Pharmaceuticals
- 14.3.1 Company Snapshot
- 14.3.2 Pipeline Product Portfolio
- 14.3.3 Financial Analysis
- 14.3.4 Recent News and Developments
- 14.4 Biogen
- 14.4.1 Company Snapshot
- 14.4.2 Pipeline Product Portfolio
- 14.4.3 Financial Analysis
- 14.4.4 Recent News and Developments
- 14.5 Hoffmann-La Roche
- 14.5.1 Company Snapshot
- 14.5.2 Pipeline Product Portfolio
- 14.5.3 Financial Analysis
- 14.5.4 Recent News and Developments
- 14.6 Kamau Therapeutics
- 14.6.1 Company Snapshot
- 14.6.2 Pipeline Product Portfolio
- 14.6.3 Financial Analysis
- 14.6.4 Recent News and Developments
- 14.7 Fulcrum Therapeutics
- 14.7.1 Company Snapshot
- 14.7.2 Pipeline Product Portfolio
- 14.7.3 Financial Analysis
- 14.7.4 Recent News and Developments
- 14.8 CorrectSequence Therapeutics Co., Ltd.
- 14.8.1 Company Snapshot
- 14.8.2 Pipeline Product Portfolio
- 14.8.3 Financial Analysis
- 14.8.4 Recent News and Developments
- 14.9 Beam Therapeutics Inc.
- 14.9.1 Company Snapshot
- 14.9.2 Pipeline Product Portfolio
- 14.9.3 Financial Analysis
- 14.9.4 Recent News and Developments
- 14.10 Agios Pharmaceuticals, Inc.
- 14.10.1 Company Snapshot
- 14.10.2 Pipeline Product Portfolio
- 14.10.3 Financial Analysis
- 14.10.4 Recent News and Developments
- 15 Regulatory Framework for Drug Approval, By Region
- 16 Terminated or Suspended Pipeline Products
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