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Sickle Cell Disease Drug Pipeline Analysis Report 2025

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    Drug Pipelines

  • 200 Pages
  • June 2025
  • Region: Global
  • Expert Market Research
  • ID: 6102685
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 the publisher 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

By Drug Class

  • Small Molecules
  • Monoclonal Antibodies
  • Gene Therapies

By Route of Administration

  • Oral
  • Intravenous
  • Others

Sickle Cell Disease Pipeline Assessment Segmentation, By Phases

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 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 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.

Sickle Cell Disease Emerging Drugs Profile

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?

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Table of Contents

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 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 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 Region16 Terminated or Suspended Pipeline Products