Ultra Market Research | North America Sickle Cell Disease Treatment Market
North America Sickle Cell Disease Treatment Market
Report ID : 871
Category : Pharmaceuticals,North-America
No Of Pages : 176
Published on: November 2024
Status: Published
Format :
Key Question Answer
Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major players financial performance.
Evaluate strategies adopted by major players.
Recommendations
North America Sickle Cell Disease Treatment Market
Introduction
The North America sickle cell disease market focuses on therapies and solutions for managing and treating sickle cell anemia, a genetic disorder causing abnormal hemoglobin production. This leads to misshapen red blood cells that impair oxygen transportation, causing pain, anemia, and organ damage. The growing awareness of sickle cell disease, advancements in gene therapies, and the introduction of targeted therapies have catalyzed the market's expansion. In 2023, the SCD treatment market North America was valued at approximately $1.8 billion, with the United States accounting for a significant share. A robust drug pipeline and investments in innovative approaches like gene editing underline the market's future potential.
Segmentation
By Therapeutics
Disease-Modifying Drugs
Hydroxyurea
L-glutamine
Others
Gene Therapies
CRISPR-Cas9 Therapies
Lentiviral Vector Therapies
Curative Therapies
Hematopoietic Stem Cell Transplantation
Bone Marrow Transplants
By Diagnostics
Screening Tests
Newborn Screening
Prenatal Testing
Blood Tests
Imaging Techniques
Doppler Ultrasound
Magnetic Resonance Imaging (MRI)
Others
By End-Users
Hospitals
Specialty Care Centers
Emergency Departments
Clinics
Hematology Clinics
Genetic Counseling Centers
Research Institutes
Academic Research Labs
Clinical Trial Centers
By Distribution Channels
Hospital Pharmacies
Inpatient Pharmacies
Specialty Pharmacies
Retail Pharmacies
Chain Pharmacies
Standalone Pharmacies
Online Pharmacies
List of Market Players
Novartis AG (Switzerland)
Global Blood Therapeutics (United States)
Vertex Pharmaceuticals (United States)
Editas Medicine (United States)
CRISPR Therapeutics (Switzerland)
Sangamo Therapeutics (United States)
bluebird bio (United States)
Pfizer Inc. (United States)
Sanofi (France)
Emmaus Life Sciences (United States)
Acceleron Pharma (United States)
Bioverativ (United States)
Editas Medicine (United States)
Merck & Co. (United States)
Celgene Corporation (United States)
Drivers
The North America SCD market is driven by increasing prevalence of sickle cell disease, particularly in populations of African and Mediterranean descent. Government initiatives, such as newborn screening programs, have improved early detection rates, facilitating timely treatment. The rapid development of gene therapies and disease-modifying drugs, including hydroxyurea and l-glutamine, has provided patients with long-term benefits. Investment in research and development (R&D) by key players, coupled with favorable regulatory policies, has accelerated the approval of innovative treatments. Furthermore, collaborations between pharmaceutical companies and academic institutions contribute to new discoveries in SCD treatment innovations North America.
Restraints
Challenges in the SCD drugs market growth North America include high treatment costs, which limit accessibility for uninsured and underinsured populations. The complex nature of gene therapies requires specialized infrastructure and expertise, posing implementation barriers in smaller clinics. Additionally, disparities in healthcare access across North America affect the adoption of advanced diagnostics and treatments, particularly in rural regions. Regulatory bottlenecks and ethical concerns surrounding gene-editing technologies further hinder market growth.
Opportunity
The North America sickle cell disease market has significant opportunities due to the rising demand for personalized medicine and advancements in biotechnology. Expanding awareness campaigns and educational initiatives about disease management create a favorable environment for market players. The growing trend of collaborations among biotech firms, academic researchers, and government organizations opens pathways for affordable treatment solutions. Unmet needs in curative therapies, such as hematopoietic stem cell transplantation, offer scope for innovation. Furthermore, increasing penetration of telemedicine and remote health monitoring services can enhance accessibility to SCD treatment market North America.
Trend
Emerging trends in the SCD therapeutics market North America include the rise of gene-editing technologies like CRISPR-Cas9, which promise curative solutions. AI-driven diagnostic tools are improving precision in identifying disease subtypes and complications. The development of oral disease-modifying drugs has made treatment more convenient, boosting patient adherence. Another trend is the integration of wearable health-monitoring devices, enabling real-time symptom management. Pharmaceutical companies are also adopting value-based pricing models, ensuring affordability while delivering high-quality care.
FAQs about North America Sickle Cell Disease Market
Rising prevalence, gene therapy advancements, and newborn screening initiatives.
Hydroxyurea, gene-editing therapies, and stem cell transplants are leading options.
High treatment costs, healthcare access disparities, and regulatory hurdles.
Novartis, Vertex Pharmaceuticals, and Global Blood Therapeutics.
Adoption of gene-editing technologies, AI diagnostics, and wearable health monitors.
1. Introduction 1.1 Report Description 1.2 Research Methodology 1.2.1 Data Mining 1.2.2 Market Modeling and Forecasting 1.2.3 Data Validation 1.2.4 Industry Analysis 2. Market Dashboard 3. Market Overview 3.1 Market Definition and Scope 3.2 Market Segmentation 3.2.1 By Therapeutics 3.2.2 By Diagnostics 3.2.3 By End-Users 3.2.4 By Distribution Channels 3.3 Market Dynamics 3.3.1 Drivers 3.3.2 Restraints 3.3.3 Opportunities 3.3.4 Challenges 4. North America Sickle Cell Disease Treatment Market by Therapeutics 4.1 Introduction 4.2 Market Size and Growth Rate by Therapeutics (2024-2030) 4.2.1 Disease-Modifying Drugs 4.2.1.1 Hydroxyurea 4.2.1.2 L-glutamine 4.2.1.3 Others 4.2.2 Gene Therapies 4.2.2.1 CRISPR-Cas9 Therapies 4.2.2.2 Lentiviral Vector Therapies 4.2.3 Curative Therapies 4.2.3.1 Hematopoietic Stem Cell Transplantation 4.2.3.2 Bone Marrow Transplants 5. North America Sickle Cell Disease Treatment Market by Diagnostics 5.1 Introduction 5.2 Market Size and Growth Rate by Diagnostics (2024-2030) 5.2.1 Screening Tests 5.2.1.1 Newborn Screening 5.2.1.2 Prenatal Testing 5.2.1.3 Blood Tests 5.2.2 Imaging Techniques 5.2.2.1 Doppler Ultrasound 5.2.2.2 Magnetic Resonance Imaging (MRI) 5.2.2.3 Others 6. North America Sickle Cell Disease Treatment Market by End-Users 6.1 Introduction 6.2 Market Size and Growth Rate by End-Users (2024-2030) 6.2.1 Hospitals 6.2.1.1 Specialty Care Centers 6.2.1.2 Emergency Departments 6.2.2 Clinics 6.2.2.1 Hematology Clinics 6.2.2.2 Genetic Counseling Centers 6.2.3 Research Institutes 6.2.3.1 Academic Research Labs 6.2.3.2 Clinical Trial Centers 7. North America Sickle Cell Disease Treatment Market by Distribution Channels 7.1 Introduction 7.2 Market Size and Growth Rate by Distribution Channels (2024-2030) 7.2.1 Hospital Pharmacies 7.2.1.1 Inpatient Pharmacies 7.2.1.2 Specialty Pharmacies 7.2.2 Retail Pharmacies 7.2.2.1 Chain Pharmacies 7.2.2.2 Standalone Pharmacies 7.2.3 Online Pharmacies 8. Competitive Landscape 8.1 Company Profiles 8.1.1 Novartis AG (Switzerland) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.2 Global Blood Therapeutics (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.3 Vertex Pharmaceuticals (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.4 Editas Medicine (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.5 CRISPR Therapeutics (Switzerland) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.6 Sangamo Therapeutics (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.7 bluebird bio (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.8 Pfizer Inc. (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.9 Sanofi (France) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.10 Emmaus Life Sciences (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.11 Acceleron Pharma (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.12 Bioverativ (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.13 Merck & Co. (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.14 Celgene Corporation (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview
9. Conclusion and Recommendations 10. Appendix 10.1 List of Tables Table North America Sickle Cell Disease Treatment Market Size by Therapeutics (2024-2030) Table Disease-Modifying Drugs Market Size by Sub-type (2024-2030) Table Gene Therapies Market Size by Sub-type (2024-2030) Table Curative Therapies Market Size by Sub-type (2024-2030) Table North America Sickle Cell Disease Treatment Market Size by Diagnostics (2024-2030) Table Screening Tests Market Size by Sub-type (2024-2030) Table Imaging Techniques Market Size by Sub-type (2024-2030) Table North America Sickle Cell Disease Treatment Market Size by End-Users (2024-2030) Table Hospitals Market Size by Sub-type (2024-2030) Table Clinics Market Size by Sub-type (2024-2030) Table Research Institutes Market Size by Sub-type (2024-2030) Table North America Sickle Cell Disease Treatment Market Size by Distribution Channels (2024-2030) Table Hospital Pharmacies Market Size by Sub-type (2024-2030) Table Retail Pharmacies Market Size by Sub-type (2024-2030) 10.2 List of Figures Figure North America Sickle Cell Disease Treatment Market Size and Growth Rate by Therapeutics (2024-2030) Figure Disease-Modifying Drugs Market Growth Rate by Sub-type (2024-2030) Figure Gene Therapies Market Growth Rate by Sub-type (2024-2030) Figure Curative Therapies Market Growth Rate by Sub-type (2024-2030) Figure North America Sickle Cell Disease Treatment Market Size and Growth Rate by Diagnostics (2024-2030) Figure Screening Tests Market Growth Rate by Sub-type (2024-2030) Figure Imaging Techniques Market Growth Rate by Sub-type (2024-2030) Figure North America Sickle Cell Disease Treatment Market Size and Growth Rate by End-Users (2024-2030) Figure North America Sickle Cell Disease Treatment Market Size and Growth Rate by Distribution Channels (2024-2030) Tables Financial Overview of Key Companies Table Novartis AG Sickle Cell Disease Treatment Financial Overview Table Global Blood Therapeutics Sickle Cell Disease Treatment Financial Overview Table Vertex Pharmaceuticals Sickle Cell Disease Treatment Financial Overview Table Editas Medicine Sickle Cell Disease Treatment Financial Overview Table CRISPR Therapeutics Sickle Cell Disease Treatment Financial Overview Table Sangamo Therapeutics Sickle Cell Disease Treatment Financial Overview Table bluebird bio Sickle Cell Disease Treatment Financial Overview Table Pfizer Inc. Sickle Cell Disease Treatment Financial Overview Table Sanofi Sickle Cell Disease Treatment Financial Overview Table Emmaus Life Sciences Sickle Cell Disease Treatment Financial Overview Table Acceleron Pharma Sickle Cell Disease Treatment Financial Overview Table Bioverativ Sickle Cell Disease Treatment Financial Overview Table Merck & Co. Sickle Cell Disease Treatment Financial Overview Table Celgene Corporation Sickle Cell Disease Treatment Financial Overview
Research Methodology
The process of market research at Ultra Market Research is an iterative in application and usually follows following path. Information from secondary used to build data models, then results from data models are validated from primary participants. Then cycle repeats where, according to inputs from primary participants, additional secondary research is done and new Information is again incorporated into data model. The process continues till desired level of Information is not generated
To calculate the market size, the report considers the revenue generated from the sales of Ultra Market Research providers. The revenue generated from the sales of Ultra Market Research has been calculated through primary and secondary research. The report also presents the key players operating in the Ultra Market Research market across the globe identified through secondary research and a corresponding detailed analysis of the top vendors in the market. The market size calculation also includes distribution channel segmentation determined using secondary sources and verified through primary sources.
Secondary Research
The secondary research source that are typically referred to include, but are not limited to:
Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings
Internal and external proprietary databases, relevant patent and regulatory databases
National government documents, statistical databases and market reports
News articles, press releases and web-casts specific to the companies operating in the market
The source for secondary research includes but is not limited to: Factiva, Hoovers and Statista
Inner Circle Represents – Stage of Research Process
Middle Circle Represents – Source of Information
Outer Circle Represents – Information Derived from that source
Primary Research We conduct primary interviews on an ongoing basis with industry participants and commentators in order to validate data and analysis. A typical research interview fulfills the following functions:
It provides first-hand Information on the market size, market trends, growth trends, competitive landscape, future outlook etc.
Helps in validating and strengthening the secondary research findings
Further develops the analysis team’s expertise and market understanding
Primary research involves E-mail interactions, telephonic interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies
The participants who typically take part in such a process include, but are not limited to:
Industry participants: CEOs, VPs, marketing/ distribution channel managers, market intelligence managers and national sales managers
Purchasing managers, technical personnel, distributors and resellers
Outside experts: Investment bankers, valuation experts, research analysts specializing in specific markets
Key opinion leaders specializing in different areas corresponding to different industry distribution channels
Models Where no hard data is available, we use modeling and estimates in order to produce comprehensive data sets. A rigorous methodology is adopted in which the available hard data is cross referenced with the following data distribution channels to produce estimates:
Demographic data: Population split by segment
Macro-economic indicators: GDP, etc.
Industry indicators: Expenditure, distribution channel stage & infrastructure, sector growth and facilities.
Data is then cross checked by the expert panel.
2..1 Company Share Analysis Model
Company share analysis is used to derive the size of North America market. As well as study of revenues of companies for last three to five years also provide the base for forecasting the market size and its growth rate. This model is built in following steps: 2..2 Revenue Based Modeling
Revenue based models can be built in two ways – Top-Down or Bottom-Up irrespective of industry. Market size estimated from company share analysis acts as a validation point for bottom-up approach where as it acts as starting point for top-down approach.
2.1 Research Limitations Inflation is not a part of pricing in this report. Prices of North America Sickle Cell Disease Treatment Market and its derivatives vary in each region and hence similar revenue ratio does not follow for each individual region. The same price for each distribution channel has been taken into account while estimating and forecasting market revenue North Americaly. Regional average price has been considered while breaking down this market by application in each region. This report provides market size of North America Sickle Cell Disease Treatment Market for the past year and forecasts for the next six years. North America Sickle Cell Disease Treatment Market. Market size is given in terms of revenue. Market revenue is defined in USD Million. Market numbers are given on the basis of different North America Sickle Cell Disease Treatment Market categories. Market size and forecasts for each major application is provided in the context of North America market. The numbers provided in this report are derived on the basis of demand for North America Sickle Cell Disease Treatment Market from different application industries in different regions.
