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North America Hemoglobinopathies Market Size, Share, Trends: By Diagnosis: Red Blood Cell (RBC) Count Test, Genetic Testing, High Performance Liquid Chromatography (HPLC) Test, Others; By Type; By Therapy; By Treatment; By Distribution channel; By End User; Regional Analysis; Market Dynamics; Supplier Landscape; 2024-2032
North America Hemoglobinopathies Market Outlook
North America hemoglobinopathies market size is expected to grow at a CAGR of 5.10% during the forecast period of 2024-2032, driven by the growing prevalence of sickle cell anemia and thalassemia along with the rising advancements in treatment and diagnostics in the region.
Key Takeaways
- In the United States, around 100,000 individuals are affected by sickle cell disease. It is highly prevalent in African Americans, affecting 1 out of every 365 African American births.
- In December 2023, the FDA approved the first cell-based gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease (SCD) in patients aged 12 years and more.
- In July 2023, the National Institutes of Health awarded a USD 2.6 million grant to a Rice University research team to investigate the efficacy and safety of gene editing technology to treat sickle cell disease.
North America Hemoglobinopathies Market Analysis
Hemoglobinopathies are a group of inherited disorders characterized by the abnormal structure or production of the hemoglobin molecule. Sickle cell anemia and thalassemia are some common types of hemoglobinopathies. In the United States, around 100,000 individuals are affected by sickle cell disease. It is highly prevalent in African Americans, occurring in 1 out of every 365 African American births. The rising prevalence of sickle cell anemia disease is expected to increase demand for effective treatment options, driving North America hemoglobinopathies market growth.
One of the major market trends is the development of novel therapies to combat the growing cases of hemoglobinopathies. Furthermore, the rise in regulatory approvals by health regulatory bodies including United States Food and Drug Administration (FDA) to address the unmet of clinical needs of a large patient base is likely to boost market size in the region. In December 2023, the FDA granted approval to the first cell-based gene therapies, Casgevy and Lyfgenia, to treat sickle cell disease (SCD) in patients aged 12 years and more. Such innovations along with their high acceptance rate is projected to augment the North America hemoglobinopathies market share in the forecast period.
Casgevy, developed by Vertex Pharmaceuticals Incorporated, and a Swiss–American biotechnology CRISPR Therapeutics AG, is the first FDA-approved CRISPR/Cas9 therapy for the treatment of sickle cell disease in patients with recurrent vaso-occlusive crises. On the other hand, Bluebird Bio, Inc.’s Lyfgenia is a cell-based gene therapy that utilizes a lentiviral vector for gene delivery. It is used for the treatment of sickle cell disease in patients showing a history of vaso-occlusive events. The approval of these novel gene therapies which have the potential to cure the inherited disease is anticipated to fuel the North America hemoglobinopathies market demand.
The market is also driven by increased investments from both government and private sectors to support research activities aimed at the advancement of diagnostic methods and treatment of hemoglobinopathies. For instance, in July 2023, the National Institutes of Health awarded a USD 2.6 million grant to a Rice University bioengineer and his research team. The 4-year grant was intended to be used in investigating the efficacy and safety of gene editing technology to treat sickle cell disease. The rise in funding will help in the development of new treatment therapies which will propel the market growth in the region.
North America Hemoglobinopathies Market Segmentation
The report offers a detailed analysis of the market based on the following segments:
| Market Breakup | Categories |
| Diagnosis | Red Blood Cell (RBC) Count Test, Genetic Testing, High Performance Liquid Chromatography (HPLC) Test, Hemoglobin Isoelectric (Hb IEF) Focusing, Hemoglobin Electrophoresis (Hb ELP) Test, Hemoglobin Solubility Test |
| Type | Thalassemia, Sickle Cell Disease, Others |
| Therapy | Monoclonal Antibody Medication, Ace Inhibitors, Hydroxyurea, Others |
| Treatment | Allogeneic Stem Cell Therapy, Autologous Stem Cell Therapy, Syngeneic Stem Cell Therapy |
| Distribution Channel | Hospital Pharmacy, Online Providers, Drug Store and Retail Pharmacy, Others |
| End User | Biopharmaceutical and Biotechnology Companies, Academic and Research Institutes, Others |
| Countries | United States of America, Canada |
North America Hemoglobinopathies Market: Competitor Landscape
The key features of the market report include patent analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:
- Sangamo Therapeutics, Inc.
- Global Blood Therapeutics
- Bluebird bio, Inc.
- Emmaus Life Science Inc.
- Pfizer, Inc.
- Novartis AG
- Prolong Pharmaceuticals, LLC
- Bioverativ Inc.
- Gamida Cell
- Celgene Corporation
Kindly note that this only represents a partial list of companies, and the complete list has been provided in the report.
Key Highlights of the Report
| REPORT FEATURES | DETAILS |
| Base Year | 2023 |
| Historical Period | 2017-2023 |
| Forecast Period | 2024-2032 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Diagnosis |
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| Breakup by Type |
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| Breakup by Therapy |
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| Breakup by Treatment |
|
| Breakup by Distribution channel |
|
| Breakup by End User |
|
| Breakup by Region |
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| Market Dynamics |
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| Supplier Landscape |
|
| Companies Covered |
|
*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
1.4 Research Methodology
2 Executive Summary
3 North America Hemoglobinopathies Market Overview
3.1 North America Hemoglobinopathies Market Historical Value (2017-2023)
3.2 North America Hemoglobinopathies Market Forecast Value (2024-2032)
4 North America Hemoglobinopathies Market Landscape*
4.1 North America Hemoglobinopathies: Developers Landscape
4.1.1 Analysis by Year of Establishment
4.1.2 Analysis by Company Size
4.1.3 Analysis by Region
4.2 North America Hemoglobinopathies: Product Landscape
4.2.1 Analysis by Diagnosis
4.2.2 Analysis by Treatment
4.2.3 Analysis by Type
5 North America Hemoglobinopathies Market Dynamics
5.1 Market Drivers and Constraints
5.2 SWOT Analysis
5.2.1 Strengths
5.2.2 Weaknesses
5.2.3 Opportunities
5.2.4 Threats
5.3 Porter’s Five Forces Analysis
5.3.1 Bargaining Power of Suppliers
5.3.2 Bargaining Power of Buyers
5.3.3 Threat of New Entrants
5.3.4 Threat of Substitutes
5.3.5 Degree of Rivalry
5.4 Key Demand Indicators
5.5 Key Price Indicators
5.6 Industry Events, Initiatives, and Trends
5.7 Value Chain Analysis
6 North America Hemoglobinopathies Market Segmentation (2017-2032)
6.1 North America Hemoglobinopathies Market (2017-2032) by Diagnosis
6.1.1 Market Overview
6.1.2 Red Blood Cell (RBC) Count Test
6.1.3 Genetic Testing
6.1.4 High Performance Liquid Chromatography (HPLC) Test
6.1.5 Hemoglobin Isoelectric (Hb IEF) Focusing
6.1.6 Hemoglobin Electrophoresis (Hb ELP) Test
6.1.7 Hemoglobin Solubility Test
6.1.8 Others
6.2 North America Hemoglobinopathies Market (2017-2032) by Type
6.2.1 Market Overview
6.2.2 Thalassemia
6.2.3 Sickle cell Disease
6.2.4 Others
6.3 North America Hemoglobinopathies Market (2017-2032) by Therapy
6.3.1 Market Overview
6.3.2 Monoclonal Antibody
6.3.3 Ace Inhibitors
6.3.4 Hydroxyurea
6.3.5 Others
6.4 North America Hemoglobinopathies Market (2017-2032) by Treatment
6.4.1 Market Overview
6.4.2 Allogeneic Stem Cell Therapy
6.4.3 Autologic Stem Cell Therapy
6.4.4 Syngeneic Stem Cell Therapy
6.5 North America Hemoglobinopathies Market (2017-2032) by Distribution channel
6.5.1 Market Overview
6.5.2 Hospital Pharmacy
6.5.3 Online Providers
6.5.4 Drug Store and Retail Pharmacy
6.5.5 Others
6.6 North America Hemoglobinopathies Market (2017-2032) by End User
6.6.1 Market Overview
6.6.2 Biopharmaceutical & Biotechnology Companies
6.6.3 Academic & Research Institutes
6.6.4 Others
6.7 North America Hemoglobinopathies Market (2017-2032) by Countries
6.7.1 Market Overview
6.7.2 United States of America
6.7.3 Canada
7 United States of America H Market (2017-2032)
7.1 United State of America Hemoglobinopathies Market (2017-2032) by Diagnosis
7.1.1 Market Overview
7.1.2 Red Blood Cell (RBC) Count Test
7.1.3 Genetic Testing
7.1.4 High Performance Liquid Chromatography (HPLC) Test
7.1.5 Hemoglobin Isoelectric (Hb IEF) Focusing
7.1.6 Hemoglobin Electrophoresis (Hb ELP) Test
7.1.7 Hemoglobin Solubility Test
7.2 United State of America Hemoglobinopathies Market (2017-2032) by Type
7.2.1 Market Overview
7.2.2 Thalassemia
7.2.3 Sickle cell Disease
7.2.4 Others
7.3 United State of America Hemoglobinopathies Market (2017-2032) by Therapy
7.3.1 Market Overview
7.3.2 Monoclonal Antibody
7.3.3 Ace Inhibitors
7.3.4 Hydroxyurea
7.3.5 Others
7.4 United State of America Hemoglobinopathies Market (2017-2032) by Treatment
7.4.1 Market Overview
7.4.2 Allogeneic Stem Cell Therapy
7.4.3 Autologic Stem Cell Therapy
7.4.4 Syngeneic Stem Cell Therapy
7.5 United State of America Hemoglobinopathies Market (2017-2032) by Distribution Channel
7.5.1 Market Overview
7.5.2 Hospital Pharmacy
7.5.3 Online Providers
7.5.4 Drug Store and Retail Pharmacy
7.5.5 Others
7.6 United State of America Hemoglobinopathies Market (2017-2032) by End User
7.6.1 Market Overview
7.6.2 Biopharmaceutical & Biotechnology Companies
7.6.3 Academic & Research Institutes
7.6.4 Others
8 Canada Stem Cell Assay Market (2017-2032)
8.1 Canada Hemoglobinopathies Market (2017-2032) by Diagnosis
8.1.1 Market Overview
8.1.2 Red Blood Cell (RBC) Count Test
8.1.3 Genetic Testing
8.1.4 High Performance Liquid Chromatography (HPLC) Test
8.1.5 Hemoglobin Isoelectric (Hb IEF) Focusing
8.1.6 Hemoglobin Electrophoresis (Hb ELP) Test
8.1.7 Hemoglobin Solubility Test
8.2 Canada Hemoglobinopathies Market (2017-2032) by Type
8.2.1 Market Overview
8.2.2 Thalassemia
8.2.3 Sickle cell Disease
8.2.4 Others
8.3 Canada Hemoglobinopathies Market (2017-2032) by Therapy
8.3.1 Market Overview
8.3.2 Monoclonal Antibody
8.3.3 Ace Inhibitors
8.3.4 Hydroxyurea
8.3.5 Others
8.4 Canada Hemoglobinopathies Market (2017-2032) by Treatment
8.4.1 Market Overview
8.4.2 Allogeneic Stem Cell Therapy
8.4.3 Autologic Stem Cell Therapy
8.4.4 Syngeneic Stem Cell Therapy
8.5 Canada Hemoglobinopathies Market (2017-2032) by Distribution channel
8.5.1 Market Overview
8.5.2 Hospital Pharmacy
8.5.3 Online Providers
8.5.4 Drug Store and Retail Pharmacy
8.5.5 Others
8.6 Canada Hemoglobinopathies Market (2017-2032) by End User
8.6.1 Market Overview
8.6.2 Biopharmaceutical & Biotechnology Companies
8.6.3 Academic & Research Institutes
8.6.4 Others
9 Regulatory Framework
10 Patent Analysis
10.1 Analysis by Type of Patent
10.2 Analysis by Publication year
10.3 Analysis by Issuing Authority
10.4 Analysis by Patent Age
10.5 Analysis by CPC Analysis
10.6 Analysis by Patent Valuation
10.7 Analysis by Key Players
11 Funding and Investment Analysis
11.1 Analysis by Funding Instances
11.2 Analysis by Type of Funding
11.3 Analysis by Funding Amount
11.4 Analysis by Leading Players
11.5 Analysis by Leading Investors
11.6 Analysis by Geography
12 Partnership and Collaborations Analysis
12.1 Analysis by Partnership Instances
12.2 Analysis by Type of Partnership
12.3 Analysis by Leading Players
12.4 Analysis by Geography
13 Supplier Landscape
13.1 Market Share by Top 5 Companies
13.2 Sangamo Therapeutics,Inc.
13.2.1 Financial Analysis
13.2.2 Product Portfolio
13.2.3 Demographic Reach and Achievements
13.2.4 Mergers and Acquisitions
13.2.5 Certifications
13.3 Global Blood Therapeutics
13.3.1 Financial Analysis
13.3.2 Product Portfolio
13.3.3 Demographic Reach and Achievements
13.3.4 Mergers and Acquisitions
13.3.5 Certifications
13.4 Bluebird bio,Inc
13.4.1 Financial Analysis
13.4.2 Product Portfolio
13.4.3 Demographic Reach and Achievements
13.4.4 Mergers and Acquisitions
13.4.5 Certifications
13.5 Emmaus Life Science Inc.
13.5.1 Financial Analysis
13.5.2 Product Portfolio
13.5.3 Demographic Reach and Achievements
13.5.4 Mergers and Acquisitions
13.5.5 Certifications
13.6 Pfizer,Inc.
13.6.1 Financial Analysis
13.6.2 Product Portfolio
13.6.3 Demographic Reach and Achievements
13.6.4 Mergers and Acquisitions
13.6.5 Certifications
13.7 Novartis AG
13.7.1 Financial Analysis
13.7.2 Product Portfolio
13.7.3 Demographic Reach and Achievements
13.7.4 Mergers and Acquisitions
13.7.5 Certifications
13.8 Prolong Pharmaceuticals,LLC
13.8.1 Financial Analysis
13.8.2 Product Portfolio
13.8.3 Demographic Reach and Achievements
13.8.4 Mergers and Acquisitions
13.8.5 Certifications
13.9 Bioverativ Inc.
13.9.1 Financial Analysis
13.9.2 Product Portfolio
13.9.3 Demographic Reach and Achievements
13.9.4 Mergers and Acquisitions
13.9.5 Certifications
13.10 Gamida Cell
13.10.1 Financial Analysis
13.10.2 Product Portfolio
13.10.3 Demographic Reach and Achievements
13.10.4 Mergers and Acquisitions
13.10.5 Certifications
13.11 Celgene Corporation
13.11.1 Financial Analysis
13.11.2 Product Portfolio
13.11.3 Demographic Reach and Achievements
13.11.4 Mergers and Acquisitions
13.11.5 Certifications
14 North America Hemoglobinopathies Market– Distribution Model (Additional Insight)
14.1 Overview
14.2 Potential Distributors
14.3 Key Parameters for Distribution Partner Assessment
15 Key Opinion Leaders (KOL) Insights (Additional Insight)
16 Company Competitiveness Analysis (Additional Insight)
16.1 Very Small Companies
16.2 Small Companies
16.3 Mid-Sized Companies
16.4 Large Companies
16.5 Very Large Companies
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket
*Additional insights provided are customisable as per client requirements.
* The coverage of the Market Landscape section depends on the data availability and may cover a minimum of 80% of the total market. The EMR team strives to make this section as comprehensive as possible.
Key Questions Answered in the Report
The market is anticipated to grow at a CAGR of 5.10% during the forecast period of 2024-2032, driven by the growing prevalence of sickle cell anemia and thalassemia in the region.
The rising advancements in the treatment and diagnostics of hemoglobinopathies are fuelling the demand for the market.
One of the significant trends in the market is the rise in regulatory approvals by the health regulatory body United States Food and Drug Administration (FDA). In December 2023, Casgevy and Lyfgenia, the first cell-based gene therapies received FDA approval for the treatment of sickle cell disease (SCD) in patients aged 12 years and more.
Based on the diagnosis, the market is segmented into red blood cell (RBC) count test, genetic testing, high performance liquid chromatography (HPLC) test, hemoglobin isoelectric (Hb IEF) focusing, hemoglobin electrophoresis (Hb ELP) test, and hemoglobin solubility test.
Treatments available in the market include allogeneic stem cell therapy, autologous stem cell therapy, and syngeneic stem cell therapy.
By type, the market is divided into thalassemia, and sickle cell disease, among others.
The market breakup by therapy includes monoclonal antibody medication, ace inhibitors, and hydroxyurea, among others.
End users of the market are biopharmaceutical & biotechnology companies, academic & research institutes, among others.
Major distribution channels of the market include hospital pharmacies, online providers, drug stores, and retail pharmacies, among others.
The market segmentation by countries includes the United States of America and Canada.
The key players in the market are Sangamo Therapeutics, Inc., Global Blood Therapeutics, Bluebird bio, Inc., Emmaus Life Science Inc., Pfizer, Inc., Novartis AG, Prolong Pharmaceuticals, LLC, Bioverativ Inc., Gamida Cell, and Celgene Corporation.
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