Global Thalassemia Market Report: 2016 Edition
Thalassemia is a hereditary blood disorder in which a person’s body makes an abnormal form of hemoglobin, which is the protein molecule in red blood cells (RBCs), which carries oxygen. The disorder results in unwarranted damage of red blood cells, which leads to anemia. Anemia is a condition in which the body doesn’t have adequate amount of normal and healthy red blood cells. There are majorly two types of thalassemia based on these proteins known as the Alpha thalassemia and the Beta thalassemia. There is also a third type viz. Sickle beta thalassemia, which is like a combination of sickle cell disease and beta thalassemia.
Even though it is a rare genetic disorder, the number of people affected by it has been growing with time. While those with thalassemia minor have hardly any threat to their life and only require occasional blood transfusion to sustain, thalassemia major patients need to go for periodic transfusions for a sustained life. In case of infants, thalassemia major often leads to still birth or cause death shortly after the baby’s birth. Presently the only cure for thalassemia is a bone marrow transplantation therapy, which is done mostly for severe cases of thalassemia.
The key factors which are anticipated to drive this market include rising healthcare expenditure, rising spending on stem cell therapy research and development and rising population in the East-Asia and Pacific region of the world. Some of the noteworthy developments of this industry include the advances in the iron chelation therapy, development of gene therapy and fetal hemoglobin inducers as potential curatives for thalassemia. However, the growth of respective industry is challenged by the high cost of treatments, lack of alternative treatments and unmet market of the Asia-Pacific.
The report provides a comprehensive study of global thalassemia market and also major regional markets. Furthermore, market dynamics such as key trends and development; and challenges are analyzed in depth. The global thalassemia industry is highly competitive consisting of several companies including the Lonza group, Acceleron Pharma, BlueBird Bio and Sangamo Biosciences etc. The competitive landscape of the respective market, along with the company profiles of the leading players are also discussed in detail.
Even though it is a rare genetic disorder, the number of people affected by it has been growing with time. While those with thalassemia minor have hardly any threat to their life and only require occasional blood transfusion to sustain, thalassemia major patients need to go for periodic transfusions for a sustained life. In case of infants, thalassemia major often leads to still birth or cause death shortly after the baby’s birth. Presently the only cure for thalassemia is a bone marrow transplantation therapy, which is done mostly for severe cases of thalassemia.
The key factors which are anticipated to drive this market include rising healthcare expenditure, rising spending on stem cell therapy research and development and rising population in the East-Asia and Pacific region of the world. Some of the noteworthy developments of this industry include the advances in the iron chelation therapy, development of gene therapy and fetal hemoglobin inducers as potential curatives for thalassemia. However, the growth of respective industry is challenged by the high cost of treatments, lack of alternative treatments and unmet market of the Asia-Pacific.
The report provides a comprehensive study of global thalassemia market and also major regional markets. Furthermore, market dynamics such as key trends and development; and challenges are analyzed in depth. The global thalassemia industry is highly competitive consisting of several companies including the Lonza group, Acceleron Pharma, BlueBird Bio and Sangamo Biosciences etc. The competitive landscape of the respective market, along with the company profiles of the leading players are also discussed in detail.
1. OVERVIEW
1.1 Introduction
1.2 Types of Thalassemia
1.2.1 Alpha Thalassemia
1.2.2 Beta Thalassemia
1.2.3 Sickle Beta Thalassemia
1.3 Signs and Symptoms
1.4 Treatment Options
1.4.1 Blood Transfusion
1.4.2 Bone Marrow Transplant
2. GLOBAL THALASSEMIA MARKET
2.1 Global Thalassemia Incidence by Severity
2.2 Global Thalassemia Diagnosis by Category
2.3 Global Transfusion-Dependent β Thalassemia by Region
2.4 Global Thalassemia Market by Therapy
2.4.1 Global Bone Marrow Transplant Revenue
2.4.2 Global LentiGlobin BB305 Revenue
2.4.3 Global Luspatercept Revenue
3. REGIONAL ANALYSIS
3.1 The US Thalassemia Market
3.1.1 The US β Thalassemia Carrier Patient Population
3.1.2 The US β Thalassemia Major Patient Population
3.1.3 The US Transfusion-Dependent β Thalassemia Patients
3.1.4 The US β Thalassemia Sibling-Matched Stem Cell Transplant
3.2 Europe Thalassemia Market
3.2.1 Europe Transfusion-Dependent β Thalassemia Patients
3.2.2 Europe Transfusion-Dependent β-Thalassemia Major/Intermediate Patients
4. MARKET DYNAMICS
4.1 Growth Drivers
4.1.1 Rising Healthcare Expenditure
4.1.2 Rising R&D Spending on Stem Cell Therapy
4.1.3 Rising Population in the East Asia and Pacific Region
4.2 Key Trends and Developments
4.2.1 Gene Therapy for Curing Thalassemia
4.2.2 Advances in Iron Chelation Therapy
4.2.3 Fetal Hemoglobin Inducers
4.3 Challenges
4.3.1 Unmet Market of the Asia-Pacific
4.3.2 High Cost of Therapies
4.3.2 Lack of Alternative Treatments
5. COMPETITIVE LANDSCAPE
5.1 Competition in Bone Marrow Therapy
5.1.1 Revenue Comparison of the Global Key Players
5.1.2 Profit Margin Comparison of the Global Key Players
5.1.3 Market Cap Comparison of the Global Key Players
5.1.4 Revenue Comparison of Key Players of the US
5.2 Competition in Emerging Therapeutics
5.2.1 Product Pipeline Comparison of Emerging Players
6. COMPANY PROFILES
6.1 Lonza Group Ltd
6.1.1 Business Overview
6.1.2 Financial Overview
6.1.3 Business Strategies
6.2 Bluebird Bio
6.2.1 Business Overview
6.2.2 Financial Overview
6.2.3 Business Strategies
6.3 Acceleron Pharma, Inc
6.3.1 Business Overview
6.3.2 Financial Overview
6.3.3 Business Strategies
6.4 Sangamo Biosciences
6.4.1 Business Overview
6.4.2 Financial Overview
6.4.3 Business Strategies
1.1 Introduction
1.2 Types of Thalassemia
1.2.1 Alpha Thalassemia
1.2.2 Beta Thalassemia
1.2.3 Sickle Beta Thalassemia
1.3 Signs and Symptoms
1.4 Treatment Options
1.4.1 Blood Transfusion
1.4.2 Bone Marrow Transplant
2. GLOBAL THALASSEMIA MARKET
2.1 Global Thalassemia Incidence by Severity
2.2 Global Thalassemia Diagnosis by Category
2.3 Global Transfusion-Dependent β Thalassemia by Region
2.4 Global Thalassemia Market by Therapy
2.4.1 Global Bone Marrow Transplant Revenue
2.4.2 Global LentiGlobin BB305 Revenue
2.4.3 Global Luspatercept Revenue
3. REGIONAL ANALYSIS
3.1 The US Thalassemia Market
3.1.1 The US β Thalassemia Carrier Patient Population
3.1.2 The US β Thalassemia Major Patient Population
3.1.3 The US Transfusion-Dependent β Thalassemia Patients
3.1.4 The US β Thalassemia Sibling-Matched Stem Cell Transplant
3.2 Europe Thalassemia Market
3.2.1 Europe Transfusion-Dependent β Thalassemia Patients
3.2.2 Europe Transfusion-Dependent β-Thalassemia Major/Intermediate Patients
4. MARKET DYNAMICS
4.1 Growth Drivers
4.1.1 Rising Healthcare Expenditure
4.1.2 Rising R&D Spending on Stem Cell Therapy
4.1.3 Rising Population in the East Asia and Pacific Region
4.2 Key Trends and Developments
4.2.1 Gene Therapy for Curing Thalassemia
4.2.2 Advances in Iron Chelation Therapy
4.2.3 Fetal Hemoglobin Inducers
4.3 Challenges
4.3.1 Unmet Market of the Asia-Pacific
4.3.2 High Cost of Therapies
4.3.2 Lack of Alternative Treatments
5. COMPETITIVE LANDSCAPE
5.1 Competition in Bone Marrow Therapy
5.1.1 Revenue Comparison of the Global Key Players
5.1.2 Profit Margin Comparison of the Global Key Players
5.1.3 Market Cap Comparison of the Global Key Players
5.1.4 Revenue Comparison of Key Players of the US
5.2 Competition in Emerging Therapeutics
5.2.1 Product Pipeline Comparison of Emerging Players
6. COMPANY PROFILES
6.1 Lonza Group Ltd
6.1.1 Business Overview
6.1.2 Financial Overview
6.1.3 Business Strategies
6.2 Bluebird Bio
6.2.1 Business Overview
6.2.2 Financial Overview
6.2.3 Business Strategies
6.3 Acceleron Pharma, Inc
6.3.1 Business Overview
6.3.2 Financial Overview
6.3.3 Business Strategies
6.4 Sangamo Biosciences
6.4.1 Business Overview
6.4.2 Financial Overview
6.4.3 Business Strategies
LIST OF CHARTS
Global Thalassemia Incidence by Severity (2015E)
Global Thalassemia Diagnosis by Category (2015E)
Global Transfusion-Dependent β Thalassemia by Region (2015E)
Global Bone Marrow Transplant Revenue (2015E-2020F)
Global LentiGlobin BB305 Revenue (2018F-2025F)
Global Luspatercept Revenue (2018F-2022F)
The US β Thalassemia Carrier Patients – Actual (2014-2015E)
The US β Thalassemia Carrier Patients – Forecast (2016F-2020F)
The US β Thalassemia Major Patients – Actual (2014-2015E)
The US β Thalassemia Major Patients – Forecast (2016F-2020F)
The US Transfusion-Dependent β Thalassemia Patients – Actual (2014-2015E)
The US Transfusion-Dependent β Thalassemia Patients – Forecast (2016F-2020F)
The US β Thalassemia Sibling-Matched Stem Cell Transplant – Actual (2014-2015E)
The US β Thalassemia Sibling-Matched Stem Cell Transplant – Forecast (2016F-2020F)
Europe Transfusion-Dependent β Thalassemia Patients (2015E-2020F)
Europe Transfusion-Dependent β-Thalassemia Major/Intermediate Patients (2015E-2020F)
Global Healthcare Expenditure (2010-2015E)
Spending on Stem Cell Research in the US (2013-2016F)
Population in East-Asia and Pacific (2010-2015E)
Revenue Comparison of the Global Key Players (2015)
Gross Profit Margin of the Global Key Players (2015)
Market Capitalization of the Global Key Players (2015)
Revenue Comparison of Key Players of the US (2015)
Revenue Share by Business Segments, Lonza Group (2015)
Revenue and Net Income, Lonza Group (2011-2015)
Revenue and Net Income, Bluebird Bio (2011-2015)
Revenue and Net Income, Acceleron Pharma (2011-2015)
Revenue and Net Income, Sangamo Biosciences (2011-2015)
Global Thalassemia Incidence by Severity (2015E)
Global Thalassemia Diagnosis by Category (2015E)
Global Transfusion-Dependent β Thalassemia by Region (2015E)
Global Bone Marrow Transplant Revenue (2015E-2020F)
Global LentiGlobin BB305 Revenue (2018F-2025F)
Global Luspatercept Revenue (2018F-2022F)
The US β Thalassemia Carrier Patients – Actual (2014-2015E)
The US β Thalassemia Carrier Patients – Forecast (2016F-2020F)
The US β Thalassemia Major Patients – Actual (2014-2015E)
The US β Thalassemia Major Patients – Forecast (2016F-2020F)
The US Transfusion-Dependent β Thalassemia Patients – Actual (2014-2015E)
The US Transfusion-Dependent β Thalassemia Patients – Forecast (2016F-2020F)
The US β Thalassemia Sibling-Matched Stem Cell Transplant – Actual (2014-2015E)
The US β Thalassemia Sibling-Matched Stem Cell Transplant – Forecast (2016F-2020F)
Europe Transfusion-Dependent β Thalassemia Patients (2015E-2020F)
Europe Transfusion-Dependent β-Thalassemia Major/Intermediate Patients (2015E-2020F)
Global Healthcare Expenditure (2010-2015E)
Spending on Stem Cell Research in the US (2013-2016F)
Population in East-Asia and Pacific (2010-2015E)
Revenue Comparison of the Global Key Players (2015)
Gross Profit Margin of the Global Key Players (2015)
Market Capitalization of the Global Key Players (2015)
Revenue Comparison of Key Players of the US (2015)
Revenue Share by Business Segments, Lonza Group (2015)
Revenue and Net Income, Lonza Group (2011-2015)
Revenue and Net Income, Bluebird Bio (2011-2015)
Revenue and Net Income, Acceleron Pharma (2011-2015)
Revenue and Net Income, Sangamo Biosciences (2011-2015)
LIST OF TABLES
Product Pipeline of Emerging Players (2015)
Product Pipeline, Bluebird Bio (2015)
Product Pipeline, Acceleron Pharma (2015)
Product Pipeline, Sangamo Biosciences (2015)
Product Pipeline of Emerging Players (2015)
Product Pipeline, Bluebird Bio (2015)
Product Pipeline, Acceleron Pharma (2015)
Product Pipeline, Sangamo Biosciences (2015)
