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市场调查报告书
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BETA地中海型贫血市场:按类型、治疗、通路和地区划分的机会和预测（2017-2031）

Beta Thalassemia Market Assessment, By Type, By Therapy, By Distribution Channel, By Region, Opportunities and Forecast, 2017-2031F
出版日期: | 出版商: Market Xcel - Markets and Data | 英文 225 Pages | 商品交期: 3-5个工作天内

价格

全球BETA地中海贫血市场预计将从2023年的5.03亿美元成长到2031年的9.1392亿美元，2024-2031年预测期间复合年增长率为7.75%。 该市场近年来取得了显着的成长，预计未来将保持稳定的扩张速度。

BETA-地中海型贫血是从父母遗传的最常见的体染色体隐性遗传疾病，其特征是由于 HBB 基因突变导致 BETA-珠蛋白链合成缺陷或缺失，导致功能性血红蛋白产生减少。 根据严重程度，疾病分为轻度、中度和重度 BETA 地中海型贫血。 有多种治疗方法可用于治疗BETA地中海贫血，包括输血、铁螯合疗法、基因疗法和骨髓疗法。 骨髓移植最常在 16 岁以下的患者中进行。

BETA 地中海贫血盛行率不断上升、治疗方法不断进步以及对即时检测需求不断增加，从而影响了 BETA 地中海贫血市场。 近年来医疗基础设施的成长和医疗费用的增加极大地支持了BETA地中海贫血市场。 此外，政府减轻血液相关疾病负担的努力正在积极影响相关产品的需求。

BETA地中海型贫血最常见于地中海、东南亚、印度次大陆及中东等低收入地区。 从这些地区向美国等高收入国家的移民显着改变了盛行率；例如，儘管报告的病例数很少，但BETA地中海贫血的盛行率在过去50年中有所增加，增加了7.5% 。 义大利等国家已实施某些政策来减少血红蛋白疾病，预计这些措施将在评估期间影响全球BETA地中海贫血市场。 此外，FDA 对BETA-地中海贫血治疗的批准在大公司之间形成了良性竞争。

BETA 地中海型贫血盛行率不断增加

BETA-地中海贫血在南亚、中东、北非和南欧非常普遍。 人口流动正在改变疾病的全球分布。 BETA地中海贫血被认为是一种罕见疾病，儘管来自叙利亚和伊拉克等受战争影响地区的难民涌入导致了疾病统计数据的动盪。 由于高携带率、移民趋势和有利于近亲结婚的文化偏好，BETA地中海贫血的全球盛行率正在增加。 随着BETA-地中海贫血盛行率的增加，预计在预测期内对其治疗的需求将会增加。

根据国际地中海贫血联盟发布的《全球地中海贫血回顾-2023》估计，全球5-7%的人口是影响体内血红蛋白产生和功能的突变基因携带者。 研究显示，每年有超过 33 万名受影响的婴儿出生，其中 83% 患有镰状细胞疾病，17% 患有地中海贫血（约 56,000 名婴儿）。

治疗进展

传统的BETA-地中海贫血治疗方法，如输血和铁螯合疗法有一定的局限性，但BETA-地中海贫血治疗的最新进展已经纠正了珠蛋白链不平衡和低效的红血球生成。治疗方法得到了改进，分为三大类:解决铁过载并改善铁过载。 这些新疗法包括研究中的红血球成熟剂、基因疗法和透过体细胞核移植获得的胚胎样干细胞。 正在进行的基因编辑和铁代谢调节剂的临床试验有望带来新的改进。 BETA-地中海贫血患者现在有多种治疗选择，进一步增强了BETA-地中海贫血市场。

监理机关聚焦重振市场

监管机构透过评估和批准新疗法在刺激BETA地中海贫血市场方面发挥关键作用。 监管机构对新疗法的关注将为患者增加新的治疗选择。 例如，ZYNTEGLO 等基因疗法的批准，引入了 BETA 珠蛋白基因的功能性人工副本，是 BETA 地中海型贫血治疗的一个重要里程碑。 此外，对新型基因疗法的监管支持以及 CRISPR/Cas9 与诱导多能干细胞 (IPSC) 技术在基因治疗中的结合显示了监管机构推动该领域进步的潜力。 透过为此类创新疗法的开发和批准提供明确的途径，监管机构可以改善研究和开发，并最终扩大BETA地中海贫血的治疗前景。

输血是BETA地中海型贫血最常见的治疗方法。 一些患有严重BETA地中海贫血的患者需要定期输血，称为输血依赖性地中海贫血。 输血有助于增加体内红血球的数量并改善组织的供氧。 然而，定期输血会导致铁超载，从而损害肝臟、心臟和胰臟等重要器官，但已被证明对BETA地中海贫血患者有效。 因此，可能需要铁螯合疗法来清除体内多余的铁。 除了输血之外，还使用几种联合疗法来提高疗效。 例如，2023 年4 月发表在PubMed 上的一篇论文发现，在一项针对输血依赖性地中海贫血患者进行的研究中，羟基尿联合治疗不仅改善了这些患者的血红蛋白水平；还被证明可以显着延长输血间隔。

医院药局是主要通路

医院药局部门预计将成为BETA地中海贫血治疗药物的主要通路。 由于治疗这种疾病需要复杂的医疗管理，该医院是BETA-地中海贫血药物的主要中心。 BETA-地中海型贫血患者需要广泛的医疗管理，通常需要定期输血、铁螯合疗法和潜在的治癒性治疗。 此外，医院设备齐全，可以提供与这些治疗相关的专门护理和监测，使它们成为实施和实施 BETA 地中海贫血治疗的主要场所。

北美主导BETA地中海贫血市场

北美预计将成为BETA地中海贫血市场份额最高的主要地区。 北美的主导地位是由于市场上主要参与者的强大存在、医疗保健成本上升、地中海贫血病例增加、研究活动增加、该地区可支配收入高、医疗保健成本高和血红蛋白疾病。它主要由以下因素主导:政府努力减少 目前，批准最多的基因疗法在美国和加拿大获得批准，贡献了BETA地中海贫血市场收入的主要份额。 由于未开发市场的巨大商机以及BETA-地中海贫血盛行率的上升，预计亚太地区、中东和非洲将以高复合年增长率成长。

未来市场情境（2024-2031）

由于移民趋势和疾病分布模式的变化，BETA 地中海贫血盛行率不断增加，这是BETA地中海贫血市场成长的主要驱动力。

BETA 地中海型贫血治疗的进展，例如研究性红血球成熟剂、基因疗法和胚胎样干细胞，对 BETA 地中海型贫血市场做出了巨大贡献。

监管部门对BETA-地中海贫血新疗法的开发和批准的关注导致商业化产品数量的增加，预计将有助于未来的市场成长。

输血疗法因其疗效高、成本效益高，可望成为BETA地中海贫血市场的主流治疗方法。

本报告调查了全球除湿机市场，提供了市场定义和概述、市场规模趋势和预测、各个细分市场和地区的详细分析、行业结构以及影响市场成长的因素分析。、案例研究、竞争格局、概况关键公司等。

目录

第一章研究方法

第 2 章专案范围与定义

第 3 章执行摘要

第 4 章 2017 年至 2031 年全球 BETA 地中海型贫血市场展望

  • 市场规模与预测
    • 按价值
    • 按数量
  • 依类型
    • 轻微BETA地中海贫血
    • 中间型BETA地中海贫血
    • 重型BETA地中海贫血
  • 透过治疗
    • 输血
    • 铁螯合疗法
    • 叶酸补充剂
    • 基因治疗
    • 骨髓移植
  • 按分销管道
    • 医院药房
    • 零售药局
    • 网路药局
  • 按地区
    • 北美
    • 欧洲
    • 亚太地区
    • 南美洲
    • 中东和非洲
  • 各公司的市占率 (%)，2023 年

第 5 章 2017-2031 年全球 Beta 地中海型贫血市场展望（按地区）

  • 北美
    • 市场规模与预测
    • 依类型
    • 透过治疗
    • 按分销管道
    • 美国
    • 加拿大
    • 墨西哥

所有细分市场均在所有符合资格的地区和国家/地区提供。

  • 欧洲
    • 德国
    • 法国
    • 义大利
    • 英国
    • 俄罗斯
    • 荷兰
    • 西班牙
    • 土耳其
    • 波兰
  • 亚太地区
    • 印度
    • 中国
    • 日本
    • 澳大利亚
    • 越南
    • 韩国
    • 印尼
    • 菲律宾
  • 南美洲
    • 巴西
    • 阿根廷
  • 中东和非洲
    • 沙乌地阿拉伯
    • 阿拉伯联合大公国
    • 南非

第 6 章市场地图，2023 年

  • 依类型
  • 透过治疗
  • 按分销管道
  • 按地区

第七章宏观环境与产业结构

  • 需求与供给分析
  • 汇入/汇出分析
  • 价值链分析
  • PESTEL 分析
    • 政治因素
    • 经济体系
    • 社会影响
    • 技术进步
    • 环境影响
    • 法律合规性和监管政策（包括法律当局）
  • 波特五力分析
    • 供应商实力
    • 买家力量
    • 替代威胁
    • 新进入者的威胁
    • 竞争公司之间的对抗关係

第 8 章市场动态

  • 成长动力
  • 生长抑制剂（挑战与抑制）

第 9 章监理架构与创新

  • 临床试验
  • 专利情况
  • 监理机关的核准
  • 创新/新兴技术

第十章主要公司状况

  • 前 5 名市场领导者的竞争矩阵
  • 前 5 位市场领导者的市场收入分析（%，2023 年）
  • 併购/合资企业（如适用））
  • SWOT 分析（针对 5 家进入市场的公司）

第 11 章案例研究

第十二章主要公司展望

  • Bluebird Bio, Inc.
    • 公司简介
    • 主要管理人员
    • 产品与服务
    • 财务状况（据报告）
    • 重点市场与地域分布
    • 近期趋势
  • Bristol-Myers Squibb Co
  • GlaxoSmithKline plc
  • Incyte Corporation
  • CRISPR Therapeutics AG
  • Ionis Pharmaceuticals Inc
  • Vertex Pharmaceuticals Incorporated
  • Apotex Inc.
  • Novartis International AG

上述公司并未根据其市场份额持有订单，并可能根据研究工作期间获得的资讯进行更改。

第 13 章策略建议

第14章关于美国及免责声明

Product Code: MX10997

Global beta thalassemia market is projected to witness a CAGR of 7.75% during the forecast period 2024-2031F, growing from USD 503 million in 2023, to USD 913.92 million in 2031. The market has experienced significant growth in recent years and is expected to maintain a strong pace of expansion in the coming years.

Beta thalassemia is the most common autosomal recessive disorder inherited from parents and characterized by a reduced production of functional hemoglobin due to a mutation in the HBB gene that leads to the deficient or absent synthesis of beta-globin chains. Based on the severity, the disorder is categorized into minor, intermediate, and major types of beta thalassemia. A variety of therapies like blood transfusion, iron chelation therapy, gene therapy, and bone marrow therapy treat the beta thalassemia condition. Bone marrow transplants are most common in patients under the age of 16 years.

The beta thalassemia marktherapies andly influenced by the growing prevalence of beta thalassemia, escalated advancement in therapies, and increasing demand for point-of-care testing. Growing healthcare infrastructure and increased healthcare spending in recent years have significantly supported the beta thalassemia market. Additionally, government initiatives to reduce the blood-related disease burden have positively impacted the demand for relevant products.

Beta thalassemia is most common in low-income regions like Mediterranean, Southeast Asia, the Indian subcontinent, and the Middle East. The migration of people from these regions to high-income countries like the United States has significantly changed the prevalence, for example, the prevalence of beta thalassemia has increased by 7.5% in the last 50 years despite the low number of reported cases. Countries like Italy implemented specific policies for reducing hemoglobinopathies, these initiatives are anticipated to impact the global beta thalassemia market during the assessment period. Alongside, FDA approval of beta thalassemia therapies in recent times has developed healthy competition among the key players.

In January 2024, Vertex Pharmaceuticals Incorporated announced that U.S. Food and Drug Administration (FDA) approved its CASGEVY (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited cell therapy. The therapy is meant for treating transfusion-dependent beta thalassemia (TDT) in patients above the age of 12 years. The therapy is available in the United States for now in hospitals with expertise in stem cell transplantation. CASGEVY has shown a 91.4% success rate in patients with TDT.

Growing Prevalence of Beta Thalassemia

Beta thalassemia has a significant prevalence in South Asia, Middle East, North Africa, and Southern Europe. Population migration is changing the global distribution of disease. The flow of refugee populations from war-affected zones, like Syria and Iraq, is contributing to the disturbance of disease statistics, but beta thalassemia is considered a rare disease. The global prevalence of beta thalassemia is increasing due to a high carrier rate, migration trends, and cultural preference for consanguineous marriages. With the increasing prevalence of beta thalassemia, the demand for its treatment is anticipated to rise during the forecast period.

As per 'Global Thalassemia Review - 2023' published by Thalassemia International Federation estimates, 5-7% of the global population is the carrier of mutated genes affecting the production or function of the hemoglobin in the body. It suggests that over 330,000 affected infants are born annually out of which 83% have sickle cell disorders and 17% are thalassemia (counting around 56,000 infants).

Escalated Advancement in Therapies

Conventional therapies for beta thalassemia, such as transfusion and iron chelation, have several limitations, however, recent advancements in beta thalassemia therapies have led to improved therapies that can be categorized into three major categories, correction of the globin chain imbalance, addressing ineffective erythropoiesis, and improving iron overload. These emerging therapies include investigational erythroid maturation agents, gene therapy, and embryonic-like stem cells obtained by somatic cell nuclear transfer. Ongoing clinical trials of gene editing and agents modulating iron metabolism promise new improvements. Patients with beta thalassemia have several therapeutic options nowadays, further strengthening the beta thalassemia market.

For example, in August 2023, the U.S. FDA approved Bristol Myers Squibb's Reblozyl (luspatercept-aamt) for providing first-line treatment of anemia in adults with lower-risk myelodysplastic syndromes requiring transfusions. Reblozyl is commercialized through global collaboration between Bristol Mayer Squibb and Merck since November 2021. With this approval, Reblozyl is now approved in the United States for patients of anemia, beta thalassemia, and myelodysplastic syndromes.

Focus of Regulatory Bodies to Catalyze the Market

Regulatory bodies play a crucial role in catalyzing the beta thalassemia market by evaluating and approving novel therapies. The focus of regulatory bodies on emerging treatments can improve the availability of novel treatment options for patients. For instance, the approval of gene therapies such as ZYNTEGLO, which introduces functional, engineered copies of beta-globin genes, represents a significant milestone in the treatment of beta thalassemia. Additionally, the regulatory support for novel genetic therapies and the combination of CRISPR/Cas9 and induced pluripotent stem cell (IPSC) technologies in gene therapy demonstrate the potential for regulatory bodies to foster advancements in the field. By providing a clear pathway for developing and approving these innovative therapies, regulatory bodies can improvise the research and development, ultimately expanding the treatment landscape for beta thalassemia.

For example, in March 2023, Bristol Myers Squibb received European Commission approval for Reblozyl (luspatercept) for anemia in adult patients with non-transfusion-dependent beta thalassemia. After the approval, Reblozyl is approved and available in the European Union (EU), the United States, and Canada for treating beta thalassemia patients. The European Commission approved it based on the phase 2 BEYOND study, which proved the efficacy and safety of Reblozyl versus placebo in 145 patients.

Blood Transfusion Segment Dominate the Market

Blood transfusion is the most common type of therapy for beta thalassemia. In more severe cases, such as beta thalassemia major, some patients may require periodic blood transfusions known as transfusion-dependent thalassemia. Blood transfusions help to increase the number of red blood cells in the body and improve oxygen delivery to the tissues. However, regular blood transfusions can lead to iron overload, which can damage vital organs like the liver, heart, and pancreas, but is proven effective in beta thalassemia patients. Therefore, iron chelation therapy may be necessary to remove excess iron from the body. Several conjunctive therapies are provided in addition to blood transfusion to increase efficacy. For instance, as per an article published in PubMed in April 2023, the study conducted among transfusion-dependent thalassemia patients showed that conjunctive therapy of hydroxyurea significantly increases the time interval between transfusions as well as improves the hemoglobin level in these patients.

Hospital Pharmacies are the Leading Distribution Channel

The hospital pharmacies segment is anticipated to be the leading distribution channel for beta thalassemia treatments. Hospitals are major centers for beta thalassemia drugs due to the complex medical management required for this condition. Patients with beta thalassemia often need routine blood transfusions, iron chelation therapy, and potentially curative treatments, which demand extensive medical supervision. Additionally, hospitals are equipped to handle the specialized care and monitoring associated with these treatments, making them primary location for administering and managing beta thalassemia therapies.

North America Dominates Beta Thalassemia Market

North America is anticipated to be the leading region in the beta thalassemia market with the highest market share. The dominance of North America is governed by factors like the strong presence of major players in the market and is characterized by increasing healthcare expenditure, rising cases of thalassemia, a growing number of research activities, high disposable income of the region, high healthcare expenditure, and government initiatives to reduce hemoglobinopathies. Presently, the most approved gene therapies are approved in the United States and Canada, contributing the major share of revenue to the beta thalassemia market. Asia-Pacific and Middle East and Africa are anticipated to grow with high CAGR due to large opportunities in untapped markets and the rising prevalence of beta thalassemia.

Future Market Scenario (2024 - 2031F)

Growing prevalence of beta thalassemia due to migration trends and changes in disease distribution patterns are majorly driving the growth in the beta thalassemia market.

Advancements in beta thalassemia therapies such as investigational erythroid maturation agents, gene therapy, and embryonic-like stem cells drastically contribute to the beta thalassemia market.

Regulatory emphasis on the development and approval of novel therapies for beta thalassemia is leading to an increase in the number of commercialized products which is anticipated to contribute towards the future growth of the market.

Blood transfusion segment is anticipated to dominate therapy type in the beta thalassemia market due to high efficacy and cost-effectiveness.

Key Players Landscape and Outlook

Key participants in the beta thalassemia market include Bluebird Bio, Inc., Bristol-Myers Squibb Co., and Vertex Pharmaceuticals Incorporated. The market caters to several pharmaceutical and biotechnology companies and startups. These companies are involved in the development and commercialization of various treatments and therapies for beta thalassemia. The players in this market are actively involved in product development and approvals, collaboration, and other market tactics. Several players have promising product pipelines and therapies under different phases of clinical trials.

For instance, Shanghai BDgene Co., Ltd., Shenzhen Hemogen, Editas Medicine, Inc., Vertex Pharmaceuticals Incorporated, Bristol-Myers Squibb, Editas Medicine, Inc., CorrectSequence Therapeutics Co., Ltd, are some market players that have genetic and biological products under clinical investigation, presently.

In November 2023, MHRA authorized the world's first gene therapy (Casgevy) to cure sickle-cell anemia and transfusion-dependent B-thalassemia. The therapy was developed by CRISPR therapeutics. Casgevy is the first approved medicine that utilizes the innovative gene-editing technique CRISPR, for which its inventors were awarded the Nobel Prize in 2020.

Table of Contents

1. Research Methodology

2. Project Scope & Definitions

3. Executive Summary

4. Global Beta Thalassemia Market Outlook, 2017-2031F

  • 4.1. Market Size & Forecast
    • 4.1.1. By Value
    • 4.1.2. By Volume
  • 4.2. By Type
    • 4.2.1. Beta Thalassemia Minor
    • 4.2.2. Beta Thalassemia Intermedia
    • 4.2.3. Beta Thalassemia Major
  • 4.3. By Therapy
    • 4.3.1. Blood Transfusions
    • 4.3.2. Iron Chelation Therapy
    • 4.3.3. Folic Acid Supplements
    • 4.3.4. Gene Therapy
    • 4.3.5. Bone Marrow Transplants
  • 4.4. By Distribution Channel
    • 4.4.1. Hospital Pharmacies
    • 4.4.2. Retail Pharmacies
    • 4.4.3. Online Pharmacies
  • 4.5. By Region
    • 4.5.1. North America
    • 4.5.2. Europe
    • 4.5.3. Asia-Pacific
    • 4.5.4. South America
    • 4.5.5. Middle East and Africa
  • 4.6. By Company Market Share (%), 2023

5. Global Beta Thalassemia Market Outlook, By Region, 2017-2031F

  • 5.1. North America*
    • 5.1.1. Market Size & Forecast
      • 5.1.1.1. By Value
      • 5.1.1.2. By Volume
    • 5.1.2. By Type
      • 5.1.2.1. Beta Thalassemia Minor
      • 5.1.2.2. Beta Thalassemia Intermedia
      • 5.1.2.3. Beta Thalassemia Major
    • 5.1.3. By Therapy
      • 5.1.3.1. Blood Transfusions
      • 5.1.3.2. Iron Chelation Therapy
      • 5.1.3.3. Folic Acid Supplements
      • 5.1.3.4. Gene Therapy
      • 5.1.3.5. Bone Marrow Transplants
    • 5.1.4. By Distribution Channel
      • 5.1.4.1. Hospital Pharmacies
      • 5.1.4.2. Retail Pharmacies
      • 5.1.4.3. Online Pharmacies
    • 5.1.5. United States*
      • 5.1.5.1. Market Size & Forecast
      • 5.1.5.1.1. By Value
      • 5.1.5.1.2. By Volume
      • 5.1.5.2. By Type
      • 5.1.5.2.1. Beta Thalassemia Minor
      • 5.1.5.2.2. Beta Thalassemia Intermedia
      • 5.1.5.2.3. Beta Thalassemia Major
      • 5.1.5.3. By Therapy
      • 5.1.5.3.1. Blood Transfusions
      • 5.1.5.3.2. Iron Chelation Therapy
      • 5.1.5.3.3. Folic Acid Supplements
      • 5.1.5.3.4. Gene Therapy
      • 5.1.5.3.5. Bone Marrow Transplants
      • 5.1.5.4. By Distribution Channel
      • 5.1.5.4.1. Hospital Pharmacies
      • 5.1.5.4.2. Retail Pharmacies
      • 5.1.5.4.3. Online Pharmacies
    • 5.1.6. Canada
    • 5.1.7. Mexico

All segments will be provided for all regions and countries covered

  • 5.1. Europe
    • 5.1.6. Germany
    • 5.1.7. France
    • 5.1.8. Italy
    • 5.1.9. United Kingdom
    • 5.1.10. Russia
    • 5.1.11. Netherlands
    • 5.1.12. Spain
    • 5.1.13. Turkey
    • 5.1.14. Poland
  • 5.2. Asia-Pacific
    • 5.2.6. India
    • 5.2.7. China
    • 5.2.8. Japan
    • 5.2.9. Australia
    • 5.2.10. Vietnam
    • 5.2.11. South Korea
    • 5.2.12. Indonesia
    • 5.2.13. Philippines
  • 5.3. South America
    • 5.3.6. Brazil
    • 5.3.7. Argentina
  • 5.4. Middle East & Africa
    • 5.4.6. Saudi Arabia
    • 5.4.7. UAE
    • 5.4.8. South Africa

6. Market Mapping, 2023

  • 6.1. By Type
  • 6.2. By Therapy
  • 6.3. By Distribution Channel
  • 6.4. By Region

7. Macro Environment and Industry Structure

  • 7.1. Demand Supply Analysis
  • 7.2. Import Export Analysis
  • 7.3. Value Chain Analysis
  • 7.4. PESTEL Analysis
    • 7.4.6. Political Factors
    • 7.4.7. Economic System
    • 7.4.8. Social Implications
    • 7.4.9. Technological Advancements
    • 7.4.10. Environmental Impacts
    • 7.4.11. Legal Compliances and Regulatory Policies (Statutory Bodies Included)
  • 7.5. Porter's Five Forces Analysis
    • 7.5.6. Supplier Power
    • 7.5.7. Buyer Power
    • 7.5.8. Substitution Threat
    • 7.5.9. Threat from New Entrant
    • 7.5.10. Competitive Rivalry

8. Market Dynamics

  • 8.1. Growth Drivers
  • 8.2. Growth Inhibitors (Challenges and Restraints)

9. Regulatory Framework and Innovation

  • 9.1. Clinical Trials
  • 9.2. Patent Landscape
  • 9.3. Regulatory Approvals
  • 9.4. Innovations/Emerging Technologies

10. Key Players Landscape

  • 10.1. Competition Matrix of Top Five Market Leaders
  • 10.2. Market Revenue Analysis of Top Five Market Leaders (in %, 2023)
  • 10.3. Mergers and Acquisitions/Joint Ventures (If Applicable)
  • 10.4. SWOT Analysis (For Five Market Players)

11. Case Studies

12. Key Players Outlook

  • 12.1. Bluebird Bio, Inc.
    • 12.1.1. Company Details
    • 12.1.2. Key Management Personnel
    • 12.1.3. Products & Services
    • 12.1.4. Financials (As reported)
    • 12.1.5. Key Market Focus & Geographical Presence
    • 12.1.6. Recent Developments
  • 12.2. Bristol-Myers Squibb Co
  • 12.3. GlaxoSmithKline plc
  • 12.4. Incyte Corporation
  • 12.5. CRISPR Therapeutics AG
  • 12.6. Ionis Pharmaceuticals Inc
  • 12.7. Vertex Pharmaceuticals Incorporated
  • 12.8. Apotex Inc.
  • 12.9. Novartis International AG

Companies mentioned above DO NOT hold any order as per market share and can be changed as per information available during research work.

13. Strategic Recommendations

14. About Us & Disclaimer

List of Tables

  • Table 1. Pricing Analysis of Products from Key Players
  • Table 2. Competition Matrix of Top 5 Market Leaders
  • Table 3. Mergers & Acquisitions/ Joint Ventures (If Applicable)
  • Table 4. About Us - Regions and Countries Where We Have Executed Client Projects

List of Figures

  • Figure 1. Global Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 2. Global Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 3. Global Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 4. Global Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 5. Global Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 6. Global Beta Thalassemia Market Share (%), By Region, 2017-2031F
  • Figure 7. North America Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 8. North America Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 9. North America Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 10. North America Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 11. North America Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 12. North America Beta Thalassemia Market Share (%), By Country, 2017-2031F
  • Figure 13. United States Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 14. United States Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 15. United States Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 16. United States Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 17. United States Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 18. Canada Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 19. Canada Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 20. Canada Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 21. Canada Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 22. Canada Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 23. Mexico Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 24. Mexico Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 25. Mexico Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 26. Mexico Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 27. Mexico Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 28. Europe Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 29. Europe Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 30. Europe Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 31. Europe Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 32. Europe Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 33. Europe Beta Thalassemia Market Share (%), By Country, 2017-2031F
  • Figure 34. Germany Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 35. Germany Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 36. Germany Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 37. Germany Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 38. Germany Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 39. France Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 40. France Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 41. France Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 42. France Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 43. France Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 44. Italy Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 45. Italy Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 46. Italy Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 47. Italy Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 48. Italy Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 49. United Kingdom Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 50. United Kingdom Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 51. United Kingdom Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 52. United Kingdom Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 53. United Kingdom Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 54. Russia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 55. Russia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 56. Russia Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 57. Russia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 58. Russia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 59. Netherlands Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 60. Netherlands Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 61. Netherlands Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 62. Netherlands Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 63. Netherlands Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 64. Spain Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 65. Spain Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 66. Spain Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 67. Spain Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 68. Spain Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 69. Turkey Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 70. Turkey Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 71. Turkey Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 72. Turkey Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 73. Turkey Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 74. Poland Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 75. Poland Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 76. Poland Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 77. Poland Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 78. Poland Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 79. South America Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 80. South America Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 81. South America Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 82. South America Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 83. South America Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 84. South America Beta Thalassemia Market Share (%), By Country, 2017-2031F
  • Figure 85. Brazil Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 86. Brazil Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 87. Brazil Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 88. Brazil Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 89. Brazil Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 90. Argentina Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 91. Argentina Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 92. Argentina Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 93. Argentina Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 94. Argentina Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 95. Asia-Pacific Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 96. Asia-Pacific Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 97. Asia-Pacific Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 98. Asia-Pacific Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 99. Asia-Pacific Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 100. Asia-Pacific Beta Thalassemia Market Share (%), By Country, 2017-2031F
  • Figure 101. India Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 102. India Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 103. India Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 104. India Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 105. India Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 106. China Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 107. China Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 108. China Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 109. China Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 110. China Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 111. Japan Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 112. Japan Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 113. Japan Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 114. Japan Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 115. Japan Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 116. Australia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 117. Australia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 118. Australia Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 119. Australia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 120. Australia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 121. Vietnam Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 122. Vietnam Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 123. Vietnam Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 124. Vietnam Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 125. Vietnam Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 126. South Korea Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 127. South Korea Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 128. South Korea Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 129. South Korea Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 130. South Korea Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 131. Indonesia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 132. Indonesia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 133. Indonesia Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 134. Indonesia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 135. Indonesia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 136. Philippines Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 137. Philippines Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 138. Philippines Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 139. Philippines Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 140. Philippines Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 141. Middle East & Africa Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 142. Middle East & Africa Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 143. Middle East & Africa Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 144. Middle East & Africa Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 145. Middle East & Africa Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 146. Middle East & Africa Beta Thalassemia Market Share (%), By Country, 2017-2031F
  • Figure 147. Saudi Arabia Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 148. Saudi Arabia Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 149. Saudi Arabia Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 150. Saudi Arabia Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 151. Saudi Arabia Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 152. UAE Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 153. UAE Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 154. UAE Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 155. UAE Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 156. UAE Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 157. South Africa Beta Thalassemia Market, By Value, in USD Million, 2017-2031F
  • Figure 158. South Africa Beta Thalassemia Market, By Volume, in Number of Patients, 2017-2031F
  • Figure 159. South Africa Beta Thalassemia Market Share (%), By Type, 2017-2031F
  • Figure 160. South Africa Beta Thalassemia Market Share (%), By Therapy, 2017-2031F
  • Figure 161. South Africa Beta Thalassemia Market Share (%), By Distribution Channel, 2017-2031F
  • Figure 162. By Type Map-Market Size (USD Million) & Growth Rate (%), 2023
  • Figure 163. By Therapy Map-Market Size (USD Million) & Growth Rate (%), 2023
  • Figure 164. By Distribution Channel Map-Market Size (USD Million) & Growth Rate (%), 2023
  • Figure 165. By Region Map-Market Size (USD Million) & Growth Rate (%), 2023