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BETA地中海型貧血市場:按類型、治療、通路和地區劃分的機會和預測（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個工作天內

價格

全球BETA地中海貧血市場預計將從2023年的5.03億美元成長到2031年的9.1392億美元，2024-2031年預測期間複合年增長率為7.75%。 該市場近年來取得了顯著的成長，預計未來將保持穩定的擴張速度。

BETA-地中海型貧血是從父母遺傳的最常見的體染色體隱性遺傳疾病，其特徵是由於 HBB 基因突變導致 BETA-珠蛋白鏈合成缺陷或缺失，導致功能性血紅蛋白產生減少。 根據嚴重程度，疾病分為輕度、中度和重度 BETA 地中海型貧血。 有多種治療方法可用於治療BETA地中海貧血，包括輸血、鐵螯合療法、基因療法和骨髓療法。 骨髓移植最常在 16 歲以下的患者中進行。

BETA 地中海貧血盛行率不斷上升、治療方法不斷進步以及對即時檢測需求不斷增加，從而影響了 BETA 地中海貧血市場。 近年來醫療基礎設施的成長和醫療費用的增加極大地支持了BETA地中海貧血市場。 此外，政府減輕血液相關疾病負擔的努力正在積極影響相關產品的需求。

BETA地中海型貧血最常見於地中海、東南亞、印度次大陸及中東等低收入地區。 從這些地區向美國等高收入國家的移民顯著改變了盛行率；例如，儘管報告的病例數很少，但BETA地中海貧血的盛行率在過去50年中有所增加，增加了7.5% 。 義大利等國家已實施某些政策來減少血紅蛋白疾病，預計這些措施將在評估期間影響全球BETA地中海貧血市場。 此外，FDA 對BETA-地中海貧血治療的批准在大公司之間形成了良性競爭。

BETA 地中海型貧血盛行率不斷增加

BETA-地中海貧血在南亞、中東、北非和南歐非常普遍。 人口流動正在改變疾病的全球分佈。 BETA地中海貧血被認為是一種罕見疾病，儘管來自敘利亞和伊拉克等受戰爭影響地區的難民湧入導致了疾病統計數據的動盪。 由於高攜帶率、移民趨勢和有利於近親結婚的文化偏好，BETA地中海貧血的全球盛行率正在增加。 隨著BETA-地中海貧血盛行率的增加，預計在預測期內對其治療的需求將會增加。

根據國際地中海貧血聯盟發布的《全球地中海貧血回顧-2023》估計，全球5-7%的人口是影響體內血紅蛋白產生和功能的突變基因攜帶者。 研究顯示，每年有超過 33 萬名受影響的嬰兒出生，其中 83% 患有鐮狀細胞疾病，17% 患有地中海貧血（約 56,000 名嬰兒）。

治療進展

傳統的BETA-地中海貧血治療方法，如輸血和鐵螯合療法有一定的局限性，但BETA-地中海貧血治療的最新進展已經糾正了珠蛋白鏈不平衡和低效的紅血球生成。治療方法得到了改進，分為三大類:解決鐵過載並改善鐵過載。 這些新療法包括研究中的紅血球成熟劑、基因療法和透過體細胞核移植獲得的胚胎樣幹細胞。 正在進行的基因編輯和鐵代謝調節劑的臨床試驗有望帶來新的改進。 BETA-地中海貧血患者現在有多種治療選擇，進一步增強了BETA-地中海貧血市場。

監理機關聚焦重振市場

監管機構透過評估和批准新療法在刺激BETA地中海貧血市場方面發揮關鍵作用。 監管機構對新療法的關注將為患者增加新的治療選擇。 例如，ZYNTEGLO 等基因療法的批准，引入了 BETA 珠蛋白基因的功能性人工副本，是 BETA 地中海型貧血治療的一個重要裡程碑。 此外，對新型基因療法的監管支持以及 CRISPR/Cas9 與誘導多能幹細胞 (IPSC) 技術在基因治療中的結合顯示了監管機構推動該領域進步的潛力。 透過為此類創新療法的開發和批准提供明確的途徑，監管機構可以改善研究和開發，並最終擴大BETA地中海貧血的治療前景。

輸血是BETA地中海型貧血最常見的治療方法。 一些患有嚴重BETA地中海貧血的患者需要定期輸血，稱為輸血依賴性地中海貧血。 輸血有助於增加體內紅血球的數量並改善組織的供氧。 然而，定期輸血會導致鐵超載，從而損害肝臟、心臟和胰臟等重要器官，但已被證明對BETA地中海貧血患者有效。 因此，可能需要鐵螯合療法來清除體內多餘的鐵。 除了輸血之外，還使用幾種聯合療法來提高療效。 例如，2023 年4 月發表在PubMed 上的一篇論文發現，在一項針對輸血依賴性地中海貧血患者進行的研究中，羥基尿聯合治療不僅改善了這些患者的血紅蛋白水平；還被證明可以顯著延長輸血間隔。

醫院藥局是主要通路

醫院藥局部門預計將成為BETA地中海貧血治療藥物的主要通路。 由於治療這種疾病需要複雜的醫療管理，該醫院是BETA-地中海貧血藥物的主要中心。 BETA-地中海型貧血患者需要廣泛的醫療管理，通常需要定期輸血、鐵螯合療法和潛在的治癒性治療。 此外，醫院設備齊全，可以提供與這些治療相關的專門護理和監測，使它們成為實施和實施 BETA 地中海貧血治療的主要場所。

北美主導BETA地中海貧血市場

北美預計將成為BETA地中海貧血市場份額最高的主要地區。 北美的主導地位是由於市場上主要參與者的強大存在、醫療保健成本上升、地中海貧血病例增加、研究活動增加、該地區可支配收入高、醫療保健成本高和血紅蛋白疾病。它主要由以下因素主導:政府努力減少 目前，批准最多的基因療法在美國和加拿大獲得批准，貢獻了BETA地中海貧血市場收入的主要份額。 由於未開發市場的巨大商機以及BETA-地中海貧血盛行率的上升，預計亞太地區、中東和非洲將以高複合年增長率成長。

未來市場情境（2024-2031）

由於移民趨勢和疾病分佈模式的變化，BETA 地中海貧血盛行率不斷增加，這是BETA地中海貧血市場成長的主要驅動力。

BETA 地中海型貧血治療的進展，例如研究性紅血球成熟劑、基因療法和胚胎樣幹細胞，對 BETA 地中海型貧血市場做出了巨大貢獻。

監管部門對BETA-地中海貧血新療法的開發和批准的關注導致商業化產品數量的增加，預計將有助於未來的市場成長。

輸血療法因其療效高、成本效益高，可望成為BETA地中海貧血市場的主流治療方法。

本報告調查了全球除濕機市場，提供了市場定義和概述、市場規模趨勢和預測、各個細分市場和地區的詳細分析、行業結構以及影響市場成長的因素分析。、案例研究、競爭格局、概況關鍵公司等。

目錄

第一章研究方法

第 2 章專案範圍與定義

第 3 章執行摘要

第 4 章 2017 年至 2031 年全球 BETA 地中海型貧血市場展望

  • 市場規模與預測
    • 按價值
    • 按數量
  • 依類型
    • 輕微BETA地中海貧血
    • 中間型BETA地中海貧血
    • 重型BETA地中海貧血
  • 透過治療
    • 輸血
    • 鐵螯合療法
    • 葉酸補充劑
    • 基因治療
    • 骨髓移植
  • 按分銷管道
    • 醫院藥房
    • 零售藥局
    • 網路藥局
  • 按地區
    • 北美
    • 歐洲
    • 亞太地區
    • 南美洲
    • 中東和非洲
  • 各公司的市佔率 (%)，2023 年

第 5 章 2017-2031 年全球 Beta 地中海型貧血市場展望（按地區）

  • 北美
    • 市場規模與預測
    • 依類型
    • 透過治療
    • 按分銷管道
    • 美國
    • 加拿大
    • 墨西哥

所有細分市場均在所有符合資格的地區和國家/地區提供。

  • 歐洲
    • 德國
    • 法國
    • 義大利
    • 英國
    • 俄羅斯
    • 荷蘭
    • 西班牙
    • 土耳其
    • 波蘭
  • 亞太地區
    • 印度
    • 中國
    • 日本
    • 澳大利亞
    • 越南
    • 韓國
    • 印尼
    • 菲律賓
  • 南美洲
    • 巴西
    • 阿根廷
  • 中東和非洲
    • 沙烏地阿拉伯
    • 阿拉伯聯合大公國
    • 南非

第 6 章市場地圖，2023 年

  • 依類型
  • 透過治療
  • 按分銷管道
  • 按地區

第七章宏觀環境與產業結構

  • 需求與供給分析
  • 匯入/匯出分析
  • 價值鏈分析
  • PESTEL 分析
    • 政治因素
    • 經濟體系
    • 社會影響
    • 技術進步
    • 環境影響
    • 法律合規性和監管政策（包括法律當局）
  • 波特五力分析
    • 供應商實力
    • 買家力量
    • 替代威脅
    • 新進入者的威脅
    • 競爭公司之間的對抗關係

第 8 章市場動態

  • 成長動力
  • 生長抑制劑（挑戰與抑制）

第 9 章監理架構與創新

  • 臨床試驗
  • 專利情況
  • 監理機關的核准
  • 創新/新興技術

第十章主要公司狀況

  • 前 5 名市場領導者的競爭矩陣
  • 前 5 位市場領導者的市場收入分析（%，2023 年）
  • 併購/合資企業（如適用））
  • SWOT 分析（針對 5 家進入市場的公司）

第 11 章案例研究

第十二章主要公司展望

  • Bluebird Bio, Inc.
    • 公司簡介
    • 主要管理人員
    • 產品與服務
    • 財務狀況（據報告）
    • 重點市場與地域分佈
    • 近期趨勢
  • Bristol-Myers Squibb Co
  • GlaxoSmithKline plc
  • Incyte Corporation
  • CRISPR Therapeutics AG
  • Ionis Pharmaceuticals Inc
  • Vertex Pharmaceuticals Incorporated
  • Apotex Inc.
  • Novartis International AG

上述公司並未根據其市場份額持有訂單，並可能根據研究工作期間獲得的資訊進行更改。

第 13 章策略建議

第14章關於美國及免責聲明

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