異常ヘモグロビン症の市場規模、シェア、動向分析レポート：タイプ別、診断別、治療別、地域別、セグメント予測、2023年～2030年

異常ヘモグロビン症市場の成長と動向：

Grand View Research, Inc.の最新レポートによると、世界の異常ヘモグロビン症市場規模は2030年までに207億米ドルに達し、2023年から2030年までのCAGRは12.6%で拡大すると予測されています。

同市場は、鎌状赤血球症（SCD）やサラセミアなどの疾患の有病率の増加により、着実な成長を遂げています。促進要因としては、診断技術の進歩、意識の高まり、医療インフラの改善などが挙げられます。さらに、革新的な治療法の開発や政府の取り組みも市場拡大に寄与しています。

近年、鎌状赤血球症の診断技術は著しく進歩しています。顕著な動向のひとつは、鎌状赤血球症に関連する特定の変異を同定するための分子遺伝学的検査、特にDNA解析の利用が増加していることです。このアプローチにより、精密で正確な診断が可能となり、医療専門家は個人の鎌状赤血球形質またはSCDの存在を判定することができます。さらに、技術の進歩によりポイントオブケア検査装置が開発され、資源が限られた環境でも迅速なスクリーニングと診断が可能になりました。このような診断技術の進歩は、早期発見と早期介入を促進するだけでなく、遺伝カウンセリングや、鎌状赤血球症を受け継ぐリスクのある人の家族計画を容易にします。

SCDやサラセミアを含む異常ヘモグロビン症に対する治療の進歩は、近年有望な動向を見せています。遺伝子治療アプローチの開発と利用は、市場で広く普及している動向の一つです。遺伝子治療は、罹患細胞に機能的遺伝子を導入することにより、これらの疾患の原因となる遺伝子変異を修正することを目的としています。いくつかの臨床試験が成功し、患者の疾患症状の大幅な改善と輸血依存度の低下が実証されています。さらに、CRISPR-Cas9のような遺伝子編集技術の出現は、患者自身の細胞内の欠陥遺伝子を正確に修正する大きな可能性を秘めています。この革新的なアプローチは、前臨床試験で有望な結果を示しており、近い将来、異常ヘモグロビン症に対する根治的な選択肢を提供するかもしれないです。さらに、輸血プロトコール、鉄キレート療法、幹細胞移植技術の向上などの支持療法の進歩は、異常ヘモグロビン症患者の予後改善に貢献し続けています。

異常ヘモグロビン症を治療するための新規治療法開発への投資や資金調達の増加は、予測期間中の市場成長をさらに押し上げると思われます。例えば、2023年5月、米国上院議員は、増加する鎌状赤血球症患者の生活の質を向上させ、鎌状赤血球症を治療する医療スタッフを支援するサービスのための資金を提供する法案、Sickle Cell Care Expansion Actを提出しました。さらに、ブロンクス血液研究基金（BBRF）のような民間の資金援助は、サラセミアやその他の異常ヘモグロビン症の研究と管理のための基盤を提供しています。

治療費の高騰は市場に悪影響を及ぼす可能性があります。例えば、米国国立衛生研究所（National Institutes of Health）の新しい調査によると、鎌状赤血球症を患っている64歳以下のアメリカ人は、鎌状赤血球症を患っていない人に比べ、一生涯にかかる医療費の自己負担額が約44,000米ドル多いです。さらに、保険会社は鎌状赤血球症患者1人につき平均170万米ドルを負担しています。

異常ヘモグロビン症市場レポート・ハイライト

• 2022年の異常ヘモグロビン症市場において、鎌状赤血球病セグメントは58.6%の最大シェアを占め、予測期間中最も速い速度で成長すると予測されています。この背景には、鎌状赤血球症治療へのアクセス改善に焦点を当てたバイオ医薬品企業や非営利団体によるイニシアチブの高まりがあります。
• 鎌状赤血球症診断分野は異常ヘモグロビン症市場を独占し、北米と欧州における高い診断率により、2022年には50.5％のシェアを占めました。
• 治療法別では、鎌状赤血球症分野が2022年に62.1％の最大売上シェアを占めました。鎌状赤血球症を治療するための新規治療法開発のための政府資金と相まった広範な研究が、同分野の成長に寄与する主な要因です。
• 北米は、異常ヘモグロビン症に対する認識の高まり、医療施設の改善、同地域における新製品の発売が、同地域の成長を増大させる主な要因となっているため、2022年には37.7%の最大収益シェアを占めました。
• アジア太平洋地域は、予測期間中最も速い成長率を示すと予測されます。鎌状赤血球症やサラセミアに罹患する患者が多いことが、今後数年間の市場成長を促進すると予想されます。

目次

第1章 調査手法と範囲

第2章 エグゼクティブサマリー

第3章 市場変数、動向、および範囲

• 市場系統の見通し
  • 親市場の見通し
  • 関連/付随市場の見通し
• 市場力学
  • 市場促進要因の分析
  • 市場抑制要因分析
• 業界分析ツール
  • ポーターのファイブフォース分析
  • PESTEL分析
  • 新型コロナウイルス感染症（COVID-19）の影響分析

第4章 タイプのビジネス分析

• 異常ヘモグロビン症市場：タイプの変動分析
• サラセミア
• 鎌状赤血球症
• Hbバリアント

第5章 診断のビジネス分析

• 異常ヘモグロビン症市場：診断の変動分析
• サラセミア
• 鎌状赤血球症
• Hbバリアント

第6章 治療のビジネス分析

• 異常ヘモグロビン症市場：治療の変動分析
• サラセミア
• 鎌状赤血球症
• Hbバリアント

第7章 地域のビジネス分析

• 地域別の異常ヘモグロビン症市場シェア、2022年および2030年
• 北米
• 欧州
• アジア太平洋地域
• ラテンアメリカ
• 中東・アフリカ

第8章 競合情勢

• 参入企業
  • Sangamo Therapeutics
  • Bluebird bio, Inc.
  • Global Blood Therapeutics, Inc
  • Pfizer, Inc.
  • Emmaus Life Sciences, Inc.
  • Prolong Pharmaceuticals, LLC
  • Celgene Corporation (BMS)
  • Bioverativ, Inc. (Sanofi)
  • Gamida Cell
  • Novartis AG
• 参入企業の概要
• 財務実績
• 製品のベンチマーク
• 企業の市場シェア分析、2022年
• 戦略マッピング

第9章 アナリストの視点

List of Tables

• Table 1 List of secondary sources
• Table 2 List of abbreviations
• Table 3 Global hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 4 Global hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 5 Global hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 6 Global hemoglobinopathies market, by region, 2018 - 2030 (USD Million)
• Table 7 North America hemoglobinopathies market, by country, 2018 - 2030 (USD Million)
• Table 8 North America hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 9 North America hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 10 North America hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 11 U.S. hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 12 U.S. hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 13 U.S. hemoglobinopathies Market, by therapy, 2018 - 2030 (USD Million)
• Table 14 Canada hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 15 Canada hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 16 Canada hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 17 Europe hemoglobinopathies market, by country, 2018 - 2030 (USD Million)
• Table 18 Europe hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 19 Europe hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 20 Europe hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 21 UK hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 22 UK hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 23 UK hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 24 Germany hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 25 Germany hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 26 Germany hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 27 France hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 28 France hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 29 France hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 30 Italy hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 31 Italy hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 32 Italy hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 33 Spain hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 34 Spain hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 35 Spain hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 36 Denmark hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 37 Denmark hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 38 Denmark hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 39 Sweden hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 40 Sweden hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 41 Sweden hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 42 Norway hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 43 Norway hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 44 Norway hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 45 Asia Pacific hemoglobinopathies market, by country, 2018 - 2030 (USD Million)
• Table 46 Asia Pacific hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 47 Asia Pacific hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 48 Asia Pacific hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 49 Japan hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 50 Japan hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 51 Japan hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 52 China hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 53 China hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 54 China hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 55 India hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 56 India hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 57 India hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 58 South Korea hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 59 South Korea hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 60 South Korea hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 61 Australia hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 62 Australia hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 63 Australia hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 64 Singapore hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 65 Singapore hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 66 Singapore hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 67 Thailand hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 68 Thailand hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 69 Thailand hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 70 Latin America hemoglobinopathies market, by country, 2018 - 2030 (USD Million)
• Table 71 Latin America hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 72 Latin America hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 73 Latin America hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 74 Brazil hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 75 Brazil hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 76 Brazil hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 77 Mexico hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 78 Mexico hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 79 Mexico hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 80 Argentina hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 81 Argentina hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 82 Argentina hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 83 Middle East & Africa hemoglobinopathies market, by country, 2018 - 2030 (USD Million)
• Table 84 Middle East & Africa hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 85 Middle East & Africa hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 86 Middle East & Africa hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 87 South Africa hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 88 South Africa hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 89 South Africa hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 90 UAE hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 91 UAE hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 92 UAE hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 93 Saudi Arabia hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 94 Saudi Arabia hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 95 Saudi Arabia hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 96 Kuwait hemoglobinopathies market, by type, 2018 - 2030 (USD Million)
• Table 97 Kuwait hemoglobinopathies market, by diagnosis, 2018 - 2030 (USD Million)
• Table 98 Kuwait hemoglobinopathies market, by therapy, 2018 - 2030 (USD Million)
• Table 99 Company Overview
• Table 100 Financial Performance
• Table 101 Product Benchmarking
• Table 102 Strategic Initiatives

List of Figures

• Fig. 1 Hemoglobinopathies market segmentation
• Fig. 2 Market research process
• Fig. 3 Information procurement
• Fig. 4 Primary research pattern
• Fig. 5 Market research approaches
• Fig. 6 Value chain - based sizing & forecasting
• Fig. 7 Market formulation & validation
• Fig. 8 Hemoglobinopathies market segmentation
• Fig. 9 Market snapshot, 2022
• Fig. 10 Market trends & outlook
• Fig. 11 Market driver relevance analysis (current & future impact)
• Fig. 12 Market restraint relevance analysis (current & future impact)
• Fig. 13 PESTEL analysis
• Fig. 14 Porter's five forces analysis
• Fig. 15 Global Hemoglobinopathies market: Type movement analysis
• Fig. 16 Global Hemoglobinopathies market, for thalassemia, 2018 - 2030 (USD Million)
• Fig. 17 Global Hemoglobinopathies market, for sickle cell disease, 2018 - 2030 (USD Million)
• Fig. 18 Global Hemoglobinopathies market, for Hb variants, 2018 - 2030 (USD Million)
• Fig. 19 Global Hemoglobinopathies market: Diagnosis movement analysis
• Fig. 20 Global Hemoglobinopathies market, for thalassemia, 2018 - 2030 (USD Million)
• Fig. 21 Global Hemoglobinopathies market, for sickle cell disease, 2018 - 2030 (USD Million)
• Fig. 22 Global Hemoglobinopathies market, for Hb variants, 2018 - 2030 (USD Million)
• Fig. 23 Global Hemoglobinopathies market: Therapy movement analysis
• Fig. 24 Global Hemoglobinopathies market, for thalassemia, 2018 - 2030 (USD Million)
• Fig. 25 Global Hemoglobinopathies market, for sickle cell disease, 2018 - 2030 (USD Million)
• Fig. 26 Global Hemoglobinopathies market, for other Hb variants, 2018 - 2030 (USD Million)
• Fig. 27 Regional marketplace: Key takeaways
• Fig. 28 Regional outlook, 2022 & 2030
• Fig. 29 Global Hemoglobinopathies market: Region movement analysis
• Fig. 30 North America Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 31 U.S. Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 32 Canada Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 33 Europe Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 34 Germany Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 35 UK Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 36 France Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 37 Italy Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 38 Spain Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 39 Denmark Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 40 Sweden Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 41 Norway Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 42 Asia Pacific Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 43 Japan Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 44 China Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 45 India Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 46 Australia Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 47 South Korea Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 48 Thailand Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 49 Singapore Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 50 Latin America Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 51 Brazil Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 52 Mexico Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 53 Argentina Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 54 Middle East and Africa Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 55 South Africa Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 56 Saudi Arabia Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 57 UAE Hemoglobinopathies market, 2018 - 2030 (USD Million)
• Fig. 58 Kuwait Hemoglobinopathies market, 2018 - 2030 (USD Million)

Hemoglobinopathies Market Growth & Trends:

The global hemoglobinopathies market size is expected to reach USD 20.70 billion by 2030 and is expected to expand at a CAGR of 12.6% from 2023 to 2030, according to a new report by Grand View Research, Inc.. The market is experiencing steady growth due to increasing prevalence of disorders like sickle cell disease (SCD) and thalassemia. The driving factors include advancements in diagnostic techniques, growing awareness, and improving healthcare infrastructure. Additionally, the development of innovative therapies and government initiatives are contributing to market expansion.

In recent years, there have been significant advancements in diagnostic techniques for sickle cell disease. One prominent trend is the increased utilization of molecular genetic testing, specifically DNA analysis, to identify specific mutations associated with the disease. This approach allows for precise and accurate diagnosis, enabling healthcare professionals to determine the presence of sickle cell trait or SCD in individuals. Additionally, technological advancements have led to the development of point-of-care testing devices, enabling rapid screening and diagnosis in resource-limited settings. These advancements in diagnostic techniques not only enhance early detection and intervention but also facilitate genetic counseling and family planning for individuals at risk of passing on the disease.

Advancements in therapy for hemoglobinopathies, including SCD and thalassemia, have shown promising trends in recent years. Development and utilization of gene therapy approaches is one of the prevalent trend in the market. Gene therapy aims to correct the genetic mutations responsible for these conditions by introducing functional genes into affected cells. Several successful clinical trials have demonstrated significant improvements in disease symptoms and reduced transfusion dependence in patients. Additionally, the emergence of gene editing techniques, such as CRISPR-Cas9, holds great potential for precisely modifying faulty genes in a patient's own cells. This innovative approach has shown promising results in preclinical studies and may offer a curative option for hemoglobinopathies in the near future. Furthermore, advancements in supportive care, including transfusion protocols, iron chelation therapy, and improved stem cell transplantation techniques, continue to contribute to enhanced outcomes for patients with hemoglobinopathies.

An increase in investment and funding for the development of novel therapies to treat hemoglobinopathies will further boost the market growth over the forecast period. For instance, in May 2023, U.S. Senators introduced the Sickle Cell Care Expansion Act, legislation to offer funding for services to advance the quality of life for the growing number of patients living with Sickle Cell and support the medical staff that treats sickle cell disease. Furthermore, private funding such as the Bronx Blood Research Fund (BBRF) provides a platform for research and management of thalassemia and other hemoglobinopathies.

High cost of treatment might an adverse impact on the market. For instance, according to new research supported by the National Institutes of Health, living with sickle cell disease costs Americans ages 64 and under with commercial health insurance about USD 44,000 more in out-of-pocket medical expenses over their lifetimes than people without the condition. Additionally, insurers shell out an average of USD 1.7 million for each sickle cell disease patient.

Hemoglobinopathies Market Report Highlights:

• The sickle cell disease segment held the largest share of 58.6% in 2022 of the hemoglobinopathies market and is expected to grow at the fastest rate during the forecast period. This is attributed to growing initiatives by biopharmaceutical companies and nonprofit organizations focused on improving access to SCD treatment
• The sickle cell disease diagnosis segment dominated the hemoglobinopathies market and accounted for a 50.5% share in 2022 owing to high diagnostic rates in North America and Europe
• By therapy, the sickle cell disease segment held the largest revenue share of 62.1% in 2022. Extensive research coupled with government funding for the development of novel therapies for treating the disorder is the major factor contributing to the segment growth
• North America held the largest revenue share of 37.7% in 2022 owing to increasing awareness of hemoglobinopathies, improving healthcare facilities and launch of new products in the region are the major factor augmenting the regional growth
• Asia Pacific is estimated to exhibit the fastest growth rate over the forecast period. The presence of a large number of patients suffering from sickle cell disease and thalassemia is expected to propel market growth in the coming years
• Major players in the market are Sangamo Therapeutics, bluebird bio, Inc., Global Blood Therapeutics, Inc., Pfizer, Inc., Emmaus Life Sciences, Inc., Prolong Pharmaceuticals, LLC, Celgene Corporation, Bioverativ Inc., Gamida Cell and Novartis AG

Table of Contents

Chapter 1. Methodology and Scope

• 1.1. Market Segmentation and Scope
  • 1.1.1. Segment Definitions
    • 1.1.1.1. Type segment
    • 1.1.1.2. Diagnosis segment
    • 1.1.1.3. Therapy segment
• 1.2. Regional Scope
• 1.3. Estimates and Forecast Timeline
• 1.4. Objectives
  • 1.4.1. Objective - 1
  • 1.4.2. Objective - 2
  • 1.4.3. Objective - 3
• 1.5. Research Methodology
• 1.6. Information Procurement
  • 1.6.1. Purchased Database
  • 1.6.2. GVR's Internal Database
  • 1.6.3. Secondary Sources
  • 1.6.4. Primary research
• 1.7. Information or Data Analysis
  • 1.7.1. Data Analysis Models
• 1.8. Market Formulation & Validation
• 1.9. Model Details
  • 1.9.1. Commodity Flow Analysis
• 1.10. List of Secondary Sources
• 1.11. List of Abbreviations

Chapter 2. Executive Summary

• 2.1. Market Snapshot
• 2.2. Type Snapshot
• 2.3. Diagnosis and Therapy Snapshot
• 2.4. Competitive Landscape Snapshot

Chapter 3. Market Variables, Trends, & Scope

• 3.1. Market Lineage Outlook
  • 3.1.1. Parent Market Outlook
  • 3.1.2. Related/Ancillary Market Outlook
• 3.2. Market Dynamics
  • 3.2.1. Market Driver Analysis
    • 3.2.1.1. Increasing prevalence of hemoglobinopathies in under - developed countries
    • 3.2.1.2. High unmet needs
    • 3.2.1.3. Increasing R&D investments
    • 3.2.1.4. Initiatives to improve disease awareness levels
  • 3.2.2. Market Restraint Analysis
    • 3.2.2.1. Lack of healthcare infrastructure
    • 3.2.2.2. High cost of treatment
• 3.3. Industry Analysis Tools
  • 3.3.1. Porter's Five Forces Analysis
  • 3.3.2. PESTEL Analysis
  • 3.3.3. COVID - 19 Impact Analysis

Chapter 4. Type Business Analysis

• 4.1. Hemoglobinopathies Market: Type Movement Analysis
• 4.2. Thalassemia
  • 4.2.1. Thalassemia Market, 2018 - 2030 (USD Million)
• 4.3. Sickle Cell Disease
  • 4.3.1. Sickle Cell Disease Market, 2018 - 2030 (USD Million)
• 4.4. Hb Variants
  • 4.4.1. Hb Variants Market, 2018 - 2030 (USD Million)

Chapter 5. Diagnosis Business Analysis

• 5.1. Hemoglobinopathies Market: Diagnosis Movement Analysis
• 5.2. Thalassemia
  • 5.2.1. Thalassemia Market, 2018 - 2030 (USD Million)
• 5.3. Sickle Cell Disease
  • 5.3.1. Sickle Cell Disease Market, 2018 - 2030 (USD Million)
• 5.4. Hb Variants
  • 5.4.1. Hb Variants Market, 2018 - 2030 (USD Million)

Chapter 6. Therapy Business Analysis

• 6.1. Hemoglobinopathies Market: Therapy Movement Analysis
• 6.2. Thalassemia
  • 6.2.1. Thalassemia Market, 2018 - 2030 (USD Million)
• 6.3. Sickle Cell Disease
  • 6.3.1. Sickle Cell Disease Market, 2018 - 2030 (USD Million)
• 6.4. Other Hb Variants
  • 6.4.1. Other Hb Variants Market, 2018 - 2030 (USD Million)

Chapter 7. Regional Business Analysis

• 7.1. Hemoglobinopathies Market Share By Region, 2022 & 2030
• 7.2. North America
  • 7.2.1. SWOT Analysis
  • 7.2.2. North America Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.2.3. U.S.
    • 7.2.3.1. Key Country Dynamics
    • 7.2.3.2. Target Disease Prevalence
    • 7.2.3.3. Competitive Scenario
    • 7.2.3.4. Regulatory Framework
    • 7.2.3.5. U.S. Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.2.4. Canada
    • 7.2.4.1. Key Country Dynamics
    • 7.2.4.2. Target Disease Prevalence
    • 7.2.4.3. Competitive Scenario
    • 7.2.4.4. Regulatory Framework
    • 7.2.4.5. Canada Hemoglobinopathies Market, 2018 - 2030 (USD Million)
• 7.3. Europe
  • 7.3.1. Europe Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.2. UK
    • 7.3.2.1. Key Country Dynamics
    • 7.3.2.2. Target Disease Prevalence
    • 7.3.2.3. Competitive Scenario
    • 7.3.2.4. Regulatory Framework
    • 7.3.2.5. UK Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.3. Germany
    • 7.3.3.1. Key Country Dynamics
    • 7.3.3.2. Target Disease Prevalence
    • 7.3.3.3. Competitive Scenario
    • 7.3.3.4. Regulatory Framework
    • 7.3.3.5. Germany Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.4. France
    • 7.3.4.1. Key Country Dynamics
    • 7.3.4.2. Target Disease Prevalence
    • 7.3.4.3. Competitive Scenario
    • 7.3.4.4. Regulatory Framework
    • 7.3.4.5. France Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.5. Italy
    • 7.3.5.1. Key Country Dynamics
    • 7.3.5.2. Target Disease Prevalence
    • 7.3.5.3. Competitive Scenario
    • 7.3.5.4. Regulatory Framework
    • 7.3.5.5. Italy Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.6. Spain
    • 7.3.6.1. Key Country Dynamics
    • 7.3.6.2. Target Disease Prevalence
    • 7.3.6.3. Competitive Scenario
    • 7.3.6.4. Regulatory Framework
    • 7.3.6.5. Spain Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.7. Denmark
    • 7.3.7.1. Key Country Dynamics
    • 7.3.7.2. Target Disease Prevalence
    • 7.3.7.3. Competitive Scenario
    • 7.3.7.4. Regulatory Framework
    • 7.3.7.5. Denmark Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.8. Sweden
    • 7.3.8.1. Key Country Dynamics
    • 7.3.8.2. Target Disease Prevalence
    • 7.3.8.3. Competitive Scenario
    • 7.3.8.4. Regulatory Framework
    • 7.3.8.5. Sweden Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.9. Norway
    • 7.3.9.1. Key Country Dynamics
    • 7.3.9.2. Target Disease Prevalence
    • 7.3.9.3. Competitive Scenario
    • 7.3.9.4. Regulatory Framework
    • 7.3.9.5. Norway Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.3.10. Russia
    • 7.3.10.1. Key Country Dynamics
    • 7.3.10.2. Target Disease Prevalence
    • 7.3.10.3. Competitive Scenario
    • 7.3.10.4. Regulatory Framework
    • 7.3.10.5. Russia Hemoglobinopathies Market, 2018 - 2030 (USD Million)
• 7.4. Asia Pacific
  • 7.4.1. Asia Pacific Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.2. Japan
    • 7.4.2.1. Key Country Dynamics
    • 7.4.2.2. Target Disease Prevalence
    • 7.4.2.3. Competitive Scenario
    • 7.4.2.4. Regulatory Framework
    • 7.4.2.5. Japan Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.3. China
    • 7.4.3.1. Key Country Dynamics
    • 7.4.3.2. Target Disease Prevalence
    • 7.4.3.3. Competitive Scenario
    • 7.4.3.4. Regulatory Framework
    • 7.4.3.5. China Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.4. India
    • 7.4.4.1. Key Country Dynamics
    • 7.4.4.2. Target Disease Prevalence
    • 7.4.4.3. Competitive Scenario
    • 7.4.4.4. Regulatory Framework
    • 7.4.4.5. India Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.5. Australia
    • 7.4.5.1. Key Country Dynamics
    • 7.4.5.2. Target Disease Prevalence
    • 7.4.5.3. Competitive Scenario
    • 7.4.5.4. Regulatory Framework
    • 7.4.5.5. Australia Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.6. Thailand
    • 7.4.6.1. Key Country Dynamics
    • 7.4.6.2. Target Disease Prevalence
    • 7.4.6.3. Competitive Scenario
    • 7.4.6.4. Regulatory Framework
    • 7.4.6.5. Thailand Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.7. South Korea
    • 7.4.7.1. Key Country Dynamics
    • 7.4.7.2. Target Disease Prevalence
    • 7.4.7.3. Competitive Scenario
    • 7.4.7.4. Regulatory Framework
    • 7.4.7.5. South Korea Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.4.8. Singapore
    • 7.4.8.1. Key Country Dynamics
    • 7.4.8.2. Target Disease Prevalence
    • 7.4.8.3. Competitive Scenario
    • 7.4.8.4. Regulatory Framework
    • 7.4.8.5. Singapore Hemoglobinopathies Market, 2018 - 2030 (USD Million)
• 7.5. Latin America
  • 7.5.1. Latin America Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.5.2. Brazil
    • 7.5.2.1. Key Country Dynamics
    • 7.5.2.2. Target Disease Prevalence
    • 7.5.2.3. Competitive Scenario
    • 7.5.2.4. Regulatory Framework
    • 7.5.2.5. Brazil Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.5.3. Mexico
    • 7.5.3.1. Key Country Dynamics
    • 7.5.3.2. Target Disease Prevalence
    • 7.5.3.3. Competitive Scenario
    • 7.5.3.4. Regulatory Framework
    • 7.5.3.5. Mexico Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.5.4. Argentina
    • 7.5.4.1. Key Country Dynamics
    • 7.5.4.2. Target Disease Prevalence
    • 7.5.4.3. Competitive Scenario
    • 7.5.4.4. Regulatory Framework
    • 7.5.4.5. Argentina Hemoglobinopathies Market, 2018 - 2030 (USD Million)
• 7.6. MEA
  • 7.6.1. MEA Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.6.2. South Africa
    • 7.6.2.1. Key Country Dynamics
    • 7.6.2.2. Target Disease Prevalence
    • 7.6.2.3. Competitive Scenario
    • 7.6.2.4. Regulatory Framework
    • 7.6.2.5. South Africa Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.6.3. Saudi Arabia
    • 7.6.3.1. Key Country Dynamics
    • 7.6.3.2. Target Disease Prevalence
    • 7.6.3.3. Competitive Scenario
    • 7.6.3.4. Regulatory Framework
    • 7.6.3.5. Saudi Arabia Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.6.4. UAE
    • 7.6.4.1. Key Country Dynamics
    • 7.6.4.2. Target Disease Prevalence
    • 7.6.4.3. Competitive Scenario
    • 7.6.4.4. Regulatory Framework
    • 7.6.4.5. UAE Hemoglobinopathies Market, 2018 - 2030 (USD Million)
  • 7.6.5. Kuwait
    • 7.6.5.1. Key Country Dynamics
    • 7.6.5.2. Target Disease Prevalence
    • 7.6.5.3. Competitive Scenario
    • 7.6.5.4. Regulatory Framework
    • 7.6.5.5. Kuwait Hemoglobinopathies Market, 2018 - 2030 (USD Million)

Chapter 8. Competitive Landscape

• 8.1. Participant Categorization
  • 8.1.1. Sangamo Therapeutics
    • 8.1.1.1. Overview
    • 8.1.1.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.1.3. Product Benchmarking
    • 8.1.1.4. Strategic Initiatives
  • 8.1.2. Bluebird bio, Inc.
    • 8.1.2.1. Overview
    • 8.1.2.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.2.3. Product Benchmarking
    • 8.1.2.4. Strategic Initiatives
  • 8.1.3. Global Blood Therapeutics, Inc
    • 8.1.3.1. Overview
    • 8.1.3.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.3.3. Product Benchmarking
    • 8.1.3.4. Strategic Initiatives
  • 8.1.4. Pfizer, Inc.
    • 8.1.4.1. Overview
    • 8.1.4.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.4.3. Product Benchmarking
    • 8.1.4.4. Strategic Initiatives
  • 8.1.5. Emmaus Life Sciences, Inc.
    • 8.1.5.1. Overview
    • 8.1.5.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.5.3. Product Benchmarking
    • 8.1.5.4. Strategic Initiatives
  • 8.1.6. Prolong Pharmaceuticals, LLC
    • 8.1.6.1. Overview
    • 8.1.6.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.6.3. Product Benchmarking
    • 8.1.6.4. Strategic Initiatives
  • 8.1.7. Celgene Corporation (BMS)
    • 8.1.7.1. Overview
    • 8.1.7.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.7.3. Product Benchmarking
    • 8.1.7.4. Strategic Initiatives
  • 8.1.8. Bioverativ, Inc. (Sanofi)
    • 8.1.8.1. Overview
    • 8.1.8.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.8.3. Product Benchmarking
    • 8.1.8.4. Strategic Initiatives
  • 8.1.9. Gamida Cell
    • 8.1.9.1. Overview
    • 8.1.9.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.9.3. Product Benchmarking
    • 8.1.9.4. Strategic Initiatives
  • 8.1.10. Novartis AG
    • 8.1.10.1. Overview
    • 8.1.10.2. Financial Performance (Net Revenue/Sales/EBITDA/Gross Profit)
    • 8.1.10.3. Product Benchmarking
    • 8.1.10.4. Strategic Initiatives
• 8.2. Participant's Overview
• 8.3. Financial Performance
• 8.4. Product Benchmarking
• 8.5. Company Market Share Analysis, 2022
• 8.6. Strategy Mapping
  • 8.6.1. Expansion
  • 8.6.2. Acquisition
  • 8.6.3. Product/Service Launch
  • 8.6.4. Others

Chapter 9. Analyst View