Global Gene Therapy for Blood Disorders Market

Report ID : 1156 | Published : 2021-07-27 | Pages: 140 | Format: PDF/EXCEL

The Arena of Gene Therapy is determined to outline a path to the clinic and the market. In the gene therapy market, over 2000 human gene therapy clinical trials have been reported globally, and about 20 gene therapy products are approved to treat various disorders. Hence, the demand for gene therapy to treat blood disorders is increasing. Gene therapy accounts great to cure blood disorders such as sickle cell disease and hemophilia, which have inadequate treatment choices. The gene therapy helps to understand the detailed pathomechanisms of conditions as well as the advancement of specific and efficient gene targeting and delivery tools that are transforming the global market. The approval of gene therapy for the treatment of β-thalassemia in Europe is propelling the research activities for the approval of the drug in different regions and new drug development. The various global biopharmaceutical companies are strategically taking initiatives to develop advanced therapy such as gene therapy for severe blood disorders. This is expected to boost market growth significantly in the coming years.


Globally around 35 companies are working on regenerative medicine therapies for hereditary blood disorders, counting 18 clinical-stage companies. Additionally, the presence of strong pipeline drugs for the treatment of blood disorders with gene therapy is estimated to drive the growth of the market in the coming years. The global Gene Therapy for Blood Disorders market size is expected to reach US$ xx million by 2028, growing at a CAGR of xx% over the forecast period. Additionally, growing strategic partnership, acquisitions among the key players operating in the market is expected to propel the market growth significantly. However, the high cost of treatment is the major factor expected to restrain the growth of gene therapy in the blood disorders market. However, increasing demand for personalized medicine and growing healthcare expenditure globally is estimated to drive the market growth substantially during the estimated time frame.
The Global Gene Therapy for Blood Disorders market is segmented on the basis of indication, mechanism of action, and region. Based on the indication, the market is divided into Sickle Cell Anemia, β-thalassemia, Hemophilia B, and Hemophilia A.
Based on the mechanism of action, the market is segmented into gene therapy and gene editing. Based on the region, the market is studied across North America, Asia-Pacific, Europe, and LAMEA. Among them, North America accounted for the largest share of the market, followed by Europe. The well-established healthcare system in the U.S. and the presence of a large number of companies operating in the market are propelling the growth of the U.S. gene therapy for blood disorders market.
The key players of this market include Biomarin Therapeutics, Spark Therapeutics, Shire, bluebird bio, Inc., CRISPR Therapeutics, Medgenics, OrphageniX, Freeline Therapeutics, Errant Gene Therapeutics, Sangamo Therapeutics, Sigilon Therapeutics, Inc., uniQure, Angiocrine Bioscience, and Sanofi Genzyme among others. The growing collaboration among various research centers and companies to develop a gene therapy to treat severe blood disorders. Gene therapy has enormous potential for blood disorders such as hemophilia and is becoming a large market. Some corporations, such as CRISPR Therapeutics, are revolving to the gene-editing technique CRISPR-Cas9 as a tool for fighting blood disorders. The company is conducting a phase I/II trial for its CRISPR tool to cure sickle cell disease in partnership with the German company Vertex Pharmaceuticals.

Chapter 1. Methodology and Scope

1.1. Research Methodology

1.2. Research Scope & Assumptions

Chapter 2. Executive Summary

Chapter 3. Global Gene Therapy for Blood Disorders Market Snapshot

Chapter 4. Global Gene Therapy for Blood Disorders Market Variables, Trends & Scope

4.1. Market Segmentation & Scope

4.2. Drivers

4.3. Challenges

4.4. Trends

4.5. Penetration & Growth Prospect Mapping

4.6. Clinical Trial/Pipeline Analysis

4.7. Industry Analysis – Porter’s Five Forces Analysis

4.8. Competitive Landscape & Market Share Analysis

4.9. Technology Advancement in Gene Therapy for Blood Disorders Market

4.10. Key Trends in the Market

Chapter 5. Market Segmentation 1: Indication Estimates & Trend Analysis

5.1. Indication Type & Market Share, 2020 & 2030

5.2. Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2020 to 2030 for the following Indication:

5.2.1. Sickle Cell Anemia

5.2.2. β-thalassemia

5.2.3. Hemophilia B

5.2.4. Hemophilia A

Chapter 6. Market Segmentation 1: Mechanism of Action Estimates & Trend Analysis

6.1. Mechanism of Action Type & Market Share, 2020 & 2030

6.2. Market Size (Value US$ Mn) & Forecasts and Trend Analyses, 2020 to 2030 for the following Mechanism of Action:

6.2.1. Gene Therapy

6.2.2. Gene Editing

Chapter 7. Gene Therapy for Blood Disorders Market Segmentation 3: Regional Estimates & Trend Analysis

7.1. North America

7.1.1. North America Gene Therapy for Blood Disorders Market revenue (US$ Million) estimates and forecasts by Indication, 2020-2030

7.1.2. North America Gene Therapy for Blood Disorders Market revenue (US$ Million) estimates and forecasts by Mechanism of Action, 2020-2030

7.1.3. North America Gene Therapy for Blood Disorders Market revenue (US$ Million) estimates and forecasts by country, 2020-2030

7.1.3.1. U.S.

7.1.3.2. Canada

7.2. Europe

7.2.1. Europe Gene Therapy for Blood Disorders Market revenue (US$ Million) by Indication, 2020-2030

7.2.2. Europe Gene Therapy for Blood Disorders Market revenue (US$ Million) by Mechanism of Action, 2020-2030

7.2.3. Europe Gene Therapy for Blood Disorders Market revenue (US$ Million) by country, 2020-2030

7.2.3.1. Germany

7.2.3.2. Poland

7.2.3.3. France

7.2.3.4. Italy

7.2.3.5. Spain

7.2.3.6. UK

7.2.3.7. Rest of Europe

7.3. Asia Pacific

7.3.1. Asia Pacific Gene Therapy for Blood Disorders Market revenue (US$ Million) by Indication, 2020-2030

7.3.2. Asia Pacific Gene Therapy for Blood Disorders Market revenue (US$ Million) by Mechanism of Action, 2020-2030

7.3.3. Asia Pacific Gene Therapy for Blood Disorders Market revenue (US$ Million) by country, 2020-2030

7.3.3.1. China

7.3.3.2. India

7.3.3.3. Japan

7.3.3.4. Australia

7.3.3.5. Rest of Asia Pacific

7.4. Latin America

7.4.1. Latin America Gene Therapy for Blood Disorders Market revenue (US$ Million) by Indication, (US$ Million)

7.4.2. Latin America Gene Therapy for Blood Disorders Market revenue (US$ Million) by Mechanism of Action, (US$ Million)

7.4.3. Latin America Gene Therapy for Blood Disorders Market revenue (US$ Million) by country, (US$ Million) 2020-2030

7.4.3.1. Brazil

7.4.3.2. Rest of Latin America

7.5. MEA

7.5.1. MEA revenue Gene Therapy for Blood Disorders Market revenue (US$ Million) by Indication, (US$ Million) 2020-2030

Chapter 8. Competitive Landscape

8.1. Major Mergers and Acquisitions/Strategic Alliances

8.2. Company Profiles

8.2.1. Biomarin Therapeutics

8.2.2. Spark Therapeutics

8.2.3. Shire

8.2.4. bluebird bio, Inc.

8.2.5. CRISPR Therapeutics

8.2.6. Expression Therapeutics LLC

8.2.7. Medgenics

8.2.8. OrphageniX

8.2.9. Freeline Therapeutics

8.2.10. Errant Gene Therapeutics

8.2.11. Sangamo Therapeutics

8.2.12. Sigilon Therapeutics, Inc.

8.2.13. uniQure

8.2.14. Angiocrine Bioscience

8.2.15. Sanofi Genzyme

8.2.16. Other Prominent Players

Global Gene Therapy for Blood Disorders Market Segmentation

Global Gene Therapy for Blood Disorders Market by Indication

  • Sickle Cell Anemia
  • β-thalassemia
  • Hemophilia B
  • Hemophilia A

Global Gene Therapy for Blood Disorders Market Based on Mechanism of Action

  • Gene Therapy
  • Gene Editing

Global Gene Therapy for Blood Disorders Market Based on Region

Europe

  • U.K.
  • Germany
  • France
  • Italy
  • Spain
  • Russia
  • Rest of Europe

North America

  • U.S.
  • Canada

Asia Pacific

  • India
  • China
  • Japan
  • South Korea
  • Australia & New Zealand

Latin America

  • Brazil
  • Mexico
  • Rest of Latin America

Middle East & Africa

  • GCC Countries
  • South Africa
  • Rest of Middle East & Africa

 

InsightAce Analytic follows a standard and comprehensive market research methodology focused on offering the most accurate and precise market insights. The methods followed for all our market research studies include three significant steps – primary research, secondary research, and data modeling and analysis - to derive the current market size and forecast it over the forecast period. In this study, these three steps were used iteratively to generate valid data points (minimum deviation), which were cross-validated through multiple approaches mentioned below in the data modeling section.

Through secondary research methods, information on the market under study, its peer, and the parent market was collected. This information was then entered into data models. The resulted data points and insights were then validated by primary participants.

Based on additional insights from these primary participants, more directional efforts were put into doing secondary research and optimize data models. This process was repeated till all data models used in the study produced similar results (with minimum deviation). This way, this iterative process was able to generate the most accurate market numbers and qualitative insights.

Secondary research

The secondary research sources that are typically mentioned to include, but are not limited to:

  • Company websites, financial reports, annual reports, investor presentations, broker reports, and SEC filings.
  • External and internal proprietary databases, regulatory databases, and relevant patent analysis
  • Statistical databases, National government documents, and market reports
  • Press releases, news articles, and webcasts specific to the companies operating in the market

The paid sources for secondary research like Factiva, OneSource, Hoovers, and Statista

Primary Research:

Primary research involves telephonic interviews, e-mail interactions, as well as face-to-face interviews for each market, category, segment, and subsegment across geographies

The contributors who typically take part in such a course include, but are not limited to: 

  • Industry participants: CEOs, CBO, CMO, VPs, marketing/ type managers, corporate strategy managers, and national sales managers, technical personnel, purchasing managers, resellers, and distributors.
  • Outside experts: Valuation experts, Investment bankers, research analysts specializing in specific markets
  • Key opinion leaders (KOLs) specializing in unique areas corresponding to various industry verticals
  • End-users: Vary mainly depending upon the market

Data Modeling and Analysis:

In the iterative process (mentioned above), data models received inputs from primary as well as secondary sources. But analysts working on these models were the key. They used their extensive knowledge and experience about industry and topic to make changes and fine-tuning these models as per the product/service under study.

The standard data models used while studying this market were the top-down and bottom-up approaches and the company shares analysis model. However, other methods were also used along with these – which were specific to the industry and product/service under study.

To know more about the research methodology used for this study, kindly contact us/click here.

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