The global market for Inherited Orphan Blood Disorders Therapeutics was valued at US$ 1875 million in the year 2024 and is projected to reach a revised size of US$ 3540 million by 2031, growing at a CAGR of 9.9% during the forecast period.

Inherited Orphan Blood Disorders Therapeutics Market

Inherited Orphan Blood Disorders Therapeutics refers to the portfolio of treatments developed for congenital or genetically driven abnormalities in hematopoiesis, blood cell function, or blood metabolism. This domain encompasses gene therapies, gene editing approaches, cell therapies, targeted small molecules and biologics, as well as supportive care and long-term monitoring programs. Treatments aim not only to alleviate clinical manifestations but increasingly to correct or durably modify underlying causes, emphasizing individualized design and longitudinal safety surveillance. Given the heterogeneity and low prevalence of these disorders, development pathways demand interdisciplinary collaboration, precise patient stratification, and early regulatory engagement to ensure clinical accessibility and sustainable commercial strategies. The field is shifting from symptomatic management toward potentially curative, mechanism-targeted interventions, marking a strategic intersection between rare-disease therapeutics and precision medicine.The average gross profit margin of this product is 83%.

Opportunities in Inherited Orphan Blood Disorders Therapeutics stem from concurrent advances in scientific methodologies and translational capability. The maturation of gene therapy and gene-editing platforms, advances in cell manufacturing and delivery technologies, have enabled causative, mechanism-targeted interventions to progress into regulated clinical development. Improvements in molecular diagnostics and biomarker-driven patient identification enhance patient stratification, reducing trial uncertainty and increasing treatment accessibility. Collaborative ecosystems—comprising academia, industry, patient organizations, and regulators—have fostered accelerated pathways, orphan drug incentives, and innovative reimbursement experiments, creating structural opportunities for companies to capture sustainable value in high-impact yet small-population markets.

Challenges span technical, regulatory, and commercial domains. Scientific uncertainties around long-term safety of gene and cell therapies—such as immunogenicity and insertional risks—necessitate extended follow-up and complex regulatory assessment. Limited patient populations and phenotypic heterogeneity complicate evidence generation and limit reliance on large randomized trials, placing pressure on regulatory approval and payer acceptance. High R&D and manufacturing costs, complex cold-chain logistics, and heterogeneous reimbursement environments across geographies further constrain broad commercialization and equitable access.

Downstream demand is shifting from symptomatic care toward durable disease modification and lifecycle management. Clinicians expect sustained clinical benefit and reduced complication burden, while care pathways must adapt to the long-term monitoring needs of gene and cell therapies. Patient cohorts and advocacy groups play increasingly central roles in trial recruitment, real-world evidence generation, and defining meaningful outcomes, driving patient-centric efficacy frameworks. Payers and health-economics stakeholders emphasize long-term benefit and lifecycle cost-effectiveness, favoring products with demonstrable durable effect and traceable safety profiles.

The upstream supply base includes plasmids and viral packaging systems for vector production, cell culture media and primary cell sources, high-purity biologic inputs (e.g., recombinant proteins, modified nucleotides), and critical manufacturing and quality-control equipment. Production is highly dependent on GMP-grade facilities, scalable and reproducible processes, and rigorous quality systems. Traceability, batch-to-batch consistency, and supply reliability of raw materials directly impact product safety and efficacy. As the field industrializes, upstream specialization and concentration among contract manufacturers and suppliers are growing, creating competitive moats in quality assurance, scalability, and regulatory-compliant services.

This report aims to provide a comprehensive presentation of the global market for Inherited Orphan Blood Disorders Therapeutics, with both quantitative and qualitative analysis, to help readers develop business/growth strategies, assess the market competitive situation, analyze their position in the current marketplace, and make informed business decisions regarding Inherited Orphan Blood Disorders Therapeutics.

The Inherited Orphan Blood Disorders Therapeutics market size, estimations, and forecasts are provided in terms of and revenue ($ millions), considering 2024 as the base year, with history and forecast data for the period from 2020 to 2031. This report segments the global Inherited Orphan Blood Disorders Therapeutics market comprehensively. Regional market sizes, concerning products by Type, by Application, by Treatment Mechanism and by players, are also provided.

For a more in-depth understanding of the market, the report provides profiles of the competitive landscape, key competitors, and their respective market ranks. The report also discusses technological trends and new product developments.

The report will help the Inherited Orphan Blood Disorders Therapeutics companies, new entrants, and industry chain related companies in this market with information on the revenues for the overall market and the sub-segments across the different segments, by company, by Type, by Application, and by regions.

Market Segmentation

Scope of Inherited Orphan Blood Disorders Therapeutics Market Report

Report Metric	Details
Report Name	Inherited Orphan Blood Disorders Therapeutics Market
Accounted market size in year	US$ 1875 million
Forecasted market size in 2031	US$ 3540 million
CAGR	9.9%
Base Year	year
Forecasted years	2025 - 2031
Segment by Type	Hereditary Hemolytic Diseases Coagulation Disorders Metabolic Blood Diseases
Segment by Treatment Mechanism	Gene Replacement Therapy Gene Editing Therapy Cell Therapy Protein Replacement Therapy
Segment by Product Technology	Adeno-associated virus (AAV) Vector Platform Lentiviral Vector Platform CRISPR/Cas
Segment by Application	Hospital Retail Pharmacy Other
By Region	North America (United States, Canada) Europe (Germany, France, UK, Italy, Russia) Rest of Europe Nordic Countries Asia-Pacific (China, Japan, South Korea) Southeast Asia (India, Australia) Rest of Asia Latin America (Mexico, Brazil) Rest of Latin America Middle East & Africa (Turkey, Saudi Arabia, UAE, Rest of MEA)
By Company	Biogen, Novartis, Gilead Sciences, Sarepta Therapeutics, Alnylam Pharmaceuticals, Amgen, Spark Therapeutics, Akcea Therapeutics, Sunway Biotech, SIBIONO, AnGes, Orchard Therapeutics, Human Stem Cells Institute
Forecast units	USD million in value
Report coverage	Revenue and volume forecast, company share, competitive landscape, growth factors and trends

Chapter Outline

Chapter 1: Introduces the report scope of the report, executive summary of different market segments (by Type, by Application, by Treatment Mechanism etc), including the market size of each market segment, future development potential, and so on. It offers a high-level view of the current state of the market and its likely evolution in the short to mid-term, and long term.
Chapter 2: Introduces executive summary of global market size, regional market size, this section also introduces the market dynamics, latest developments of the market, the driving factors and restrictive factors of the market, the challenges and risks faced by companies in the industry, and the analysis of relevant policies in the industry.
Chapter 3: Detailed analysis of Inherited Orphan Blood Disorders Therapeutics company competitive landscape, revenue market share, latest development plan, merger, and acquisition information, etc.
Chapter 4: Provides the analysis of various market segments by Type, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different market segments.
Chapter 5: Provides the analysis of various market segments by Application, covering the market size and development potential of each market segment, to help readers find the blue ocean market in different downstream markets.
Chapter 6, 7, 8, 9, 10: North America, Europe, Asia Pacific, Latin America, Middle East and Africa segment by country. It provides a quantitative analysis of the market size and development potential of each region and its main countries and introduces the market development, future development prospects, market space, and capacity of each country in the world.
Chapter 11: Provides profiles of key players, introducing the basic situation of the main companies in the market in detail, including product sales, revenue, price, gross margin, product introduction, recent development, etc.
Chapter 12: The main points and conclusions of the report.

FAQ for this report

How fast is Inherited Orphan Blood Disorders Therapeutics Market growing?

Ans: The Inherited Orphan Blood Disorders Therapeutics Market witnessing a CAGR of 9.9% during the forecast period 2025-2031.

What is the Inherited Orphan Blood Disorders Therapeutics Market size in 2031?

Ans: The Inherited Orphan Blood Disorders Therapeutics Market size in 2031 will be US$ 3540 million.

Who are the main players in the Inherited Orphan Blood Disorders Therapeutics Market report?

Ans: The main players in the Inherited Orphan Blood Disorders Therapeutics Market are Biogen, Novartis, Gilead Sciences, Sarepta Therapeutics, Alnylam Pharmaceuticals, Amgen, Spark Therapeutics, Akcea Therapeutics, Sunway Biotech, SIBIONO, AnGes, Orchard Therapeutics, Human Stem Cells Institute

What are the Application segmentation covered in the Inherited Orphan Blood Disorders Therapeutics Market report?

Ans: The Applications covered in the Inherited Orphan Blood Disorders Therapeutics Market report are Hospital, Retail Pharmacy, Other

What are the Type segmentation covered in the Inherited Orphan Blood Disorders Therapeutics Market report?

Ans: The Types covered in the Inherited Orphan Blood Disorders Therapeutics Market report are Hereditary Hemolytic Diseases, Coagulation Disorders, Metabolic Blood Diseases

1 Report Overview

1.1 Study Scope

1.2 Market Analysis by Type

1.2.1 Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Type: 2020 VS 2024 VS 2031

1.2.2 Hereditary Hemolytic Diseases

1.2.3 Coagulation Disorders

1.2.4 Metabolic Blood Diseases

1.3 Market by Treatment Mechanism

1.3.1 Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Treatment Mechanism: 2020 VS 2024 VS 2031

1.3.2 Gene Replacement Therapy

1.3.3 Gene Editing Therapy

1.3.4 Cell Therapy

1.3.5 Protein Replacement Therapy

1.4 Market by Product Technology

1.4.1 Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Product Technology: 2020 VS 2024 VS 2031

1.4.2 Adeno-associated virus (AAV) Vector Platform

1.4.3 Lentiviral Vector Platform

1.4.4 CRISPR/Cas

1.5 Market by Application

1.5.1 Global Inherited Orphan Blood Disorders Therapeutics Market Growth by Application: 2020 VS 2024 VS 2031

1.5.2 Hospital

1.5.3 Retail Pharmacy

1.5.4 Other

1.6 Assumptions and Limitations

1.7 Study Objectives

1.8 Years Considered

2 Global Growth Trends

2.1 Global Inherited Orphan Blood Disorders Therapeutics Market Perspective (2020-2031)

2.2 Global Inherited Orphan Blood Disorders Therapeutics Growth Trends by Region

2.2.1 Global Inherited Orphan Blood Disorders Therapeutics Market Size by Region: 2020 VS 2024 VS 2031

2.2.2 Inherited Orphan Blood Disorders Therapeutics Historic Market Size by Region (2020-2025)

2.2.3 Inherited Orphan Blood Disorders Therapeutics Forecasted Market Size by Region (2026-2031)

2.3 Inherited Orphan Blood Disorders Therapeutics Market Dynamics

2.3.1 Inherited Orphan Blood Disorders Therapeutics Industry Trends

2.3.2 Inherited Orphan Blood Disorders Therapeutics Market Drivers

2.3.3 Inherited Orphan Blood Disorders Therapeutics Market Challenges

2.3.4 Inherited Orphan Blood Disorders Therapeutics Market Restraints

3 Competition Landscape by Key Players

3.1 Global Top Inherited Orphan Blood Disorders Therapeutics Players by Revenue

3.1.1 Global Top Inherited Orphan Blood Disorders Therapeutics Players by Revenue (2020-2025)

3.1.2 Global Inherited Orphan Blood Disorders Therapeutics Revenue Market Share by Players (2020-2025)

3.2 Global Top Inherited Orphan Blood Disorders Therapeutics Players by Company Type and Market Share by Company Type (Tier 1, Tier 2, and Tier 3)

3.3 Global Key Players Ranking by Inherited Orphan Blood Disorders Therapeutics Revenue

3.4 Global Inherited Orphan Blood Disorders Therapeutics Market Concentration Ratio

3.4.1 Global Inherited Orphan Blood Disorders Therapeutics Market Concentration Ratio (CR5 and HHI)

3.4.2 Global Top 10 and Top 5 Companies by Inherited Orphan Blood Disorders Therapeutics Revenue in 2024

3.5 Global Key Players of Inherited Orphan Blood Disorders Therapeutics Head office and Area Served

3.6 Global Key Players of Inherited Orphan Blood Disorders Therapeutics, Product and Application

3.7 Global Key Players of Inherited Orphan Blood Disorders Therapeutics, Date of Enter into This Industry

3.8 Mergers & Acquisitions, Expansion Plans

4 Inherited Orphan Blood Disorders Therapeutics Breakdown Data by Type

4.1 Global Inherited Orphan Blood Disorders Therapeutics Historic Market Size by Type (2020-2025)

4.2 Global Inherited Orphan Blood Disorders Therapeutics Forecasted Market Size by Type (2026-2031)

5 Inherited Orphan Blood Disorders Therapeutics Breakdown Data by Application

5.1 Global Inherited Orphan Blood Disorders Therapeutics Historic Market Size by Application (2020-2025)

5.2 Global Inherited Orphan Blood Disorders Therapeutics Forecasted Market Size by Application (2026-2031)

6 North America

6.1 North America Inherited Orphan Blood Disorders Therapeutics Market Size (2020-2031)

6.2 North America Inherited Orphan Blood Disorders Therapeutics Market Growth Rate by Country: 2020 VS 2024 VS 2031

6.3 North America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025)

6.4 North America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031)

6.5 United States

6.6 Canada

7 Europe

7.1 Europe Inherited Orphan Blood Disorders Therapeutics Market Size (2020-2031)

7.2 Europe Inherited Orphan Blood Disorders Therapeutics Market Growth Rate by Country: 2020 VS 2024 VS 2031

7.3 Europe Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025)

7.4 Europe Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031)

7.5 Germany

7.6 France

7.7 U.K.

7.8 Italy

7.9 Russia

7.10 Ireland

8 Asia-Pacific

8.1 Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size (2020-2031)

8.2 Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Growth Rate by Region: 2020 VS 2024 VS 2031

8.3 Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size by Region (2020-2025)

8.4 Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size by Region (2026-2031)

8.5 China

8.6 Japan

8.7 South Korea

8.8 Southeast Asia

8.9 India

8.10 Australia & New Zealand

9 Latin America

9.1 Latin America Inherited Orphan Blood Disorders Therapeutics Market Size (2020-2031)

9.2 Latin America Inherited Orphan Blood Disorders Therapeutics Market Growth Rate by Country: 2020 VS 2024 VS 2031

9.3 Latin America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025)

9.4 Latin America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031)

9.5 Mexico

9.6 Brazil

10 Middle East & Africa

10.1 Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size (2020-2031)

10.2 Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Growth Rate by Country: 2020 VS 2024 VS 2031

10.3 Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025)

10.4 Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031)

10.5 Israel

10.6 Saudi Arabia

10.7 UAE

11 Key Players Profiles

11.1 Biogen

11.1.1 Biogen Company Details

11.1.2 Biogen Business Overview

11.1.3 Biogen Inherited Orphan Blood Disorders Therapeutics Introduction

11.1.4 Biogen Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.1.5 Biogen Recent Development

11.2 Novartis

11.2.1 Novartis Company Details

11.2.2 Novartis Business Overview

11.2.3 Novartis Inherited Orphan Blood Disorders Therapeutics Introduction

11.2.4 Novartis Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.2.5 Novartis Recent Development

11.3 Gilead Sciences

11.3.1 Gilead Sciences Company Details

11.3.2 Gilead Sciences Business Overview

11.3.3 Gilead Sciences Inherited Orphan Blood Disorders Therapeutics Introduction

11.3.4 Gilead Sciences Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.3.5 Gilead Sciences Recent Development

11.4 Sarepta Therapeutics

11.4.1 Sarepta Therapeutics Company Details

11.4.2 Sarepta Therapeutics Business Overview

11.4.3 Sarepta Therapeutics Inherited Orphan Blood Disorders Therapeutics Introduction

11.4.4 Sarepta Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.4.5 Sarepta Therapeutics Recent Development

11.5 Alnylam Pharmaceuticals

11.5.1 Alnylam Pharmaceuticals Company Details

11.5.2 Alnylam Pharmaceuticals Business Overview

11.5.3 Alnylam Pharmaceuticals Inherited Orphan Blood Disorders Therapeutics Introduction

11.5.4 Alnylam Pharmaceuticals Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.5.5 Alnylam Pharmaceuticals Recent Development

11.6 Amgen

11.6.1 Amgen Company Details

11.6.2 Amgen Business Overview

11.6.3 Amgen Inherited Orphan Blood Disorders Therapeutics Introduction

11.6.4 Amgen Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.6.5 Amgen Recent Development

11.7 Spark Therapeutics

11.7.1 Spark Therapeutics Company Details

11.7.2 Spark Therapeutics Business Overview

11.7.3 Spark Therapeutics Inherited Orphan Blood Disorders Therapeutics Introduction

11.7.4 Spark Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.7.5 Spark Therapeutics Recent Development

11.8 Akcea Therapeutics

11.8.1 Akcea Therapeutics Company Details

11.8.2 Akcea Therapeutics Business Overview

11.8.3 Akcea Therapeutics Inherited Orphan Blood Disorders Therapeutics Introduction

11.8.4 Akcea Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.8.5 Akcea Therapeutics Recent Development

11.9 Sunway Biotech

11.9.1 Sunway Biotech Company Details

11.9.2 Sunway Biotech Business Overview

11.9.3 Sunway Biotech Inherited Orphan Blood Disorders Therapeutics Introduction

11.9.4 Sunway Biotech Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.9.5 Sunway Biotech Recent Development

11.10 SIBIONO

11.10.1 SIBIONO Company Details

11.10.2 SIBIONO Business Overview

11.10.3 SIBIONO Inherited Orphan Blood Disorders Therapeutics Introduction

11.10.4 SIBIONO Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.10.5 SIBIONO Recent Development

11.11 AnGes

11.11.1 AnGes Company Details

11.11.2 AnGes Business Overview

11.11.3 AnGes Inherited Orphan Blood Disorders Therapeutics Introduction

11.11.4 AnGes Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.11.5 AnGes Recent Development

11.12 Orchard Therapeutics

11.12.1 Orchard Therapeutics Company Details

11.12.2 Orchard Therapeutics Business Overview

11.12.3 Orchard Therapeutics Inherited Orphan Blood Disorders Therapeutics Introduction

11.12.4 Orchard Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.12.5 Orchard Therapeutics Recent Development

11.13 Human Stem Cells Institute

11.13.1 Human Stem Cells Institute Company Details

11.13.2 Human Stem Cells Institute Business Overview

11.13.3 Human Stem Cells Institute Inherited Orphan Blood Disorders Therapeutics Introduction

11.13.4 Human Stem Cells Institute Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

11.13.5 Human Stem Cells Institute Recent Development

12 Analyst's Viewpoints/Conclusions

13 Appendix

13.1 Research Methodology

13.1.1 Methodology/Research Approach

13.1.1.1 Research Programs/Design

13.1.1.2 Market Size Estimation

13.1.1.3 Market Breakdown and Data Triangulation

13.1.2 Data Source

13.1.2.1 Secondary Sources

13.1.2.2 Primary Sources

13.2 Author Details

13.3 Disclaimer

FEATURED COMPANIES

Alnylam Pharmaceuticals

Human Stem Cells Institute

List of Tables

Table 1. Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Type (US$ Million): 2020 VS 2024 VS 2031

Table 2. Key Players of Hereditary Hemolytic Diseases

Table 3. Key Players of Coagulation Disorders

Table 4. Key Players of Metabolic Blood Diseases

Table 5. Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Treatment Mechanism (US$ Million): 2020 VS 2024 VS 2031

Table 6. Key Players of Gene Replacement Therapy

Table 7. Key Players of Gene Editing Therapy

Table 8. Key Players of Cell Therapy

Table 9. Key Players of Protein Replacement Therapy

Table 10. Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Product Technology (US$ Million): 2020 VS 2024 VS 2031

Table 11. Key Players of Adeno-associated virus (AAV) Vector Platform

Table 12. Key Players of Lentiviral Vector Platform

Table 13. Key Players of CRISPR/Cas

Table 14. Global Inherited Orphan Blood Disorders Therapeutics Market Size Growth by Application (US$ Million): 2020 VS 2024 VS 2031

Table 15. Global Inherited Orphan Blood Disorders Therapeutics Market Size by Region (US$ Million): 2020 VS 2024 VS 2031

Table 16. Global Inherited Orphan Blood Disorders Therapeutics Market Size by Region (2020-2025) & (US$ Million)

Table 17. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Region (2020-2025)

Table 18. Global Inherited Orphan Blood Disorders Therapeutics Forecasted Market Size by Region (2026-2031) & (US$ Million)

Table 19. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Region (2026-2031)

Table 20. Inherited Orphan Blood Disorders Therapeutics Market Trends

Table 21. Inherited Orphan Blood Disorders Therapeutics Market Drivers

Table 22. Inherited Orphan Blood Disorders Therapeutics Market Challenges

Table 23. Inherited Orphan Blood Disorders Therapeutics Market Restraints

Table 24. Global Inherited Orphan Blood Disorders Therapeutics Revenue by Players (2020-2025) & (US$ Million)

Table 25. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Players (2020-2025)

Table 26. Global Top Inherited Orphan Blood Disorders Therapeutics Players by Company Type (Tier 1, Tier 2, and Tier 3) & (based on the Revenue in Inherited Orphan Blood Disorders Therapeutics as of 2024)

Table 27. Ranking of Global Top Inherited Orphan Blood Disorders Therapeutics Companies by Revenue (US$ Million) in 2024

Table 28. Global 5 Largest Players Market Share by Inherited Orphan Blood Disorders Therapeutics Revenue (CR5 and HHI) & (2020-2025)

Table 29. Global Key Players of Inherited Orphan Blood Disorders Therapeutics, Headquarters and Area Served

Table 30. Global Key Players of Inherited Orphan Blood Disorders Therapeutics, Product and Application

Table 31. Global Key Players of Inherited Orphan Blood Disorders Therapeutics, Date of Enter into This Industry

Table 32. Mergers & Acquisitions, Expansion Plans

Table 33. Global Inherited Orphan Blood Disorders Therapeutics Market Size by Type (2020-2025) & (US$ Million)

Table 34. Global Inherited Orphan Blood Disorders Therapeutics Revenue Market Share by Type (2020-2025)

Table 35. Global Inherited Orphan Blood Disorders Therapeutics Forecasted Market Size by Type (2026-2031) & (US$ Million)

Table 36. Global Inherited Orphan Blood Disorders Therapeutics Revenue Market Share by Type (2026-2031)

Table 37. Global Inherited Orphan Blood Disorders Therapeutics Market Size by Application (2020-2025) & (US$ Million)

Table 38. Global Inherited Orphan Blood Disorders Therapeutics Revenue Market Share by Application (2020-2025)

Table 39. Global Inherited Orphan Blood Disorders Therapeutics Forecasted Market Size by Application (2026-2031) & (US$ Million)

Table 40. Global Inherited Orphan Blood Disorders Therapeutics Revenue Market Share by Application (2026-2031)

Table 41. North America Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031

Table 42. North America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025) & (US$ Million)

Table 43. North America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031) & (US$ Million)

Table 44. Europe Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031

Table 45. Europe Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025) & (US$ Million)

Table 46. Europe Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031) & (US$ Million)

Table 47. Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Region (US$ Million): 2020 VS 2024 VS 2031

Table 48. Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size by Region (2020-2025) & (US$ Million)

Table 49. Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size by Region (2026-2031) & (US$ Million)

Table 50. Latin America Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031

Table 51. Latin America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025) & (US$ Million)

Table 52. Latin America Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031) & (US$ Million)

Table 53. Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size Growth Rate by Country (US$ Million): 2020 VS 2024 VS 2031

Table 54. Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2020-2025) & (US$ Million)

Table 55. Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size by Country (2026-2031) & (US$ Million)

Table 56. Biogen Company Details

Table 57. Biogen Business Overview

Table 58. Biogen Inherited Orphan Blood Disorders Therapeutics Product

Table 59. Biogen Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 60. Biogen Recent Development

Table 61. Novartis Company Details

Table 62. Novartis Business Overview

Table 63. Novartis Inherited Orphan Blood Disorders Therapeutics Product

Table 64. Novartis Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 65. Novartis Recent Development

Table 66. Gilead Sciences Company Details

Table 67. Gilead Sciences Business Overview

Table 68. Gilead Sciences Inherited Orphan Blood Disorders Therapeutics Product

Table 69. Gilead Sciences Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 70. Gilead Sciences Recent Development

Table 71. Sarepta Therapeutics Company Details

Table 72. Sarepta Therapeutics Business Overview

Table 73. Sarepta Therapeutics Inherited Orphan Blood Disorders Therapeutics Product

Table 74. Sarepta Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 75. Sarepta Therapeutics Recent Development

Table 76. Alnylam Pharmaceuticals Company Details

Table 77. Alnylam Pharmaceuticals Business Overview

Table 78. Alnylam Pharmaceuticals Inherited Orphan Blood Disorders Therapeutics Product

Table 79. Alnylam Pharmaceuticals Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 80. Alnylam Pharmaceuticals Recent Development

Table 81. Amgen Company Details

Table 82. Amgen Business Overview

Table 83. Amgen Inherited Orphan Blood Disorders Therapeutics Product

Table 84. Amgen Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 85. Amgen Recent Development

Table 86. Spark Therapeutics Company Details

Table 87. Spark Therapeutics Business Overview

Table 88. Spark Therapeutics Inherited Orphan Blood Disorders Therapeutics Product

Table 89. Spark Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 90. Spark Therapeutics Recent Development

Table 91. Akcea Therapeutics Company Details

Table 92. Akcea Therapeutics Business Overview

Table 93. Akcea Therapeutics Inherited Orphan Blood Disorders Therapeutics Product

Table 94. Akcea Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 95. Akcea Therapeutics Recent Development

Table 96. Sunway Biotech Company Details

Table 97. Sunway Biotech Business Overview

Table 98. Sunway Biotech Inherited Orphan Blood Disorders Therapeutics Product

Table 99. Sunway Biotech Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 100. Sunway Biotech Recent Development

Table 101. SIBIONO Company Details

Table 102. SIBIONO Business Overview

Table 103. SIBIONO Inherited Orphan Blood Disorders Therapeutics Product

Table 104. SIBIONO Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 105. SIBIONO Recent Development

Table 106. AnGes Company Details

Table 107. AnGes Business Overview

Table 108. AnGes Inherited Orphan Blood Disorders Therapeutics Product

Table 109. AnGes Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 110. AnGes Recent Development

Table 111. Orchard Therapeutics Company Details

Table 112. Orchard Therapeutics Business Overview

Table 113. Orchard Therapeutics Inherited Orphan Blood Disorders Therapeutics Product

Table 114. Orchard Therapeutics Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 115. Orchard Therapeutics Recent Development

Table 116. Human Stem Cells Institute Company Details

Table 117. Human Stem Cells Institute Business Overview

Table 118. Human Stem Cells Institute Inherited Orphan Blood Disorders Therapeutics Product

Table 119. Human Stem Cells Institute Revenue in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025) & (US$ Million)

Table 120. Human Stem Cells Institute Recent Development

Table 121. Research Programs/Design for This Report

Table 122. Key Data Information from Secondary Sources

Table 123. Key Data Information from Primary Sources

Table 124. Authors List of This Report

List of Figures

Figure 1. Inherited Orphan Blood Disorders Therapeutics Picture

Figure 2. Global Inherited Orphan Blood Disorders Therapeutics Market Size Comparison by Type (2020-2031) & (US$ Million)

Figure 3. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Type: 2024 VS 2031

Figure 4. Hereditary Hemolytic Diseases Features

Figure 5. Coagulation Disorders Features

Figure 6. Metabolic Blood Diseases Features

Figure 7. Global Inherited Orphan Blood Disorders Therapeutics Market Size Comparison by Treatment Mechanism (2020-2031) & (US$ Million)

Figure 8. Gene Replacement Therapy Features

Figure 9. Gene Editing Therapy Features

Figure 10. Cell Therapy Features

Figure 11. Protein Replacement Therapy Features

Figure 12. Global Inherited Orphan Blood Disorders Therapeutics Market Size Comparison by Product Technology (2020-2031) & (US$ Million)

Figure 13. Adeno-associated virus (AAV) Vector Platform Features

Figure 14. Lentiviral Vector Platform Features

Figure 15. CRISPR/Cas Features

Figure 16. Global Inherited Orphan Blood Disorders Therapeutics Market Size by Application (2020-2031) & (US$ Million)

Figure 17. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Application: 2024 VS 2031

Figure 18. Hospital Case Studies

Figure 19. Retail Pharmacy Case Studies

Figure 20. Other Case Studies

Figure 21. Inherited Orphan Blood Disorders Therapeutics Report Years Considered

Figure 22. Global Inherited Orphan Blood Disorders Therapeutics Market Size (US$ Million), Year-over-Year: 2020-2031

Figure 23. Global Inherited Orphan Blood Disorders Therapeutics Market Size, (US$ Million), 2020 VS 2024 VS 2031

Figure 24. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Region: 2024 VS 2031

Figure 25. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Players in 2024

Figure 26. Global Inherited Orphan Blood Disorders Therapeutics Market Share by Company Type (Tier 1, Tier 2, and Tier 3)

Figure 27. The Top 10 and 5 Players Market Share by Inherited Orphan Blood Disorders Therapeutics Revenue in 2024

Figure 28. North America Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 29. North America Inherited Orphan Blood Disorders Therapeutics Market Share by Country (2020-2031)

Figure 30. United States Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 31. Canada Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 32. Europe Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 33. Europe Inherited Orphan Blood Disorders Therapeutics Market Share by Country (2020-2031)

Figure 34. Germany Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 35. France Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 36. U.K. Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 37. Italy Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 38. Russia Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 39. Ireland Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 40. Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 41. Asia-Pacific Inherited Orphan Blood Disorders Therapeutics Market Share by Region (2020-2031)

Figure 42. China Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 43. Japan Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 44. South Korea Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 45. Southeast Asia Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 46. India Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 47. Australia & New Zealand Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 48. Latin America Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 49. Latin America Inherited Orphan Blood Disorders Therapeutics Market Share by Country (2020-2031)

Figure 50. Mexico Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 51. Brazil Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 52. Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 53. Middle East & Africa Inherited Orphan Blood Disorders Therapeutics Market Share by Country (2020-2031)

Figure 54. Israel Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 55. Saudi Arabia Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 56. UAE Inherited Orphan Blood Disorders Therapeutics Market Size YoY Growth (2020-2031) & (US$ Million)

Figure 57. Biogen Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 58. Novartis Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 59. Gilead Sciences Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 60. Sarepta Therapeutics Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 61. Alnylam Pharmaceuticals Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 62. Amgen Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 63. Spark Therapeutics Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 64. Akcea Therapeutics Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 65. Sunway Biotech Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 66. SIBIONO Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 67. AnGes Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 68. Orchard Therapeutics Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 69. Human Stem Cells Institute Revenue Growth Rate in Inherited Orphan Blood Disorders Therapeutics Business (2020-2025)

Figure 70. Bottom-up and Top-down Approaches for This Report

Figure 71. Data Triangulation

Figure 72. Key Executives Interviewed

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