Hemoglobinopathies Market Size & Outlook, 2026-2034

Hemoglobinopathies Market Overview

The global hemoglobinopathies market size is valued at USD 9.67 billion in 2025 and is anticipated to grow from USD 10.48 billion in 2026 to USD 20.50 billion by 2034, growing at a CAGR of 8.75% from 2026 to 2034. The global hemoglobinopathies market growth is attributed to the rising prevalence of sickle cell disease and β-thalassemia, which are increasing the demand for effective treatments. Additionally, advances in cell-based therapies, expanding newborn screening programs, and supportive government initiatives in high-burden regions also drive market growth.

Key Market Insights

• North held a dominant share of the global market, accounting for a 38.77% share in 2025, owing to high adoption of advanced therapies and higher diagnosis and awareness rates.
• The Asia Pacific region is growing at the fastest pace, with a CAGR of 13%, due to high disease prevalence and carrier rates and rising newborn screening programs.
• Based on type, the sickle cell disease segment held the highest market share of 37%. This growth is attributed to the increasing global incidence of sickle cell disease and the growth in newborn screening programs.
• On the basis of treatment type, the stem cell transplant segment is expected to register the fastest CAGR growth of 76%. This growth is attributed to improved donor matching technologies and the growing demand for stem cell transplants in India and other countries.
• Based on end-user, the hospital segment dominated the market in 2025.
• U.S. dominates the hemoglobinopathies market, valued at USD 3.21 billion in 2024 and reaching USD 3.47 billion in 2025.

Market Size & Forecast

• 2025 Market Size: USD 9.67 billion
• 2034 Projected Market Size: USD 20.50 billion
• CAGR (2026-2034): 8.75%
• Dominating Region: North America
• Fastest-Growing Region: Asia Pacific

Source: Straits Research Analysis

Market Trends

Emergence of gene and cell-based therapies for hemoglobinopathies

The global market is shifting towards single administration gene therapies that aim to cure transfusion-dependent beta thalassemia, expanding the market growth.

• For instance, in December 2023, the U.S. FDA approved CASGEVY, an exagamglogene autotemcel cell therapy as the first approved cell therapy for sickle cell disease treatment.

This trend represents a shift from chronic supportive care to one-time durable cell therapy treatment.

Expansion of newborn screening programs for hemoglobinopathies

The growing implementation of newborn screening initiatives is a key trend driving earlier diagnosis and treatment adoption for hemoglobinopathies.

• For instance, in 2023, India launched the National Sickle Cell Anaemia Elimination Mission, which aims to secure about 70 million people in tribal and high-prevalence regions by 2025-2026, significantly increasing the diagnosis rate.

This expansion is a major trend shifting from underdiagnosis to early disease screening, which, in turn, expands access to therapies.

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Market Drivers

Rising Prevalence of Sickle Cell Disease and Thalassemia

The increasing global prevalence of hemoglobinopathies such as sickle cell disease and beta thalassemia is a significant driver propelling the demand for effective treatments.

• For instance, according to the Journal of Hematology and Allied Sciences 2023, about 7.74 million people were living with sickle cell disease in 2021, and about 515,000 newborns were affected annually.

This surge in hemoglobinopathies is driving the need for innovative diagnostics and therapies, thereby contributing to market growth.

Advancements in Stem Cell Transplantation Techniques

Improvements in hematopoietic stem cell transplantation are enhancing the safety and accessibility for patients with hemoglobinopathies.

• For instance, in 2023, a clinical study reported that haploidentical bone marrow transplants with post-transplant cyclophosphamide achieved more than 90% overall survival in children with sickle cell disease.

Thus, advancements in transplant techniques are expanding treatment access, which, in turn, drives the growth of hemoglobinopathies market.

Market Restraint

High Cost of Cell and Gene Therapies

The high cost associated with gene and cell therapies for hemoglobinopathies is limiting widespread adoption in low and middle-income countries.

• For instance, the U.S. FDA-approved CASGEVY for sickle cell disease is priced at USD 2.2 million per patient in the U.S., making it one of the most expensive therapies across the globe.

These high treatment cost restricts the patient accessibility and adoption, thereby restraining overall market growth.

Market Opportunity

Development of an Outcome-Based Reimbursement Model

The growing adoption of value-based payment frameworks for high-cost therapies is creating a significant opportunity for the hemoglobinopathies market.

• For instance, in July 2025, the Centers for Medicare and Medicaid Services announced that 33 states, along with the District of Columbia and Puerto Rico, will be participating in the CGT Access Model with manufacturers for sickle cell disease gene therapies.

This shift toward innovative reimbursement models is improving patient access to advanced therapies, thereby creating a major opportunity for market growth.

Regional Analysis

North America dominated the Hemoglobinopathies market in 2025, accounting for 38.77% market share. This dominance is attributed to strong adoption of gene editing and stem cell transplantation, rapid approvals by the U.S. FDA for novel therapies such as CASGEVY, and favorable reimbursement policies, which support the penetration of therapies to treat hemoglobinopathies. Moreover, significant R&D investment by leading pharmaceutical players and academic institutes also supports the regional market growth.

A significant factor driving the U.S. market growth is the federal investment in newborn screening programs. In fiscal year 2025, the Health Resources & Services Administration (HRSA) allocated USD 65,500 in supplemental funds to each of the 25 grantees of the sickle cell disease (SCD) newborn screening follow-up program. This initiative enhances early detection and access to care for infants diagnosed with SCD, thereby improving patient outcomes and contributing to the overall market size.

A key factor driving the Canada hemoglobinopathies market growth is the expansion of provincial gene therapy access programs. In 2024, Ontario Health announced funding pathways for patients with transfusion-dependent β-thalassemia to receive emerging gene editing therapies through specialized treatment centers. Under these initiatives, patients can have early access to treatments. Thus, enhanced access to gene therapy is directly boosting the Canada hemoglobinopathies market size and overall market growth.

Asia Pacific Market Trends

Asis Pacific is emerging as the fastest-growing region with a CAGR of 10.13% from 2026-2034, owing to the high prevalence of sickle cell disease and β-thalassemia in countries such as India, Thailand, and Southeast Asia. Additionally, the expansion of government-supported screening programs and awareness initiatives is increasing early diagnosis, while improvements in healthcare infrastructure, such as specialized hospitals and transplant centers, are enhancing treatment accessibility.

Source: Straits Analysis

A significant factor driving the China hemoglobinopathies market growth is the government’s investment in gene therapy research. In 2024, the China National Center for Biotechnology Development (CNCBD) announced funding for gen-editing projects targeting sickle cell disease and thalassemia. This initiative supports the development of advanced treatments within China, aiming to reduce reliance on imported therapies. By fostering domestic innovation, these investments contribute to the overall market growth of the Chinese hemoglobinopathies market.

A key factor driving the India hemoglobinopathies market growth is the National Sickle Cell Anaemia Elimination Mission (NSCAEM), launched in July 2023. This program targets tribal and high-risk regions to eliminate sickle cell disease by 2047. By June 2024, over 3.37 crore people were screened, identifying around 1.4 lakh patients and 9.75 lakh carriers. Early detection and timely care improve health outcomes, which boosts the India hemoglobinopathies market size and overall market growth.

Europe Market Trends

A key factor driving the Germany hemoglobinopathies market growth is the introduction of special programs that help teenagers with sickle cell disease (SCD) move smoothly from pediatric to adult care. In 2022, doctors from both children’s and adult hematology teams created a national plan to guide this transition. By ensuring continuous care and better support for young patients, these programs improve health outcomes and increase access to treatments, which in turn boosts the Germany hemoglobinopathies market size.

Type Insights

The sickle cell disease segment dominated the market and accounted for a 58.37% revenue share in 2025. This growth is attributed to the high prevalence of sickle cell disease and expanding newborn screening programs, enabling earlier diagnosis with the approval for innovative therapies such as CASGEVY and LYFGENIA.

Source: Straits Analysis

Treatment Type Insights

The stem cell transplant is expected to register the fastest CAGR growth of 9.76%, owing to reducing the need for chronic transfusion and chelation therapy. Additionally, advancements in transplantation techniques, supportive government initiatives, and the establishment of specialized transplant centers in high-burden areas are enhancing patient access to these procedures, which, in turn, drives the segment growth.

End User Insights

The hospitals segment dominated the market in 2025. This growth is attributed to the availability of advanced diagnostic and treatment facilities, such as transfusion services, stem cell transplantation, and administration of novel gene and cell therapies. Additionally, the designation of specialized hospitals as authorized treatment centers for newly approved gene therapies facilitates patient access, thereby driving the segment growth.

Competitive Landscape

The global hemoglobinopathies market is fragmented, with leading pharmaceutical and biotechnology companies capturing a significant market share. Established companies maintain leadership through innovative therapies, extensive clinical trial networks, and strong R&D pipelines focused on gene and cell-based treatments, as well as supportive care therapies.

The major players in the market include Novartis AG, Vertex Pharmaceuticals, CRISPR Therapeutics, Bluebird Bio, Pfizer Inc., and Bristol Myers Squibb. The industry players are inclined towards adopting various strategies, such as product launch and product approvals, to remain competitive in the market.

Tessera Therapeutics Inc.: An Emerging Market Player

Tessera Therapeutics, a biotechnology company, is emerging in the hemoglobinopathies market with a focus on in vivo gene therapies for sickle cell disease. The company’s lead candidate, TESS-101, uses its proprietary Gene Writing platform to directly edit the genome that causes sickle cell disease.

• In December 2024, Tessera received a USD 50 million investment from the Bill & Melinda Gates Foundation to advance the development of TESS-101, a gene therapy to treat sickle cell disease.
• In 2025, the company initiated Phase 3 trials in the U.S. and Europe, reflecting regulatory confidence and potential market entry.

Thus, Tessera Therapeutics is emerging as a significant player in the hemoglobinopathies market.

List of key players in Hemoglobinopathies Market

1. Sangamo Therapeutics, Inc.
2. Global Blood Therapeutics, Inc.
3. bluebird bio, Inc.
4. Emmaus Life Sciences Inc.
5. Pfizer, Inc.
6. Novartis AG
7. Prolong Pharmaceuticals, LLC
8. Bioverativ Inc.
9. Gamida Cell
10. Celgene Corporation
11. Alnylam Pharmaceuticals, Inc.
12. Merck & Co. Inc.
13. Biogen Inc.
14. CRISPR Therapeutics
15. Others

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Recent Development

• 3^rd June 2025: The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor for sickle cell disease.