United States Thalassemia Treatment Market 2025 | Growth Drivers, Key Players & Investment Opportunities

Market Size and Growth

The Thalassemia Treatment Market is expected to grow at a CAGR of 10.5% during the forecast period 2024-2031

Key Development:

United States: Recent Industry Developments

✅ In September 2025, Dana-Farber Cancer Institute successfully treated its first patient with an FDA-approved gene therapy for beta thalassemia, marking a significant milestone in curative treatments for blood disorders.

✅ In June 2025, the National Institutes of Health (NIH) launched a multi-center trial evaluating novel gene-editing therapies for beta thalassemia, aiming to advance precision medicine in rare blood disorders.

✅ In May 2025, Agios Pharmaceuticals announced early clinical data supporting a new pyruvate kinase activator in beta thalassemia management, potentially reducing transfusion dependency.

Japan: Recent Industry Developments

✅ In September 2025, Asahi Kasei Life Science began operations focused on providing various products and services to the rapidly growing pharmaceutical industry, aiming to meet discerning market needs.

✅ In March 2025, Kissei Pharmaceutical Company commercialized seven new products in Japan, including treatments for rare and intractable diseases, expanding its presence in the pharmaceutical market.

✅ In January 2025, Agios Pharmaceuticals announced that the U.S. FDA had accepted its supplemental new drug application (sNDA) seeking label expansion for Pyrukynd to include the treatment of thalassemia, potentially offering a new therapeutic option.

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Thalassemia Treatment involves managing a genetic blood disorder characterized by reduced hemoglobin production. Treatment approaches include regular blood transfusions, iron chelation therapy to prevent iron overload, bone marrow or stem cell transplantation, and supportive care to improve quality of life and prevent complications.

Recent Mergers and acquisitions:

United States: Recent M&A Developments

✅ In June 2025, Eli Lilly announced the acquisition of gene-editing biotech Verve Therapeutics for up to $1.3 billion. The deal includes an upfront payment of $1 billion and up to $300 million in milestone-based payouts. Verve is developing a CRISPR-based gene therapy targeting the PCSK9 gene, which is associated with high cholesterol and cardiovascular disease.

✅ In February 2025, Bluebird Bio was acquired by private equity firms Carlyle Group and SK Capital Partners for approximately $29 million. The acquisition aims to provide necessary capital for scaling commercialization efforts of Bluebird's gene therapies for various diseases, including beta-thalassemia.

✅ In August 2022, Pfizer acquired Global Blood Therapeutics for $5.4 billion. This acquisition allows Pfizer to strengthen its ability to tackle sickle cell disease by incorporating innovative treatments and expertise, ultimately enhancing health equity and increasing access to life-changing therapies for underserved patient communities.

Key Players:

=> Vifor Pharma, Kiadis Pharma, Gamida Cell, ApoPharma, bluebird bio, Inc., Novartis AG, Bristol-Myers Squibb Company, Celgene Corporation, La Jolla Pharmaceutical, Sangamo Biosciences, and Acceleron Pharma. In the thalassemia treatment market, bluebird bio-Inc.

Growth Forecast Projected:

The Global Thalassemia Treatment Market is anticipated to rise at a considerable rate during the forecast period, between 2024 and 2031. In 2023, the market is growing at a steady rate, and with the rising adoption of strategies by key players, the market is expected to rise over the projected horizon.

Research Process:

Both primary and secondary data sources have been used in the global Thalassemia Treatment Market research report. During the research process, a wide range of industry-affecting factors are examined, including governmental regulations, market conditions, competitive levels, historical data, market situation, technological advancements, upcoming developments, in related businesses, as well as market volatility, prospects, potential barriers, and challenges.

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Key Segments:

➥ By Disease Type: Alpha Thalassemia, Beta Thalassemia
➥ By Treatment Type: Blood Transfusion, Iron Chelation Therapy, Bone Marrow Transplant, Gene Therapy, Surgery

Regional Analysis for Thalassemia Treatment Market:

⇥ North America (U.S., Canada, Mexico)

⇥ Europe (U.K., Italy, Germany, Russia, France, Spain, The Netherlands and Rest of Europe)

⇥ Asia-Pacific (India, Japan, China, South Korea, Australia, Indonesia Rest of Asia Pacific)

⇥ South America (Colombia, Brazil, Argentina, Rest of South America)

⇥ Middle East & Africa (Saudi Arabia, U.A.E., South Africa, Rest of Middle East & Africa)

Benefits of the Report:

Chapter 1: Sets the stage by outlining the report's coverage, summarizing key market segments by region, product type, and application. Presents a snapshot of market sizes, growth potential across segments, and anticipated industry evolution both short and long term.

Chapter 2: Highlights pivotal market insights and uncovers the most significant emerging trends driving change within the industry.

Chapter 3: Offers an in-depth look at the competitive landscape among Thalassemia Treatment producers, including revenue shares, strategic moves, and recent mergers and acquisitions.

Chapter 4: Presents comprehensive profiles of the market's key players, delving into details such as revenue, profit margins, product portfolios, and company milestones.

Chapters 5 & 6: Analyze Thalassemia Treatment revenue at both regional and country levels, providing quantitative breakdowns of market sizes, growth opportunities, and development prospects worldwide.

Chapter 7: Focuses on different market segments by type, examining their individual sizes and potential, guiding readers toward high-impact, untapped market areas.

Chapter 8: Explores segmentation by application, evaluating industry growth potential in various downstream markets and pinpointing promising sectors for expansion.

Chapter 9: Provides a thorough review of the industry's supply chain mapping out both upstream and downstream activities.

Chapter 10: Concludes with a summary of the report's key findings and highlights the most critical takeaways for industry stakeholders.

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FAQ

Q: What is the growth rate of the Thalassemia Treatment Market?

A: The market is projected to expand at a compound annual growth rate (CAGR) of 10.5% during the forecast period from 2024 to 2031.

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