Sickle Cell Disease (SCD) and β-Thalassemia Gene Therapy Market to hit US$ 380.42 Million by 2033 | Market Size & Future Opportunities

global sickle cell disease (SCD) and β-thalassemia gene therapy market size was US$ 83.87 Million in 2024 and is expected to reach US$ 380.42 Million by 2033, growing at a CAGR of 18.4% during the forecast period 2025-2033.

The U.S. SCD/β-Thal gene therapy market is estimated at USD 0.68 billion in 2024 and is anticipated to expand to USD 2.5 billion by 2032. The growth is propelled by strong healthcare infrastructure, regulatory support, and increased clinical trial activities in the country, with a CAGR of 18.2%.

Japan's SCD/β-Thal gene therapy market is valued at USD 0.13 billion in 2024 and expected to grow to USD 0.53 billion by 2032. Growth factors include government initiatives, growing awareness, and advancements in gene editing technologies, with a CAGR of 16.3%.

In the U.S., ZYNTEGLO is priced at a wholesale acquisition cost of $2.8 million, whereas LYFGENIA is priced at $3.1 million per therapy.

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Key Highlights From This Report

➠ North America leads the global sickle cell disease (SCD) and β-thalassemia gene therapy market, holding the largest revenue share of 48.5% in 2024.

➠ The Asia Pacific region is the fastest-growing market in this sector, with a CAGR of 7.7% in 2024.

➠ The ZYNTEGLO drug segment dominates the market, accounting for 60.3% of the revenue share in 2024.

➠ The intravenous administration segment holds complete dominance, capturing 100% of the market share in 2024.

Key Players:

CRISPR Therapeutics and bluebird bio, Inc., and emerging players include Sangamo Therapeutics, Inc., Editas Medicine, Beam Therapeutics, Editas Medicine, and CorrectSequence Therapeutics Co., Ltd.

Key Development:

1. In February 2025, SK pharmteco Cell & Gene Europe, AP-HP, AFM-Téléthon, and Institut Imagine partnered to produce a 200L CGMP lentiviral vector batch and support a novel sickle cell disease clinical trial, advancing gene therapy development.

2. In August 2025, CorrectSequence Therapeutics treated the first sickle cell disease patient with its base-editing therapy CS-101, achieving a clinical cure in an investigator-initiated trial.

3. In September 2025, Vertex Pharmaceuticals secured a reimbursement deal with Italy's AIFA, granting access to its CRISPR/Cas9 gene therapy CASGEVY for eligible β-thalassemia and severe sickle cell disease patients.

4. In October 2024, Editas Medicine reported progress in 2024 goals with successful in vivo preclinical proof of concept for HSPC editing and fetal hemoglobin induction using a targeted lipid nanoparticle delivery system.

5. In January 2024, Saudi Arabia's SFDA approved Vertex's CASGEVY gene therapy for treating sickle cell disease and transfusion-dependent β-thalassemia in patients aged 12 and above.

Growth Forecast Projected:

The Global SCD/β-Thal Gene Therapy Market is anticipated to rise at a considerable rate during the forecast period, between 2025 and 2032. In 2024, 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 SCD/β-Thal Gene Therapy 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.

Latest M&A Activity

Bluebird Bio remains a key player with ongoing collaborations and partnerships to scale production of their gene therapies ZYNTEGLO and exagamglogene autotemcel (exa-cel), targeting transfusion-dependent β-thalassemia and severe SCD.

Pharmaceutical CDMOs and biotech firms specializing in lentiviral vector manufacturing and gene editing technologies continue consolidation trends to meet increasing therapy demand and regulatory compliance.

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Key Segments:

By Drug: ZYNTEGLO, CASGEVY, LYFGENIA

By Route of Administration: Intravenous, Hospital Pharmacies, Retail Pharmacies

Regional Analysis for 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)

Recent Product Launches and Regulatory Approvals

1. FDA approved exagamglogene autotemcel (exa-cel) in December 2023 for transfusion-dependent β-thalassemia and January 2024 for severe SCD, marking one of the first autologous CRISPR gene-editing therapies available.​

2. In 2025, Vertex Pharmaceuticals achieved a reimbursement agreement in Italy for CASGEVY, their CRISPR/Cas9-based gene-editing therapy for β-thalassemia and SCD, enhancing patient accessibility.​

3. India launched BIRSA 101, the first indigenous CRISPR-based therapy for SCD, marking a major advancement in region-specific gene therapy development and deployment.

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Clinical Trials and Research Highlights

1. Phase 3 trials (CLIMB-SCD-121 and CLIMB-THAL-111) demonstrate durable efficacy with over 90% reduced transfusions and vaso-occlusive crises free periods extending beyond 12 months in many patients treated with exa-cel.​

2. Ongoing trials are evaluating long-term safety, immunogenicity, and quality-of-life improvements with gene therapies for pediatric and adult populations.​

3. Innovative approaches include multi-gene editing, optimized delivery vectors, and off-the-shelf allogenic therapies under early-stage development.

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