United States Gene Therapy Market 2025 | Growth Drivers, Trends & Market Forecast, Competitive Landscape & Investment Opportunities

Market Size and Growth

Gene Therapy Market size reached US$ 4.80 Billion in 2024 and is expected to reach US$ 35.91 Billion by 2033, growing at a CAGR of 23.1% during the forecast period 2025-2033.

Key Development:

United States: Recent Gene Therapy Developments

✅ October 6, 2025 - AstraZeneca entered a $555 million agreement with Algen Biotechnologies to jointly develop CRISPR-based gene-editing therapies targeting immune system diseases.

✅ September 25, 2025 - The U.S. Department of Health and Human Services launched the THRIVE program under ARPA-H, aiming to develop affordable precision genetic medicines for hereditary rare diseases.

✅ April 2025 - The FDA approved Zevaskyn (pz-cel), the first cell-based gene therapy for recessive dystrophic epidermolysis bullosa, offering new hope for patients with this rare genetic skin disorder.

Japan: Recent Gene Therapy Developments

✅ October 6, 2025 - The Taiwan Pavilion at BioJapan 2025 showcased collaborative innovations in AI, regenerative medicine, and CDMO, highlighting advancements in gene therapy within the Asia-Pacific region.

✅ October 1, 2025 - Takeda Pharmaceutical announced its decision to exit cell therapy research, seeking a partner to further develop its research and clinic-ready programs in this area.

✅ September 8, 2025 - The Japan Entry Acceleration Program (JEAP) launched an initiative to support regenerative medicine, cell, and gene therapy startups aiming to enter the Japanese market, offering mentorship and collaboration opportunities.

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Recent Mergers and Acquisitions:

United States: Recent Gene Therapy M&A Developments
✅ June 2025 - AbbVie acquired Capstan Therapeutics for $2.1 billion, enhancing its cell and gene therapy capabilities via Capstan's in vivo CAR-T platform and RNA delivery technology.

✅ April 2025 - Eli Lilly acquired Verve Therapeutics for $1.3 billion, gaining precision therapies for inherited cardiovascular conditions.

✅ March 2025 - AstraZeneca acquired EsoBiotec for up to $1 billion, focusing on in vivo CAR-T cell therapies delivered via IV injection.

✅ March 2025 - Bristol Myers Squibb acquired 2seventy bio for approximately $286 million, enhancing its CAR T-cell therapy portfolio.

✅ January 2025 - Medera announced plans to go public through a merger with Keen Vision Acquisition, supporting the development of its cardiac gene therapies.

Key Players:

=> Alnylam Pharmaceuticals, Inc., Spark Therapeutics, Inc., Novartis AG, bluebird bio, Inc., Ferring Pharmaceuticals Inc., Vertex Pharmaceuticals Incorporated, Sarepta Therapeutics, Inc., CSL Behring LLC, Amgen, Inc., Orchard Therapeutics group., Krystal Biotech, Inc., and among others.

Growth Forecast Projected:

The Global Gene Therapy 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 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.

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Key Segments:

➥ By Approach: In-Vivo, Ex-Vivo

➥ By Vector Type: Viral Vectors, Non-Viral Vectors

➥ By Technique: Gene Addition, Gene Silencing, Gene Editing

➥ By Application: Rare Diseases, Musculoskeletal Conditions, Blood Disorders, Oncology, Ophthalmology, Others

Regional Analysis for Gene Therapy 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 Gene Therapy 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 Gene Therapy 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

What is the current size of the Gene Therapy Market?

A: In 2024, the Gene Therapy Market was valued at US$ 4.80 Billion, reflecting its strong industry presence.

Q2: How large is the Gene Therapy Market expected to be by 2033?

A: By 2033, industry forecasts suggest the Gene Therapy Market will grow to around US$ 35.91 Billion , demonstrating significant expansion.

Q3: What is the growth rate of the Gene Therapy Market?

A: The market is projected to expand at a compound annual growth rate (CAGR) of 23.1% during the forecast period from 2025 to 2033.

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