U.S. Gene Therapy Market 2030: Specialty Clinics Driving Progress

The U.S. gene therapy market was valued at USD 3.11 billion in 2023 and is projected to reach USD 10.40 billion by 2030, growing at a compound annual growth rate (CAGR) of 19.21% from 2024 to 2030. Gene therapy encompasses DNA-based treatments that deliver a functional copy of a faulty gene or correct a mutated gene using viral vectors, plasmids, or gene editing tools. A robust development pipeline, led by universities and research institutions, is anticipated to significantly contribute to revenue generation over the coming years.

Despite some delays in clinical trials caused by the COVID-19 pandemic, the crisis underscored the potential of gene therapy for both prevention and treatment of diseases, leading to greater funding and investment in this space. While the pandemic temporarily slowed trial progression, it also amplified global interest and focus on the field.

Technological advancements have played a critical role in transitioning gene therapy from experimental stages to clinical applications. Innovations like CRISPR-Cas9 have transformed gene editing by enabling precise modifications with high accuracy and efficiency. In parallel, developments in viral vectors, particularly adeno-associated viruses (AAVs) and lentiviruses, have significantly improved the safety and efficacy of gene delivery to target cells.

Order a free sample PDF of the U.S. Gene Therapy Market Intelligence Study, published by Grand View Research.

Key Market Trends & Insights

• By Indication: The market includes conditions such as large B-cell lymphoma, multiple myeloma, spinal muscular atrophy (SMA), acute lymphoblastic leukemia (ALL), melanoma (lesions), inherited retinal diseases, beta-thalassemia major/sickle cell disease (SCD), and others.
• By Vector Type: The lentivirus segment led the market in 2023 with a revenue share of 43.74%, owing to its efficiency in delivering genetic material to host cells.
• By Route of Administration: Intravenous delivery dominated in 2023 and is expected to grow at the fastest pace over the forecast period, due to its widespread clinical applicability and effectiveness in systemic gene delivery.

Market Size & Forecast

• 2023 Market Size: USD 3.11 Billion
• 2030 Projected Market Size: USD 10.40 Billion
• CAGR (2024–2030): 19.21%

Key Companies & Market Dynamics

Leading players are pursuing product approvals, strategic collaborations, and geographic expansion to boost research, development, and product accessibility. Mergers and acquisitions are also being used to enhance capabilities, strengthen portfolios, and increase market share.

Notable Developments:

• December 2023: Tome Biosciences announced an investment of over USD 200 million to advance its programmable genomic integration platform, aimed at enabling targeted DNA sequencing. This development is expected to support long-term market growth.
• January 2023: Spark Therapeutics partnered with Neurochase to develop advanced delivery technologies for CNS-related gene therapies using Spark’s AAV platform and Neurochase’s expertise in direct drug delivery.
• September 2022: The U.S. FDA granted accelerated approval for Eli-cel (SKYSONA) to slow neurological decline in boys aged 4–17 with early, active cerebral adrenoleukodystrophy (CALD). Additionally, the FDA lifted a previous clinical hold on Eli-cel’s development program.

Key U.S. Gene Therapy Companies

• Amgen Inc.
• Novartis AG
• F. Hoffmann-La Roche
• Gilead Sciences, Inc.
• bluebird bio, Inc.
• Bristol-Myers Squibb Company
• Legend Biotech
• BioMarin
• uniQure N.V.
• Merck & Co.
• Sarepta Therapeutics, Inc.
• Krystal Biotech, Inc.
• CRISPR Therapeutics

Browse Horizon Databook on – U.S. gene therapy market Size & Outlook

Conclusion

The U.S. gene therapy market is entering a phase of rapid expansion, driven by scientific advancements, a strong clinical pipeline, and growing support from both private and public sectors. Despite initial setbacks during the pandemic, the industry has emerged more resilient, with heightened interest in its long-term potential. As new therapies gain approval and innovative delivery platforms are developed, gene therapy is poised to revolutionize the treatment landscape for a range of genetic and rare diseases. The market's projected growth through 2030 reflects not only its medical promise but also the strategic investments and collaborations shaping its future.