U.S. Gene Therapy Market Size, Share, Trends, Growth, & Analysis, Report | 2025 – 2034

The U.S. gene therapy market size is calculated at USD 4,370 million in 2025 and is projected to hit around USD 22,230 million by 2034, growing at a compound annual growth rate (CAGR) of 19.8% over the forecast period 2025 to 2034 , the rapid investment by leading companies, the increased prevalence of chronic diseases, and the integration of advanced technology have driven the market demand.

/EIN News/ -- Ottawa , Feb. 21, 2025 (GLOBE NEWSWIRE) -- The U.S. gene therapy market size was valued at USD 3,650 million in 2024 and is expected to surpass around USD 22,230 million by 2034, growing at a compound annual growth rate (CAGR) of 19.8% over the forecast period 2025 to 2034, a study published by Statifacts a sister firm of Precedence Statistics.

U.S. Gene Therapy Market Report Highlights

• Large B-cell lymphoma dominated the indication segment with the largest revenue share of 39.04% in 2024. This is attributed to the high prevalence of this type of cancer in the country. Additionally, gene therapy has shown promising results in treating this disease, leading to increased adoption by healthcare providers and patients.
• The intravenous route of administration held the larger market share in 2024 and is expected to grow at a higher CAGR during the forecast period. This route of administration involves the use of viruses, such as adenovirus, retrovirus, or lentivirus, to deliver the desired gene into the patient's cells.
• Based on vector type, the lentivirus segment dominated with the largest revenue share in 2024. On the other hand, AAV is anticipated to grow at the highest CAGR from 2025 to 2034, owing to the rising demand and their usage in clinical trials due to the higher accuracy level in delivering the gene to the region of interest.
  

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Gene therapy represents a groundbreaking approach to treating various illnesses by modifying, replacing, or supplementing defective or absent genes that contribute to disease onset. This innovative treatment has emerged as a focal point of research, particularly in the battle against degenerative diseases that afflict millions in the United States. The surge in cases of cancer and chronic illnesses has intensified the urgency for developing effective gene therapies. As a result, numerous advancements in these therapies are anticipated to gain regulatory approval in the near future, which is expected to significantly bolster the growth of the U.S. gene therapy market over the forecast period.

The mechanisms of gene therapy are primarily centered around the manipulation of genetic material. Strategies include inactivating problematic genes that contribute to disease or replacing them with healthy copies capable of restoring normal function. This therapeutic approach has shown promise across a diverse range of disorders, revealing the potential to cure illnesses such as diabetes, various forms of cancer, cardiovascular diseases, and even HIV/AIDS.

A number of factors facilitate the expansion of the U.S. gene therapy market. These include a robust portfolio of innovative products, increasing investments from leading industry stakeholders, and a high prevalence of diseases targeted by gene therapy. Additionally, there is growing interest in revolutionary therapeutic techniques. However, the path to widespread implementation is not without obstacles.

The high costs associated with product development, coupled with stringent regulatory frameworks, pose significant challenges that may impede growth in the market. In developed nations, advanced therapeutic solutions are increasingly replacing traditional treatment modalities such as chemotherapy that often come with severe side effects and long-term health repercussions. This shift highlights the importance of emerging gene therapies, particularly those utilizing viral vectors known for their low toxicity levels and high immunological compatibility.

Artificial intelligence (AI) plays a pivotal role in the evolution of gene therapy by analyzing extensive genomic and molecular datasets to pinpoint potential gene therapy targets and validate their therapeutic relevance. AI-driven tools facilitate the identification of novel gene candidates while assessing their stability and efficacy for therapeutic applications. Furthermore, AI can aid in crafting personalized treatment strategies, enabling healthcare providers to design tailored gene therapy interventions that are more likely to yield positive outcomes for individual patients.

In addition, AI technologies support the real-time monitoring of patient data, allowing for adaptive therapy approaches based on gene expression profiles, clinical parameters, and individual treatment responses. This improvement in monitoring can significantly enhance the overall efficacy of gene therapies. Moreover, the introduction of AI in the manufacturing processes of saline gene therapy can lead to substantial advancements. By automating workflows, optimizing production yields, and ensuring rigorous quality control, AI can help decrease production costs, thus making these advanced therapies more accessible to patients who need them.

The integration of AI into various stages of gene therapy development and production marks a significant leap toward effective, affordable, and personalized medical care, giving a significant boost to research and, in turn to demand in the U.S. gene therapy market.

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U.S. Gene Therapy Market Report Scope

Report Coverage	Details
Market Size in 2024	USD 3,650 Million
Market Size in 2025	USD 4,370 Million
Market Size by 2034	USD 22,230 Million
Growth Rate from 2025 to 2034	CAGR of 19.8%
Base Year	2024
Forecast Period	2025 to 2034
Segments Covered	By Type, By Vector, By Therapeutic Area, By Delivery Method, and By Route of Administration

U.S. Gene Therapy Market Trends

• Emergence in Different Fields:

While gene therapy initially concentrated on rare monogenic diseases, there is an increasing impetus to expand its application to more common conditions, including various forms of cancer, cardiovascular diseases, and neurological disorders such as Alzheimer's and Parkinson's. This shift is propelled by significant advancements in gene therapy technologies, coupled with a more profound understanding of the genetic underpinnings of complex diseases. Researchers are now focusing on the intricate interactions between multiple genes and environmental factors, thus broadening the scope of potential interventions.

• Advancements in Gene Editing:

CRISPR-Cas9 and other innovative gene editing technologies are transforming the landscape of gene therapy by allowing for precise modifications at the genomic level, whether through correction of deleterious mutations or targeted gene knockout. These cutting-edge tools are not only revolutionizing the treatment of hereditary genetic disorders, such as cystic fibrosis and sickle cell anemia but they are also being rigorously investigated in clinical trials for a wide range of applications, including viral infections and tumor suppressor genes in cancer therapy.

• Enhanced Vector Engineering:

In recent years, researchers have dedicated significant efforts to engineering viral vectors that feature heightened tissue specificity while minimizing off-target effects. This entails the meticulous modification of viral capsids to enhance their ability to target specific cell types, which is critical in reducing unintended immune responses. The development of these optimized vectors aims to lead to safer and more effective delivery mechanisms for gene therapies, ensuring that therapeutic genes are accurately delivered to the intended cells, thereby increasing the probability of treatment success.

• Regulatory Approvals and Market Access:

There has been a notable rise in the number of regulatory approvals granted for gene therapies, which is expanding patient access to these groundbreaking treatments. Regulatory agencies, recognizing the transformative potential of gene therapy, are actively working to streamline the approval processes. This includes providing clearer guidance for developers to navigate the regulatory landscape and fostering a more efficient pathway for bringing these innovative therapies to the U.S. gene therapy market, thus accelerating their availability to patients in need.

• Combination Therapies:

Researchers are also investigating the synergistic potential of combining gene therapies with other treatment modalities, such as immunotherapies and targeted pharmaceutical interventions. This integrative approach aims to enhance therapeutic efficacy and provide more comprehensive solutions for complex diseases. For instance, combining gene therapy with checkpoint inhibitors in cancer treatment could potentially activate the immune system to more effectively target and eliminate cancer cells, showcasing the promise of multidisciplinary strategies in modern medicine.

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Market Segmentation

Type Insights

The gene silencing segment has established itself as the predominant force in the U.S. gene therapy market in 2024, capturing the largest share primarily due to its unparalleled precision in targeting specific genes implicated in various diseases. This segment is also the fastest-growing and anticipated to grow with the highest CAGR in the market during the studied years. By employing a gene silencing mechanism, this approach effectively diminishes the expression of detrimental genes, making it particularly suitable for treating a wide array of conditions, including multiple cancer types, genetic disorders such as cystic fibrosis, and viral infections like HIV. Additionally, the utilization of gene silencing techniques is increasingly becoming integral to the development of innovative therapeutics aimed at combating cancer and other serious health issues.

Vector Insights

In the realm of gene therapy, the viral vector segment has emerged as a leader in the U.S. market in 2024, thanks to its exceptional ability to accurately target specific cell types. Viral vectors are characterized by their high transduction rates, which facilitate the effective transfer of therapeutic genes into targeted host cells. The rising demand for these specialized delivery systems has further strengthened this market segment. Furthermore, viral vectors are noted for their efficiency in delivering genetic materials, which enhances their applicability in diverse gene therapy contexts.

Conversely, the non-viral vector segment is anticipated to become increasingly opportunistic during the forthcoming forecast period 2025 to 2034. This expectation is attributed to the broad accessibility of non-viral vector gene therapies, which are often favored over their viral counterparts due to their simpler production processes and superior safety profiles. Moreover, non-viral vectors significantly mitigate the risks associated with insertional mutagenesis, thereby presenting a safer alternative for patients receiving gene therapy.

Therapeutic Area Insights

The oncology segment has firmly established itself as the leader in the U.S. gene therapy market in 2024, driven largely by the escalating incidence of cancer in the country. Gene therapies have gained considerable momentum in cancer treatment, owing to their capacity to specifically target cancerous cells while minimizing the adverse effects that are commonly associated with traditional chemotherapy. This targeted approach not only enhances treatment efficacy but also contributes to improved patient quality of life. The recent surge in the approval of gene therapies designed to treat cancer, alongside the rising incidence of hematologic tumors, has further propelled growth in this segment.

The neurology segment is poised for rapid expansion in the coming years, fueled by the increasing prevalence of neurological disorders such as Alzheimer’s disease and Parkinson’s disease. Significant advancements in targeted gene therapy methodologies have opened new avenues for treating these complex conditions through various techniques, which range from exogenous gene expression to the suppression of endogenous genes.

Delivery Method Insights

The in vivo segment has asserted its dominance within the U.S. gene therapy market in 2024. This growth can be attributed to the in vivo method's efficacy in delivering genes directly into patients’ bodies, thereby enhancing the overall effectiveness of therapeutic interventions. The in vivo approach is inherently less invasive than ex vivo methods, which often require complex surgical procedures to manipulate cells outside the body. As the demand for targeted therapies continues to rise, in vivo gene therapy has gained significant popularity, thereby driving the expansion of this segment within the market.

The ex vivo segment of the U.S. gene therapy market is projected to expand rapidly in the market in the coming years due to a number of factors. Ex vivo gene therapy deals with the modification of patient cells outside the host body and then reintroducing them after specific genetic changes. These type of therapeutics offer long-lasting effects. Ex vivo treatment is particularly used for treating genetic disorders where a one-time intervention can significantly improve patient quality of life. Advancements in gene editing technologies such as CRISPR/Cas9 have caused a surge in ex vivo therapies. 

Route of Administration Insights

Leading the charge in the U.S. gene therapy market, the intravenous (IV) segment has garnered attention due to its unique advantages over other routes of administration. Intravenous delivery allows for quicker absorption and immediate therapeutic effects since the therapeutic agents go directly into the bloodstream, bypassing the digestive system. This method is particularly advantageous as it facilitates the simultaneous treatment of multiple targeted organs, offering a comprehensive approach to patient care.

The rapid delivery of gene therapy to targeted tissues, combined with its remarkable effectiveness, has been crucial in bolstering this segment. Innovations in vector design and advanced delivery mechanisms have led to the evolution of minimally invasive intravenous therapies, further contributing to the growth and appeal of this route of administration in the gene therapy landscape.

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U.S. Gene Therapy Market Top Key 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.

Recent Developments

• In February 2025, gene therapy company AskBio Inc., a subsidiary of Bayer AG, announced that investigational genel therapy AB-1005 for the management and treatment of Parkinson’s disease, has recieved the RMAT designation from the United States Food and Drug Administration (FDA). RMAT  or Regenerative Medicine Advanced Therapy is a special designation granted by the FDA for medical treatments such as gene therapies, for the treatment, modificiation, reversal or cure of debilitating, and fatal conditions and diseases. 

• In April 2024, the President of India Droupadi Murmu launched the country’s first home-grown anti-cancer CAR-T cell therapy at the Indian Institute of Technology, (IIT) Bombay in Mumbai. She also expressed that gene therapy will be a valuable asset for many patients

U.S. Gene Therapy Market Report Segmentation

This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2019 to 2034. For this study, Statifacts has segmented the global U.S. Gene Therapy Market

U.S. Gene Therapy Market Segmentation:

By Type

• Gene silencing
• Cell replacement
• Gene augmentation
• Other therapies

By Vector

• Viral vectors
• Non-Viral vectors

By Therapeutic Area

• Neurology
• Oncology
• Hepatology
• Other therapeutic areas

By Delivery Method

• In Vivo
• Ex Vivo

By Route of Administration

• Intravenous
• Other routes of administration

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