As per MRFR analysis, the US Gene Therapy Market Size was estimated at 1.81 (USD Billion) in 2023. The US Gene Therapy Market Industry is expected to grow from 2.2 (USD Billion) in 2024 to 8.5 (USD Billion) by 2035. The US Gene Therapy Market CAGR (growth rate) is expected to be around 13.074% during the forecast period (2025 - 2035).
The US gene therapy market is witnessing several key trends that are shaping its future. One significant trend is the increased regulatory support from government agencies like the FDA, which has streamlined the approval processes for gene therapies. This regulatory flexibility encourages innovation and promotes the development of new therapies to treat rare and complex diseases.
Additionally, a growing number of collaborations between biotech companies and academic institutions in the US is fueling research and development efforts. This synergy helps in navigating the complex landscape of gene therapy while leveraging expertise and resources. Key market drivers for the US gene therapy market include the rising prevalence of genetic disorders and the demand for personalized medicine.
As the population ages, the incidence of genetic diseases is expected to rise, which drives the need for effective treatments. The increasing public awareness of gene therapy and its potential benefits also propels market growth, alongside advancements in technologies like CRISPR and viral vector design that make gene editing more accessible and effective.
There are numerous opportunities to be explored within the US gene therapy market. Investment in infrastructure for manufacturing and distribution is essential to meet the growing demand for these therapies. Additionally, there is scope for expanding gene therapies into new therapeutic areas such as oncology, cardiology, and neurology, where existing treatment options may be limited.
Trends in recent times reflect an increasing focus on developing all-in-one gene therapies that not only address the genetic cause of diseases but also provide a sustainable solution. The growing trend towards patient-centric approaches ensures that therapies are designed with input from patients, leading to improved outcomes and satisfaction.
Thus, the US gene therapy market is on a path of dynamic growth, influenced by regulatory support, technological advancements, and increasing opportunities for expansion across diverse therapeutic areas.
Source: Primary Research, Secondary Research, MRFR Database and Analyst Review
The rising prevalence of genetic disorders in the United States is a significant market driver for the US Gene Therapy Market Industry. Diseases such as cystic fibrosis, hemophilia, and muscular dystrophy collectively impact millions of Americans and are driving the demand for innovative therapies. According to the Centers for Disease Control and Prevention, approximately 1 in every 33 children in the US is born with a genetic disorder, which translates to about 120,000 births each year.
Organizations like the National Institutes of Health are heavily investing in Research and Development for gene therapies aimed at these disorders. With advancements in technology and the increasing grants for therapeutic solutions, the market is anticipated to expand, as it directly addresses the urgent medical needs of this population.
Technological advancements in gene editing, particularly the development of CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats), play a crucial role in shaping the US Gene Therapy Market Industry. Research from the National Academy of Sciences indicates that CRISPR technology can target and edit specific genes, proving successful in eliminating genetic diseases in preclinical studies.
The capability for precise modifications means that more therapies can enter the development stage, fueled by the intellectual property filings that have surged over the last few years, especially in areas like oncology. Prominent companies such as Editas Medicine are at the forefront, influencing the market trajectory and offering new possibilities for customized therapies.
A supportive regulatory framework in the United States is essential for the growth of the US Gene Therapy Market Industry. The Food and Drug Administration has streamlined the approval processes for gene therapies, reflecting its commitment to expediting innovative treatments. Since the introduction of the Breakthrough Therapy Designation, over 20 gene therapies have received expedited review.
This regulatory environment not only fosters innovation but also instills confidence in investors, propelling funding for Research and Development initiatives. The resulting commercialization of these therapies creates a unique opportunity, sectioning the US as a leader in gene therapy advancements.
An increase in investments within the biopharmaceutical sector significantly drives the US Gene Therapy Market Industry. Venture capital funding in biotech firms focusing on gene therapies reached approximately USD 22 billion in 2020, according to the Biotechnology Innovation Organization. This financial influx supports the extensive Research and Development required for therapeutic innovations while spurring collaborations between established pharmaceutical companies and emerging biotech firms.
With the rising number of partnerships, such as those seen with companies like Spark Therapeutics, the market is poised for accelerated growth, enabling the development of more effective and targeted gene therapies.
The US Gene Therapy Market, particularly focusing on the Therapeutic Area segment, showcases a promising landscape driving innovation and growth. The intended applications of gene therapies cover various critical health areas, which include oncology, genetic disorders, infectious diseases, cardiovascular diseases, and neurological disorders.
Among these, oncology has emerged as a forefront category, primarily due to the rising incidence of cancer, which leaves a significant impact on public health. Research demonstrates that gene therapies offer potential cures and targeted treatments for various cancers, thus paving a pathway for advanced and personalized treatment options that continue to attract substantial Research and Development funding.
Genetic disorders represent another vital block within the Therapeutic Area of the US Gene Therapy Market. These conditions often have a hereditary base, affecting a notable portion of the population. Gene therapies target the underlying genetic causes of these disorders, promising a transformative approach across many hereditary diseases such as cystic fibrosis and hemophilia, which were once deemed difficult to manage effectively.
The increasing recognition of the benefits of gene editing technologies has led healthcare professionals towards considering them as viable solutions for millions suffering from genetic defects. Infectious diseases have also gained significant attention in the realm of gene therapy. The COVID-19 pandemic has accelerated research and development efforts in this area, leading to innovative gene-based therapies aimed at combating various viral infections.
There is a growing understanding that gene therapies can bolster the immune response and provide enduring protection against infectious agents, thus positioning this segment favorably for future advancements. Cardiovascular diseases represent a major health burden in the US, and gene therapy has begun to play a crucial role in addressing these challenges.
With innovative approaches that aim to repair or regenerate cardiovascular tissues, the potential for cardiac gene therapies to improve outcomes for patients suffering from heart disease is considerable. The exploration of novel gene constructs and delivery methods puts cardiovascular applications at the forefront of gene therapy advancements.
Lastly, neurological disorders encompass a range of debilitating conditions impacting cognitive and motor functions. There is an increasing focus on gene therapies that target conditions such as Alzheimer's disease, Parkinson's disease, and spinal muscular atrophy. The central nervous system presents unique challenges for gene delivery, yet ongoing advancements in technology are gradually unlocking possibilities for effective gene therapy solutions in this field.
Overall, the Therapeutic Area segment within the US Gene Therapy Market holds substantial promise across diverse health conditions, with each area offering distinct opportunities for innovation. Growth is fueled by a confluence of factors including technological advancements, an increased understanding of gene editing mechanisms, and substantial investment from both the public and private sectors.
This segment not only reflects the evolving landscape of modern medicine but also positions itself strategically within the healthcare framework, aiming to address some of the most pressing health challenges faced by the population today. Subsequently, the US Gene Therapy Market statistics indicate a robust framework for continued research, exploration, and integration of gene therapies across therapeutic landscapes.
Source: Primary Research, Secondary Research, MRFR Database and Analyst Review
The Technology segment of the US Gene Therapy Market encompasses several innovative approaches driving advancements in genetic treatments. Viral vectors are widely utilized due to their efficiency in delivering therapeutic genes to target cells, making them a prominent choice in the industry. CRISPR technology represents a groundbreaking tool for precise gene editing, enabling researchers to modify the genome at specific locations, which is revolutionizing treatment strategies.
Gene editing techniques are crucial for correcting genetic defects, thus holding exceptional potential in addressing inherited disorders. RNA therapy has gained attention for its capability to manipulate gene expression, offering promising avenues for treating various diseases. Additionally, Non-Viral Methods present an alternative to traditional vectors, utilizing physical and chemical methods for gene delivery, thus expanding the toolkit for gene therapy applications.
Overall, the importance of these technologies in the US Gene Therapy Market cannot be overstated as they collectively contribute to minimizing disease burden and shaping the future landscape of therapeutic options available for patients. As the market continues to advance, these technological innovations will play a crucial role in furthering research and expanding treatment possibilities for a diverse range of conditions.
The US Gene Therapy Market focuses significantly on applications ranging from Research to Clinical and Commercial use. This segment plays a pivotal role in advancing the understanding and treatment of genetic disorders and other diseases. Research applications facilitate breakthroughs by studying the mechanisms of gene therapies, which helps in identifying potential targets and refining techniques for therapeutic purposes.
Clinical applications are vital as they implement these therapies in real-world scenarios, significantly impacting patient outcomes and offering innovative solutions for previously incurable conditions. Meanwhile, the Commercial segment is important as it translates successful therapeutic advancements into accessible treatments, ultimately enhancing the quality of healthcare in the US.
As companies invest in these areas, the demand for novel gene therapies is rising, driven by the increasing prevalence of genetic disorders and advances in genetic engineering technologies. The US government has also shown strong support through initiatives aimed at expediting the approval processes for gene therapies, further fueling growth in the market. Overall, the application segment is crucial for the development and commercialization of gene therapies, which are expected to reshape the future of medicine.
The Route of Administration segment within the US Gene Therapy Market plays a critical role in the delivery and effectiveness of therapeutic agents. As the market continues to evolve, various methods such as Intravenous, Intramuscular, Intradermal, Intrathecal, and Oral administration are being explored and utilized. Intravenous administration is often favored for its rapid deliverability, allowing for immediate therapeutic effects, while Intramuscular administration is significant for its ease of application and potential for sustained release of gene therapeutics.
Intradermal routes are gaining attention due to their application in localized therapies, and the Intrathecal method is crucial for targeting the central nervous system, addressing conditions such as spinal muscular atrophy. Additionally, Oral administration offers a patient-friendly approach, increasing adherence and accessibility. Each of these methods plays a vital role in shaping treatment paradigms within the market, driven by ongoing innovations in drug delivery technologies.
The growing demand for these administration routes reflects the broader trends of increased patient-centric care and personalized medicine, highlighting the dynamic landscape of the US Gene Therapy Market.
The competitive landscape of the US Gene Therapy Market has gained significant attention due to its transformative potential in the treatment of various genetic disorders and diseases. Key players continue to emerge as research and development in gene therapies accelerate, supported by advancements in technology and scientific understanding. Companies within this sector focus on innovative approaches to gene modification, delivery systems, and regulatory strategies to establish their market presence.
As the market evolves, collaborations with academic institutions, licensing agreements, and clinical trials remain crucial for companies looking to maintain or enhance their competitive edge. Additionally, increasing investments and favorable regulatory environments are encouraging new entrants and established pharmaceutical companies alike to invest heavily in gene therapy solutions, which enhances the overall competitiveness of the market.
Novartis has established a strong foothold in the US Gene Therapy Market through its innovative product pipeline and strategic focus on developing breakthrough therapies for rare and chronic disorders. The company's expertise in cell and gene therapy has resulted in the successful commercialization of therapies that address critical unmet needs.
Novartis boasts a deep understanding of regulatory pathways, which allows for faster development and delivery of therapies to the market. The company's robust financial resources enable significant investments in cutting-edge R&D, fostering an environment conducive to scientific advancement. Furthermore, Novartis maintains strategic partnerships, providing access to new technologies and expanding its portfolio of gene therapies, all of which contribute to its competitive position in the US market.
Verve Therapeutics is making significant strides in the US Gene Therapy Market by focusing on innovative approaches to treating genetic conditions, particularly cardiovascular diseases. The company's pioneering use of gene editing technology positions it uniquely within the industry, allowing for the development of therapies aimed at delivering long-term solutions through a one-time treatment approach.
Verve Therapeutics is actively engaged in clinical trials that leverage its proprietary gene editing platform, showcasing its commitment to advancing healthcare solutions. The company has secured strategic collaborations and funding to enhance its R&D capabilities, further strengthening its market presence. The focus on collaboration extends towards potential mergers and acquisitions to access complementary technologies that align with its long-term goals of transforming the treatment landscape for inherited diseases in the US.
Overall, Verve Therapeutics stands out for its innovative approach and strategic positioning in the evolving gene therapy sector.
Recent months have seen significant developments in the US Gene Therapy Market. Novartis received FDA approval for its gene therapy treatment Zolgensma for spinal muscular atrophy in July 2023, marking a pivotal moment in the treatment landscape. In September 2023, Gilead Sciences announced positive trial results for its gene therapy targeting hemophilia, showcasing advancements in therapeutic applications.
Mergers and acquisitions maintain momentum, with Amgen acquiring Five Prime Therapeutics in May 2023 to augment its oncology portfolio, with implications for gene therapy advancements in cancer treatment. In terms of market valuation, companies like Bluebird Bio have experienced volatility, significantly impacting investor confidence and overall market stability.
Current affairs indicate that regulatory frameworks are evolving to accommodate rapid advancements in gene therapy, driven by the FDA's commitment to expedite both approvals and patient access to innovative treatments, reflecting a broader trend in personalized medicine. Major happenings over the last few years underscore the urgency for continued investment in Research and Development, with CRISPR Therapeutics making strides in gene editing technology, further enhancing competitive dynamics within the market.
| Report Attribute/Metric Source: | Details |
| MARKET SIZE 2018 | 1.81(USD Billion) |
| MARKET SIZE 2024 | 2.2(USD Billion) |
| MARKET SIZE 2035 | 8.5(USD Billion) |
| COMPOUND ANNUAL GROWTH RATE (CAGR) | 13.074% (2025 - 2035) |
| REPORT COVERAGE | Revenue Forecast, Competitive Landscape, Growth Factors, and Trends |
| BASE YEAR | 2024 |
| MARKET FORECAST PERIOD | 2025 - 2035 |
| HISTORICAL DATA | 2019 - 2024 |
| MARKET FORECAST UNITS | USD Billion |
| KEY COMPANIES PROFILED | Novartis, Verve Therapeutics, Gilead Sciences, Editas Medicine, Sangamo Therapeutics, Bluebird Bio, CRISPR Therapeutics, Regeneron Pharmaceuticals, Applied Genetic Technologies Corporation, Amgen, AbbVie, Astellas Pharma, Humanigen, Rocket Pharmaceuticals, Spark Therapeutics |
| SEGMENTS COVERED | Therapeutic Area, Technology, Application, Route of Administration |
| KEY MARKET OPPORTUNITIES | Aging population treatment, Increased investment in R&D, Expanding rare disease pipeline, Regulatory support for innovations, Growing demand for personalized medicine |
| KEY MARKET DYNAMICS | regulatory approvals, increasing investments, growing patient population, technological advancements, competitive landscape |
| COUNTRIES COVERED | US |
Frequently Asked Questions (FAQ) :
The US Gene Therapy Market is expected to be valued at 2.2 billion USD in 2024.
By 2035, the US Gene Therapy Market is projected to reach a value of 8.5 billion USD.
The expected CAGR for the US Gene Therapy Market from 2025 to 2035 is 13.074%.
The oncology segment is expected to dominate, with a projected value of 3.5 billion USD by 2035.
The market value for genetic disorders is estimated at 0.6 billion USD in 2024 and 2.1 billion USD in 2035.
Key players include Novartis, Gilead Sciences, and Bluebird Bio, among others.
The market size for infectious diseases is expected to reach 1.5 billion USD by 2035.
The cardiovascular diseases segment is projected to be valued at 1.2 billion USD by 2035.
Emerging trends include advancements in biotechnology and increased investment in clinical research.
The growth rate varies, with oncology and genetic disorders showcasing significant increases compared to other areas.
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