U.S. Gene Therapy Market Size, Share & Growth Report, 2033

According to Nova One Advisor, the U.S. gene therapy market size was valued at USD 3.19 billion in 2023 and is poised to grow from USD 3.80 billion in 2024 to USD 18.50 billion by 2033, growing at a CAGR of 19.22% in the forecast period (2024-2033)

The gene therapy market includes DNA-based therapies involving the delivery of a functional copy of a defective gene or the correction of a mutated gene. DNA-based therapies are delivered using various methods such as viral vectors, plasmids, or gene editing tools. The robust gene therapy pipeline is expected to boost the market growth in the forecast period. Researchers are working to make gene therapy available at clinics. Various universities and institutes have a broad portfolio of products in the pipeline, which, in turn, is expected to boost revenue generation in the future.

The COVID-19 pandemic has had a notable impact on the U.S. gene therapy industry. While the market for gene therapies is expected to grow significantly in the years ahead, the pandemic caused some delays in clinical trials. Nevertheless, the pandemic also highlighted the importance of gene therapy in preventing and treating diseases, which could lead to increased investment and funding for the field. Despite the challenges posed by the pandemic, the U.S. gene therapy industry remains poised for growth and innovation in the future.

Technological advancements in gene editing, viral vector development, and delivery mechanisms have propelled gene therapy from the realms of speculation to clinical application. Innovations like CRISPR-Cas9 technology have revolutionized gene editing, allowing for precise modifications in genetic sequences with unprecedented accuracy and efficiency. Similarly, advancements in viral vectors, including adeno-associated viruses (AAVs) and lentiviruses, have enhanced the delivery of therapeutic genes to target cells, improving the efficacy and safety of gene therapy treatments.

An increase in funding and investments in gene therapy advancements is expected to provide lucrative growth opportunities to market players. For instance, in January 2022, Ori Biotech raised more than USD 100 million in a Series B funding to introduce a novel cell & gene therapy developing platform. This funding allowed for a rapid transition from precommercialization to market launch. In October 2023, NIH granted nearly USD 40 million to Yale School of Medicine which would support the development of phase 2 CRISPR-based gene therapy platform which would directly target genetic brain diseases such as H1-4 (HIST1H1E) syndrome and Angelman syndrome.

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Key Takeaways:

• The Large B-cell lymphoma segment held the largest revenue share of 36.14% in 2023.
• The inherited retinal disease segment is expected to register a significant CAGR over the forecast period.
• The lentivirus dominated with a revenue share of 43.79% in 2023.
• The AAV segment is expected to grow at a highest CAGR over the forecast period.
• The intravenous segment dominated the market share in 2023 and is anticipated to grow at a higher CAGR over the forecast period.
• The others segment is expected to grow at a significant CAGR over the forecast period.

Is gene therapy safe?

The first gene therapy trial was run more than thirty years ago. The earliest studies showed that gene therapy could have very serious health risks, such as toxicity, inflammation, and cancer. Since then, researchers have studied the mechanisms and developed improved techniques that are less likely to cause dangerous immune reactions or cancer. Because gene therapy techniques are relatively new, some risks may be unpredictable; however, medical researchers, institutions, and regulatory agencies are working to ensure that gene therapy research, clinical trials, and approved treatments are as safe as possible.

Comprehensive federal laws, regulations, and guidelines help protect people who participate in research studies (called clinical trials). The U.S. Food and Drug Administration (FDA) regulates all gene therapy products in the United States and oversees research in this area. Researchers who wish to test an approach in a clinical trial must first obtain permission from the FDA. The FDA has the authority to reject or suspend clinical trials that are suspected of being unsafe for participants.

The National Institutes of Health (NIH) also plays an important role in ensuring the safety of gene therapy research. NIH provides guidelines for investigators and institutions (such as universities and hospitals) to follow when conducting clinical trials with gene therapy. These guidelines state that clinical trials at institutions receiving NIH funding for this type of research must be registered with the NIH Office of Biotechnology Activities. The protocol, or plan, for each clinical trial is then reviewed by the NIH Recombinant DNA Advisory Committee (RAC) to determine whether it raises medical, ethical, or safety issues that warrant further discussion at a RAC public meeting.

An Institutional Review Board (IRB) and an Institutional Biosafety Committee (IBC) must approve each gene therapy clinical trial before it can be carried out. An IRB is a committee of scientific and medical advisors and consumers that reviews all research within an institution. An IBC is a group that reviews and approves an institution’s potentially hazardous research studies. Multiple levels of evaluation and oversight ensure that safety concerns are a top priority in the planning and carrying out of gene therapy research.

The field of gene therapy is at a pivotal moment. With a recent explosion of clinical activity, it is well-poised for further growth — but there are also existing challenges it will need to overcome. As the industry continues to evolve, here are five key trends Novartis Gene Therapies believes will be critical to watch.

• Gene therapy will drive transformation for patients, science and industry as it expands

Gene therapies have already transformed patient lives, with four gene therapy products approved in the United States. But this is just the beginning.

The field is poised to grow exponentially, with investigational new drug (IND) applications with the FDA for ongoing gene therapy clinical studies, and several new gene therapy indications expected to secure US and EU market approval by 2033.

Further scientific and technological advancements will continue to grow the number of therapies available, expanding their impact to more people and treating more prevalent diseases. The ability to introduce inhibition into the disease pathways through gene therapy, thereby blocking some function that is dysregulated in the course of the disease, will be one breakthrough the industry needs to move beyond genetic diseases and into chronic diseases that impact broader populations.

• US health authorities will push higher standards, but also show greater flexibility

In the United States, health authorities are expected to advocate for elevated standards while also demonstrating increased flexibility. This means that there will likely be a push for stricter regulations and guidelines to ensure the safety and efficacy of healthcare products and services. However, there will also be an emphasis on being adaptable and responsive to changing circumstances, such as emerging health threats or technological advancements. This balance between rigor and adaptability aims to promote innovation while safeguarding public health and well-being.

• Manufacturing will be key to the success of gene therapy growth and innovation:

While traditional therapeutics are designed to be easily replicated, gene therapies require an innovative manufacturing process supported by specialized equipment and personnel.

It has become apparent that regulators prefer sponsors have commercial-grade process and product ready at the time of clinical development. While investing in manufacturing is historically difficult without a close-to-market product, an early focus on manufacturing to advance a product is now non-negotiable for a product to be commercially viable and reach patients.

For example, Novartis Gene Therapies developed a manufacturing process where none existed, and as the field advances, the company continues to innovate. It has continually made process improvements — including scaling to larger bioreactors, shortening turnaround time, and other efficiency gains — allowing Novartis Gene Therapies to meet the sizeable need for treatment and unlock potential for new patients.

• Stakeholders will work together to encourage health systems to adopt new paradigms that reflect the value gene therapies bring to patients and, more broadly, society

One-time gene therapies provide incredible innovation, but they also challenge a healthcare system built around chronic treatment. Higher one-time upfront payment for gene therapy benefits is a vastly different and new concept.

Innovative financing models will play a significant role in realizing the potential benefits of these therapies. For example, outcomes-based financing models effectively articulate the value of a product and provide an opportunity for payers to recognize the potential of one-time, interventional strategies.

In the future, patient perspective will be at the heart of all value assessments. Patients — particularly those living with rare genetic diseases — may experience significant impacts on health and quality of life, including disability and a mental and emotional toll driven by anxiety due to disease progression, inability to conduct activities of daily living, and feelings of isolation. Yet, these collateral impacts are not always taken into account when evaluating medicines.

The future is likely to incorporate multi-stakeholder dialogue, centered around the patient, for a greater consensus on the determinants of a product’s value.

• There will be no one-size-fits-all approach for a therapy’s path to market

The path of each therapy, from development to commercialization, will differ based on factors like patient needs and reimbursement pressures.

The field will learn to adapt to this new reality and collect best practices as each new therapy is brought to market. Already-established platforms will play a vital role moving forward, providing consistency and experience across manufacturing, analytics, biodistribution, and safety.

Novartis Gene Therapies is reimagining medicine to transform the lives of people living with rare genetic diseases. Beginning with the company’s transformative gene therapy for spinal muscular atrophy, Novartis Gene Therapies has played a leading role in the journey the field has taken to date. Visit our website to learn more about how the company is poised to leverage these trends into the future.

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Indication Insights

Based on indication, the market is segmented into Large B-cell lymphoma, multiple myeloma, spinal muscular atrophy (SMA), acute lymphoblastic leukemia (ALL), melanoma (lesions), inherited retinal disease, beta-thalassemia major/SCD, and others. The Large B-cell lymphoma segment held the largest revenue share of 36.14% in 2023. Diffuse large B-cell lymphoma (DLBCL) is the most common non-Hodgkin Lymphoma (NHL) subtype. It is estimated to account for around 30%-40% of all cases. For instance, in May 2018, the U.S. FDA approved Kymriah for the treatment of adult patients with refractory or relapsed large B-cell lymphoma, including DLBCL.

The inherited retinal disease segment is expected to register a significant CAGR over the forecast period. An increase in R&D activities for the development of gene therapies for treating ocular diseases, including inherited retinal disease, has driven the segment. For instance, a therapy for the treatment of patients with Leber congenital amaurosis (LCA) using AAV to deliver the gene for human RPE65 to the retinal pigment epithelium is currently in phase 3 clinical trial which is expected to complete in 2029.

Vector Type Insights

Based on vector type, the market is segmented into retrovirus & gamma retrovirus, lentivirus, AAV, adenovirus, modified herpes simplex virus, and others. The lentivirus dominated with a revenue share of 43.79% in 2023. Gene therapy viral vectors extracted from lentiviruses offer many advantages over conventional gene vectors like retrovirus such as ability to provide stable and long-term gene expression, ability to induct themselves to non-dividing cells, possible generation of replication adept lentiviruses during vector preparation, and many more. This contributes to the segment’s growth, thus driving its dominance in the market. In December 2023, a numerous randomized clinical trial was started to evaluate OTL-203 (gene therapy) and stem cell transplant (standard of care) in individuals affected with MPS-IH (Hurler syndrome), is using lentivirus. This clinical trial is expected to be complete at the start of 2031. Thus, the rising usage of lentivirus in clinical trials is anticipated to boost the growth of the segment.

The AAV segment is expected to grow at a highest CAGR over the forecast period. This is attributed to the increasing demand due to the result from clinical trials pertaining to development of ocular and orthopedic gene therapy treatment exhibiting increased efficacy and efficiency. Moreover, the growing number of clinical trials and R&D activities are further anticipated to contribute to the growth of the segment over the forecast period. For instance, an ongoing clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa uses AAV as their vector which is proposed to get completed by the end of 2029.

Route Of Administration Insights

Based on route of administration, the market is segmented into intravenous and others. The intravenous segment dominated the market share in 2023 and is anticipated to grow at a higher CAGR over the forecast period. Intravenous administration typically involves shorter treatment durations and less invasive procedures compared to alternative routes such as intramuscular or subcutaneous injections. This increases the convenience and reduces the discomfort for patients thereby improving the treatment adherence and overall patient satisfaction, driving preference for intravenous gene therapy products.

The others segment is expected to grow at a significant CAGR over the forecast period. This segment includes transcutaneous, subretinal, topical, and intravitreal routes of administrations. RGX-314 is being developed as a unique one-time gene therapy for the treatment of patients with neovascular age-related macular degeneration (wet AMD) which is expected to be completed by 2026. Thus, the rising number of clinical trials is expected to drive the growth of the market.

U.S. Gene Therapy Market Recent Developments

• In February 2024, Encoded Therapeutics announced the launch of gene therapy trial for Dravet syndrome. FDA granted the organization for the investigational new drug application.
• In August 2022, ZYNTEGLO (betibeglogene autotemcel), a one-time gene therapies specifically created to treat the fundamental genetic cause of beta-thalassemia in pediatric and adult patients who necessitate regular Red Blood Cell transfusion, was approved by the U.S. FDA.
• In June 2022, REGENXBIO Inc. established its new Manufacturing Innovation Center, a gene therapy manufacturing facility. The company invested USD 65 million in the new facility, allowing end-to-end control of gene therapy manufacturing in Maryland.

Key U.S. Gene Therapy Company Insights

The market players are adopting product approval to increase the reach of their products and improve the availability of their products in diverse geographical areas, along with expansion as a strategy to enhance production/research activities. In addition, several market players are acquiring smaller players to strengthen their market position. This strategy enables companies to increase their capabilities, expand their product portfolios, and improve their competencies.

• In December 2023, Tome Biosciences announced the investment of over USD 200 million to advance the programmable genomic integration platform which would enable DNA sequencing into any programmed genomic location. This investment is anticipated to contribute to the growth of the market.
• In January 2023, Spark Therapeutics and Neurochase established a strategic collaboration to develop Neurochase’s unique delivery technology for use with selected gene treatments for rare disorders in the CNS. In this agreement, Neurochase will contribute its extensive knowledge in direct drug delivery technology to Spark’s premier AAV platform.
• In September 2022, Eli-cel, also known as SKYSONA, was given accelerated approval by the U.S. FDA to decrease the course of neurological dysfunction in 4- to 17-year-old boys with early, active Cerebral Adrenoleukodystrophy (CALD). In addition, the company declared that the clinical hold that had been placed on the eli-cel clinical development program had been lifted.

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.

Segments Covered in the Report

This report forecasts revenue growth at country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2033. For this study, Nova one advisor, Inc. has segmented the U.S. Gene Therapy market.

By Indication

• Large B-cell Lymphoma
• Multiple Myeloma
• Spinal Muscular Atrophy (SMA)
• Acute Lymphoblastic Leukemia (ALL)
• Melanoma (lesions)
• Inherited Retinal Disease
• Beta-thalassemia Major/SCD
• Others

By Route Of Administration

• Intravenous
• Others

By Vector Type

• Lentivirus
• RetroVirus & Gamma RetroVirus
• AAV
• Modified Herpes Simplex Virus
• Adenovirus
• Others

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