Gene Therapy Market - Global Industry Size, Share, Trends, Opportunity and Forecast, 2020-2030F

The Gene Therapy Market was valued at USD 6.54 Billion in 2024, and is expected to reach USD 11.29 Billion by 2030, rising at a CAGR of 9.53%. The gene therapy is described as the treatment of an illness by changing, replacing, or supplementing a missing or defective genetic combination that is responsible for the disease. The gene therapy has become one of the most desirable research goals in the fight against degenerative illnesses. The need for gene therapy is being driven by an increase in the number of cases of cancer and other chronic diseases all across the world.

The advancements in gene therapies are projected to be approved in the upcoming years, contributing to the gene therapy market’s growth during the forecast period. The gene therapies are designed to treat diseases by altering genetic information, such as inactivating malfunctioning genes or replacing a disease-causing gene with a healthy copy of gene. The gene therapy is being utilized to treat a variety of disorders and has demonstrated to be effective. This form of treatment can cure diseases such as diabetes, cancer, heart disease, and AIDS.

The strong pipeline of gene therapy products is anticipated to drive market growth over the forecast period. Researchers are actively working to make gene therapy more accessible in clinical settings. Numerous universities and research institutes are developing a diverse range of gene therapy products, which is expected to contribute significantly to revenue generation in the coming years. The number of clinical trials for gene therapy saw a notable increase between 2017 and 2018, following the FDA’s approval of the first gene therapy. According to the American Society of Gene & Cell Therapy (ASGCT), approximately 1,986 gene therapy products, including CAR T-cell therapies and other genetically modified cell treatments, are currently in development, further accelerating market expansion.

Key Market Drivers

Advancements in Genetic Research

The increasing understanding of the genetic basis of diseases has enabled scientists to identify specific genes responsible for various disorders. This knowledge is crucial for designing targeted gene therapies that can correct or replace defective genes, addressing the root cause of diseases. Genetic research has identified specific genetic mutations or abnormalities that contribute to various diseases, such as inherited genetic disorders and certain types of cancer. This has provided gene therapists with clear therapeutic targets, allowing them to develop more precise and effective treatments. Advancements in gene editing technologies, such as CRISPR-Cas9, TALENs, and zinc finger nucleases, have revolutionized gene therapy.

These techniques enable scientists to accurately and efficiently edit or modify genes, opening up new possibilities for treating genetic disorders. Genetic research has paved the way for personalized medicine, where treatments are tailored to an individual's genetic makeup. This approach enhances the effectiveness of gene therapies by considering genetic variations that might affect treatment outcomes, leading to better patient responses and reduced adverse effects. Genetic research has facilitated the development of advanced delivery systems that help target specific tissues or cells with greater precision. This has improved the efficiency of gene therapy treatments, minimized off-target effects and enhancing therapeutic outcomes.

The promising outcomes of genetic research have attracted substantial investment in the gene therapy sector. As the potential of gene therapies becomes more apparent, investors are more willing to fund research and development, driving innovation and market growth. For example, in December 2023, the U.S. FDA approved a gene therapy for sickle cell disease, marking the first-ever gene therapy based on CRISPR gene editing. Such groundbreaking advancements and product launches are expected to drive market growth over the forecast period.

Key Market Challenges

Technical Complexity

Developing effective gene therapies requires a deep understanding of genetics, molecular biology, and complex cellular processes. The technical complexity of designing therapies that accurately target and modify specific genes can lead to prolonged research and development timelines. Getting therapeutic genes into target cells poses a significant challenge. Developing efficient and safe delivery systems, such as viral vectors or nanoparticles, requires extensive optimization and testing. Technical difficulties in achieving precise and controlled delivery can affect the success of gene therapies. The interactions between modified genes and the host organism can sometimes lead to unintended consequences.

Technical complexities can make it difficult to predict the outcomes of gene therapies accurately, leading to safety concerns and regulatory challenges. Regulatory agencies require a high level of safety and efficacy data for gene therapy products due to their complexity and potential risks. Navigating the regulatory pathway for gene therapies can be challenging and time-consuming, hindering market growth. Manufacturing gene therapy products in a consistent and scalable manner is challenging due to the complexity of biological processes involved. Ensuring the quality, safety, and stability of gene therapies at a commercial scale can be technically demanding and expensive.

Key Market Trends

Expanding Therapeutic Targets

Identifying new therapeutic targets means gene therapy can be applied to a wider range of medical conditions beyond traditional monogenic disorders. This diversity includes complex diseases like cancer, cardiovascular diseases, neurodegenerative disorders, and rare diseases, expanding the addressable patient population. The availability of gene therapies for multiple therapeutic targets attracts a larger patient base, driving market growth by increasing the potential customer pool and commercial opportunities. The discovery of new therapeutic targets stimulates research and development efforts by both established pharmaceutical companies and smaller biotechnology firms.

This influx of investment fuels innovation and accelerates the development of gene therapies for various indications. Expanding therapeutic targets leads to a more diverse portfolio of clinical trials. This diversity enhances the clinical trial landscape, allowing researchers to explore different patient populations, endpoints, and treatment approaches, ultimately advancing the field. As gene therapies target a wider array of diseases, regulatory agencies may provide specialized pathways or guidelines for specific indications. This attention can expedite regulatory approval processes and facilitate market entry.

The availability of gene therapies for more common and relatable diseases can increase public awareness and acceptance of the technology. Positive outcomes in well-known conditions can build trust and reduce skepticism. Expanding therapeutic targets can lead to collaborations between academic researchers, industry partners, and patient advocacy groups. Collaborations strengthen expertise, resources, and support networks, fostering innovation and growth. The potential for gene therapies to target various indications attracts diverse investors interested in contributing to the development of treatments for specific diseases, fostering a competitive market landscape.

Key Market Players

• REGENXBIO Inc.
• Oxford Biomedica PLC.
• Voyager Therapeutics Inc.
• Human Stem Cells Institute
• Dimension Therapeutics, Inc.
• Bristol-Myers Squibb Company
• Sanofi
• F. Hoffmann-La Roche Ltd
• bluebird bio, Inc.
• Novartis AG
• uniQure NV.

Report Scope:

In this report, the Global Gene Therapy Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

Gene Therapy Market, By Vector Type:

• Viral Vector
• Non-Viral Vector

Gene Therapy Market, By Delivery Method:

• In-Vivo
• Ex-Vivo

Gene Therapy Market, By Indication:

• Rare Diseases
• Cancer
• Neurological Diseases
• Others

Gene Therapy Market, By Region:

• North America
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia-Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Gene Therapy Market.

Available Customizations:

With the given market data, the publisher offers customizations according to a company's specific needs. The following customization options are available for the report.

Company Information

• Detailed analysis and profiling of additional market players (up to five).

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