Gene Therapy Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Vector Type (Viral Vector, Non-Viral Vector), By Delivery Method (In-Vivo, Ex-Vivo), By Indication (Rare Diseases, Cancer, Neurological Diseases, Others), By Region & Competition, 2020-2030F

Market Overview

Global Gene Therapy Market was valued at USD 6.54 Billion in 2024 and is expected to reach USD 11.29 Billion by 2030 with a CAGR of 9.53% during the forecast period. 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.

Rising Prevalence of Genetic Disorders

The increasing number of individuals diagnosed with genetic disorders highlights the unmet medical need for effective treatments. Conventional treatments for many genetic disorders often focus on managing symptoms rather than addressing the root cause. Gene therapy presents an opportunity to provide targeted and long-lasting solutions for these disorders. The growing prevalence of genetic disorders means that there is a larger patient population that could potentially benefit from gene therapy. This expanded patient pool translates to a higher demand for gene therapy treatments and a larger market opportunity. The rising prevalence of genetic disorders makes the gene therapy market more attractive to pharmaceutical companies, biotechnology firms, and investors. The potential for a sizable customer base and the ability to provide innovative solutions to unmet medical needs can drive increased investment and research in the field. The prevalence of genetic disorders provides ample opportunities for conducting clinical trials to test the safety and efficacy of gene therapy treatments. This contributes to the advancement of research and development in the gene therapy sector and supports the growth of the market. Genetic disorders are not limited to specific regions; they affect populations worldwide. This global reach provides gene therapy companies with opportunities to develop and market their treatments on a global scale, further contributing to market growth. According to the World Health Organization (WHO), 10 out of every 1,000 individuals are affected by genetic disorders, with over 70 million people worldwide living with these conditions. Additionally, genetic disorders account for more than 40% of global infant mortality. To tackle these challenges, numerous research initiatives are focused on developing effective methods to deliver targeted therapies directly to affected organs or cells.

Collaborations and Partnerships

Gene therapy involves a multidisciplinary approach, including molecular biology, genetics, biotechnology, clinical research, and regulatory affairs. Collaborations bring together experts from different fields, allowing them to share knowledge, insights, and best practices. This collective expertise fosters innovation and drives the development of more advanced and effective gene therapies. Collaborative efforts can significantly speed up the research and development process. By combining resources, data, and technologies, researchers and companies can overcome challenges more efficiently, reducing the time required to bring new gene therapies from concept to clinical application. Partnerships provide access to specialized technologies and tools that may not be available within a single organization. This access can lead to breakthroughs in gene delivery systems, gene editing techniques, and other critical components of gene therapy development. Gene therapy manufacturing can be challenging due to the need for precise and consistent production processes. Partnerships can facilitate the development of scalable manufacturing techniques, ensuring the production of sufficient quantities of gene therapies for clinical trials and commercialization. Regulatory approval is a critical step in bringing gene therapies to market. Collaborations with regulatory experts and consultants can help navigate the complex regulatory landscape, ensuring that therapies meet safety and efficacy standards and gain timely approvals.

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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.

High Development Costs

Initial research to identify target genes, develop therapeutic constructs, and validate their efficacy in preclinical models can be expensive. This stage requires significant investment in laboratory resources, equipment, and skilled personnel. Conducting clinical trials is a critical step in the development of any medical intervention, including gene therapies. The cost of designing, executing, and monitoring clinical trials (Phase I to Phase III) can be substantial due to factors such as patient recruitment, regulatory compliance, data collection, and safety monitoring. Meeting regulatory requirements set by agencies such as the FDA and EMA demands rigorous testing, documentation, and quality control. The cost of compiling data and preparing submissions for regulatory approval can be significant. Developing consistent, high-quality gene therapy products for clinical trials and commercialization involves complex and costly manufacturing processes. Ensuring the scalability, stability, and safety of manufacturing adds to development expenses. Building a team of experts in gene therapy, molecular biology, clinical research, and regulatory affairs is essential for successful development. Attracting and retaining skilled professionals can be costly, especially given the specialized nature of the field. Cutting-edge technologies, such as gene editing tools and viral vectors, are crucial for developing advanced gene therapies. Acquiring and implementing these technologies requires substantial investments.

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.

Growing CRISPR-Based Therapies

CRISPR-Cas9 technology allows researchers to edit genes with unparalleled precision. This precision is crucial for correcting or modifying specific genetic mutations that underlie various diseases. The ability to target and modify genes accurately enhances the effectiveness and safety of gene therapies. CRISPR-Cas9 enables the modification of a wide range of genetic targets, from single nucleotide changes to larger genomic segments. This expands the potential applications of gene therapy to include a broader spectrum of genetic disorders, including both monogenic and complex conditions. The simplicity and accessibility of CRISPR technology have accelerated gene therapy research. Scientists can design and implement experiments more efficiently, leading to faster discoveries and a quicker translation of findings into potential therapies. CRISPR-based therapies contribute to a more diverse pipeline of potential treatments. As researchers explore new gene targets and develop innovative therapies, the gene therapy market gains momentum with a wider range of options. The precise nature of CRISPR-Cas9 editing can streamline the regulatory process by providing clear evidence of the intended genetic changes. This can facilitate faster approvals for clinical trials and eventual market entry. The accessibility of CRISPR technology has encouraged global collaboration among researchers, enabling the sharing of knowledge, techniques, and resources. This collaborative environment accelerates progress and drives market growth. The potential of CRISPR-based therapies has led to the establishment of new biotech companies dedicated to gene editing and gene therapy. This increased industry presence contributes to competition, innovation, and market expansion.

Segmental Insights

Delivery Method Insights

Based on the Delivery Method, the In-Vivo segment held the largest market share in the market. In vivo gene therapy avoids the need for invasive procedures such as surgical implantation. This non-invasive nature makes it more acceptable to patients and reduces the risks associated with surgical interventions. In vivo gene therapy can be administered systemically, making it accessible to a broader range of patients. This approach is particularly beneficial for patients with diseases affecting multiple organs or tissues. In vivo gene therapy has the potential to affect multiple target cells or tissues throughout the body. This systemic effect is advantageous for conditions that involve widespread genetic abnormalities or diseases affecting multiple organs. Successful in vivo gene therapy can result in long-lasting effects, as the introduced genetic material continues to express itself within the patient's body. In vivo gene therapy can address a wide range of therapeutic targets, from monogenic disorders to complex diseases like cancer, cardiovascular diseases, and neurodegenerative conditions. The non-invasive nature and systemic effect of in vivo gene therapy can positively influence patient acceptance, making clinical trial recruitment and market adoption smoother. The versatility of in vivo gene therapy allows for applications in a variety of medical fields, attracting a wider range of researchers, developers, and companies to the gene therapy market.

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Regional Insights

North America held the largest market share in 2024, primarily due to North America, particularly the United States, has a robust research and innovation ecosystem that includes top universities, research institutions, and biotechnology companies. This environment fosters cutting-edge gene therapy research and development. North America attracts significant investments from both private and public sources into gene therapy research and clinical trials. Venture capital firms, pharmaceutical companies, and government grants contribute to funding the advancement of gene therapies. The region boasts well-established clinical trial infrastructure, including experienced investigators, specialized medical centers, and patient recruitment networks. This infrastructure accelerates the progress of clinical trials and speeds up the translation of research into therapies. North America is home to numerous biotechnology companies focused on gene therapy. These companies are at the forefront of developing innovative therapies and technologies, contributing to the overall market growth. The advanced healthcare infrastructure and access to specialized medical expertise in North America provide a conducive environment for the clinical application and adoption of gene therapies. Furthermore, an increase in funding and investments in this sector is expected to provide lucrative growth opportunities to market players. Several biopharma companies are investing in this sector for novel product launches. For instance, in January 2022, Ori Biotech raised more than USD 100.0 million in Series B funding to introduce a novel cell & gene therapy developing platform. This funding allowed for a rapid transition from pre-commercialization to market launch.

Recent Developments

• In July 2024, Beacon Therapeutics secured USD 170 million in a Series B venture capital round to accelerate the development of its lead gene therapy candidate, AGTC-501, for the treatment of X-linked retinitis pigmentosa.
• In April 2024, the U.S. FDA approved BEQVEZ®, developed by Pfizer, for the treatment of hemophilia B.
• In March 2024, bluebird bio formed a strategic partnership with Medicaid to implement outcome-based reimbursement for its gene therapy, LYFGENIA.
• In March 2024, the U.S. FDA approved Lenmeldy™, developed by Orchard Therapeutics, for treating children with metachromatic leukodystrophy.
• In January 2024, the U.S. FDA approved Casgevy®, developed by CRISPR Therapeutics and Vertex Pharmaceuticals, for the treatment of beta-thalassemia.
• In January 2024, Biogen and Ginkgo Bioworks announced the successful completion of their gene therapy collaboration focused on AAV-based vectors. This achievement is expected to drive increased demand for gene therapies in the coming years.
• In June 2023, Sangamo Therapeutics Inc. entered into a licensed agreement with Voyager Therapeutics Inc. for the development of the epigenetic regulation treatment, a gene therapy, for treating prion disease.
• In May 2023, Oxford BioMedica Plc launched the TetraVectaTM System which is the 4th generation lentiviral vector delivery system. It is useful for gene therapy for delivering therapeutic genes into target cells.
• In December 2022, the collaboration between Cellectis SA and Mayflower Bioventures resulted in the launch of Primera Therapeutics. The purpose was to develop gene editing therapies for the treatment of mitochondrial disease.
• In June 2022, the alliance between Bristol-Myers Squibb Company and Immatics NV expanded for the development of multiple allogenic off the shelf TCR-T, and CAR-T programs. Both these are gene therapy programs that modify a patient’s T cells to target and attack cancer cells.
• In January 2022, Novartis AG collaborated with Alnylam to explore target therapies for restoring liver function. In this collaboration, Novartis utilized Alnylam’s siRNA technology which is a gene therapy to develop a treatment for the regrowth of functional livers.

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:

o   Viral Vector

o   Non-Viral Vector

• Gene Therapy Market, By Delivery Method:

o   In-Vivo

o   Ex-Vivo

• Gene Therapy Market, By Indication:

o   Rare Diseases

o   Cancer

o   Neurological Diseases

o   Others  

• Gene Therapy Market, By Region:

o   North America

§  United States

§  Canada

§  Mexico

o   Europe

§  France

§  United Kingdom

§  Italy

§  Germany

§  Spain

o   Asia-Pacific

§  China

§  India

§  Japan

§  Australia

§  South Korea

o   South America

§  Brazil

§  Argentina

§  Colombia

o   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.

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Company Information

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