Gene Therapy Market Driven by Technological and Lifestyle Shifts

The global gene therapy market size was estimated at USD 2270 million in 2024 and is projected to be worth around USD 10,854 million by 2034, growing at a CAGR of 16.93% from 2025 to 2034. The entire therapy market is driven by many factors such as the rising incidence of changing lifestyles, and medical technological advancements. The North America region led the market, while Asia Pacific is set to grow at the highest CAGR during the forecast period.

Highlights of the Gene Therapy Market

• North America dominated the global market in 2024.
• Asia Pacific is anticipated to grow at the fastest rate in the market during the forecast period.
• Europe is expected to grow at a notable rate during the forecast period.
• By therapy type, the gene augmentation segment held a dominant presence in the market and is expected to grow at the fastest CAGR during the forecast period of 2025 to 2034.

Industry Valuation and Growth Rate Projection

The gene therapy market deals with technology that examines the possibility of treating or avoiding disease in humans by gene modification or replacement, and as such, it normally looks toward genetic disorder correction at its very source by either deleting or adding to the DNA within the cells of a patient. Given the increased occurrence of chronic and genetic diseases such as cancers, cardiovascular problems, and rare hereditary disorders, the market for gene therapy has grown immensely around the globe. Genomic advances, higher spending of funds for research and development, and a strong pipeline of gene therapy candidates further compound the growth. The shift from conventional therapies, such as chemotherapy, to gene-based therapies that are targeted and low in toxicity is one of the main factors driving the growth of demand. High development costs, regulatory hurdles, and serious adverse side effects are some of the barriers.

Gene Therapy Market: Stats and Figures

• 32 gene therapies (including genetically modified cell therapies) have been approved on a global scale.
• In the first quarter of 2024, CT-053 (CARsgen) was approved in China for the treatment of multiple myeloma, and Beqvez (Pfizer) was approved for hemophilia B in Canada.
• Currently under development are 2,093 gene therapies, including genetically modified cell therapies such as CAR-T cell therapies. They cover 52% of the entire pipeline in gene, cell, and RNA therapy.
• Since the last quarter, the share of gene therapy trials treating non-oncology indications has increased by four percentage points to 43%, which falls in line with the continuing trend of shifting applications to non-oncology.

How AI Has Benefited the Market

Artificial intelligence is acting as a change agent for the gene therapy market, especially when it involves drug discovery, manufacturing, and personalization. AI identifies mutations in genes for diseases through the analysis of genomic data and also builds predictive models. It optimizes gene therapy candidates by sorting through complex datasets. From the manufacturing perspective, AI brings enhanced efficiency due to adaptive systems, real-time monitoring of cell selection, and robotics, whereas personalized medicine offers AI applications in developing patient-specific therapies by analyzing an individual genetic profile and predicting response to treatment. AI developments shorten genetic engineering timelines, cut costs, and cater treatment pathways with higher precision amid gene therapy's fast-paced environment.

What Are the Major Market Drivers in the Gene Therapy Market?

• The major market drivers in the gene therapy market are advancements in gene-editing technologies like CRISPR-Cas9, increasing prevalence of genetic and rare diseases, rising investments in research and development, and the growing demand for personalized medicine.

Advanced Editing Technologies

The market has witnessed massive growth due to advanced gene editing technologies, such as CRISPR-Cas9, which allow precise and targeted modification of genetic material with fewer off-target effects, thereby enhancing the safety and efficacy of the therapies. Such technologies have enabled genetic disorders to be treated, the commercialization of therapies, and less expensive treatment. Gene therapies proved to be important during the COVID-19 pandemic, where Moderna and Pfizer used the biotechnology as a viable strategy for developing their vaccines. Both vaccine manufacturers used COVID-19 vaccine gene therapy to product many copies of a fragment of the virus. Moderna’s vaccine, mRNA-1273, was 94.5% effective in reducing the risk of COVID-19 infection.

Investment

Interest by investigators, medical practitioners, and pharmaceutical companies has thus been one of the most important contributors driving this rapid growth. Another reason for the fast pace has to do with the availability of funding from both the public and the private sector for preclinical studies, clinical trials, and the commercialization of therapies and infrastructure, along with many grants from governments, venture capitalists, and commercial interests from pharmaceutical companies.

What Are Major Gene Therapy Market Restraints?

The growth of the market for gene therapy is restrained by factors such as the high cost of development and treatment, limited manufacturing scalability, complex regulatory frameworks, and ethical concerns.

Complicated Regulatory Frameworks

Potential risks and long-term consequences cause this market to operate under stringent guidelines, which are constantly evolving. In this respect, agencies such as the FDA could insist on exceptionally thorough collections of safety and efficacy data, which creates an extended period for product legal authorization. For smaller biotechnology companies with fewer resources, trying to interpret clinical trial requirements, patient safety materials, and post-market surveillance procedures becomes a heavy burden.

Limited Manufacturing Scale

Plus, the fact that these regulations keep changing adds to uncertainty, causing delays in product development and scaring investors away. Manufacturing gene therapies, especially viral vectors, is an extremely specialized process that requires resourcing along the lines of bioprocessing technologies and cleanroom-grade environments. Downscaling from laboratory generation to commercial generation is very expensive and time-consuming. This hampers the market's ability to efficiently serve growing demand and create routes for wider access.

What are the Gene Therapy Market Opportunities?

The opportunities in the gene therapy market associated are primarily driven by advancements in genetic engineering, the growing demand for treatments for chronic diseases caused by a rise in the geriatric population, along with developing pathogen resistance to antibiotics and rising investments in biotechnology research.

• In March 2025, Novartis announced it would be expanding the trials for Zolgensma (onasemnogene abeparvovec) to older children with spinal muscular atrophy. The Phase III results for the drug are pending, but the company seems confident enough to seek regulatory approval in the first half of the year for an improved iteration of their one-time gene therapy given through spinal injection.

Precision Medicine

The gene therapy market presents two major opportunities: precision medicine and personalized therapies. While precision medicine develops treatments tailored to a person's genetic constitution, this involves treating the underlying cause of diseases rather than producing dysfunction. Such targeted treatment, with minimal side effects, is a better fit for the treatment of rare and chronic genetic disorders.

Personalized Therapies

Significantly, with the advancement of genomics through technologies such as biomarker discoveries or the creation of next-generation sequencing platforms, molecular diagnosis and treatment have become commercially feasible. As healthcare systems are moving from an episodic care framework to a patient-centric or individualized care framework, gene therapy is an integral part of this transition. Further, mechanisms such as CRISPR and AI-powered platforms have started opening newer growth avenues in gene therapy by enabling precision genetic modifications and supporting manufacturing. This makes gene therapies highly accessible and commercially viable.

Industry Leader Announcements

• Fabio Romerio, Associate Professor of Molecular and Comparative Pathobiology at Johns Hopkins School of Medicine:

“Our goal is to enhance this natural mechanism through gene therapy, possibly enabling long-term viral suppression with just a single treatment.”

• Jean Tang, MD, PhD, a professor of dermatology who treats children with EB at Lucile Packard Children’s Hospital Stanford:

“With our novel gene therapy technique, we successfully treated the hardest-to-heal wounds, which were usually also the most painful ones for these patients.”

Gene Therapy Market Segmental Insights

By Therapy Type

Gene augmentation segment dominated the market and is expected to grow at the fastest rate during the forecast period. Gene augmentation is a gene therapy strategy whereby a healthy gene is introduced into cells where an original gene is missing or defective. The goal is to restore normal functions to cells and, in some instances, cure or significantly lessen the onset of genetic diseases. It is effective in treating inherited disorders such as cystic fibrosis, hemophilia, and some retinal diseases. Gene augmentation is also under exploration for the treatment of cancer by making the immune system cells attack cancer cells effectively. The advantages include a possibility for long-term correction of diseases, restoration of lost functions, and a direct delivery procedure that is minimally invasive. Yet, the challenges include the larger need for delivery methods and the unknown effects over the long term, along with ethical considerations, especially regarding germ line interventions.

Geographical Insights

Which region dominated the gene therapy market?

North America dominated the market in 2024. The region has a well-built regulatory landscape, especially in the United States, where the FDA is a proponent of accelerated approval pathways and innovation. These bodies are the highest investors in higher research on gene therapies, supporting academic institutions and public-private partnerships. The existing biopharmaceutical companies and contract manufacturing organizations in the United States and Canada are driving regular advancements in the field. Rocket Pharmaceuticals, a late-stage biotechnology company, announced clearance from the U.S. Food and Drug Administration in June 2025 for RP-A701, an AAVrh.74-based gene therapy. This FDA approved gene therapy is designed to treat a rare, inherited heart condition called BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), characterized by progressive ventricular enlargement and impaired systolic function.

The Asia Pacific region is anticipated to be the fastest-growing market for gene therapy with an increasing occurrence of chronic and genetic diseases, developing healthcare facilities, and awareness regarding advanced therapeutic solutions. China, Japan, South Korea, and India are investing heavily in biotechnology and genomic medicine, fostering local gene therapy manufacturing and research and development through various initiatives, also setting up an environment conducive for clinical trials. Europe is expected to emerge as the most profitable region for the gene therapy market throughout the forecast period. The region boasts a highly developed healthcare infrastructure, reimbursement policies, and numerous biotechnology and pharmaceutical companies. Growth is being fueled by funding from both public and private sectors, collaborative research efforts, and a strong focus on addressing unmet medical needs.

Recent Developments in the Gene Therapy Market:

• In June 2025, Johns Hopkins University scientists discovered that gene therapy could induce HIV into a permanent dormant state, potentially offering a long-term, durable treatment for AIDS by manipulating and amplifying the "antisense transcript" (AST).
• In May 2025, the Alzheimer's Association awarded four $5 million grants to support genetic therapies for Alzheimer's disease. The grants are part of the non-profit’s Translational Gene Targeting Challenge, part of its Part the Cloud research acceleration program.

Competitive Landscape:

The gene therapy market is moderately fragmented with local players like Biogen, Novartis, Gilead Sciences, and Bluebird Bio, etc., wishing to take the time to expand their presence through investments, partnerships, acquisitions, and mergers. They also invest in product development and competitive pricing. These strategies will promote market growth and lucrative opportunities for market players.

• Company Name: Biogen
• Year of Incorporation: 1978
• Headquarters: Cambridge, Massachusetts

Biogen's annual revenue for the year ending December 31, 2024, was $9.676 billion, a 1.62% decrease compared to 2023.

• Company Name: Novartis
• Year of Incorporation: 1996
• Headquarters: Basel, Switzerland

Novartis's annual revenue for 2024 was $51.722 billion, a 10.85% increase compared to 2023. In 2023, the company's revenue was $46.66 billion, a 7.36% increase from 2022.

• Company Name: Bluebird Bio
• Year of Incorporation: 1992
• Headquarters: Massachusetts, USA

Bluebird Bio's annual revenue for 2023 was $29.5 million, primarily driven by sales of its gene therapies ZYNTEGLO and SKYSONA.