According to Fortune Business Insights, the global gene therapy market was valued at USD 3.57 billion in 2025 and is projected to grow from USD 4.22 billion in 2026 to USD 20.68 billion by 2034, registering a CAGR of 21.97% during the forecast period from 2026 to 2034. North America dominated the market, accounting for a 65.83% share in 2025.
Gene therapy represents an important area of advanced medical treatment because it is designed to address the underlying genetic causes of disease. Growing demand for therapies capable of treating genetic disorders and rare diseases is contributing to the expansion of the market.
The industry is also benefiting from increased investment in research and development, product launches, regulatory approvals, and funding for innovative technologies. For example, SpliceBio received USD 127.7 million in Series B funding in June 2025 to advance gene therapy. Such investments demonstrate the increasing focus on developing innovative treatment approaches and expanding the gene therapy pipeline.
For detailed market insights: https://www.fortunebusinessinsights.com/industry-reports/gene-therapy-market-100243
Improving manufacturing scalability is one of the prominent trends shaping the gene therapy market. Growing demand for viral vectors and an expanding number of clinical candidates are encouraging companies to increase manufacturing capabilities.
Companies are integrating next-generation bioprocessing technologies, automated systems, and modular facilities to achieve higher yields and consistent product quality. In October 2024, the Cell and Gene Therapy Catapult partnered with Pharmaron Biologics (UK) Ltd and Complement Therapeutics and received a USD 1.5 million grant under the U.K. Transforming Medicines Manufacturing Programme. The initiative aimed to reduce AAV production costs and improve scalability.
Innovation in gene delivery is another important development. New delivery systems are being developed to improve tissue specificity, reduce immune activation, and simplify large-scale production. In May 2025, Dyno Therapeutics launched three new AAV capsid gene delivery vectors targeting the eye, musculoskeletal system, and central nervous system.
The increasing prevalence of genetic disorders is a major factor driving gene therapy market growth. Conditions such as spinal muscular atrophy, hemophilia, β-thalassemia, and inherited retinal diseases are increasing demand for treatments that can address the root cause of disease.
The World Federation of Hemophilia reported 218,804 patients with hemophilia in 2023, highlighting the need for effective therapeutic options. Increasing demand for curative treatments is expected to strengthen the adoption of gene therapies.
Advances in gene therapy technologies, greater research activity, funding opportunities, product launches, and regulatory approvals are also contributing to market expansion. Strong investments by biotechnology and pharmaceutical companies are helping develop new therapies and broaden the availability of treatment options.
Despite strong growth prospects, the high cost of gene therapies remains a significant barrier to patient access. The substantial financial burden affects patients, payers, and healthcare systems. For example, the first gene therapy for hemophilia B approved by the U.S. FDA in December 2022 carried a one-time treatment cost of USD 3.5 million.
Maintaining clinical efficiency over the long term is another challenge. Transgene expression may decline because of immune responses, promoter silencing, or loss of modified cells, making long-term monitoring and follow-up necessary.