Gene Therapy Market: Current Analysis and Forecast (2025-2033)

Gene Therapy Market Size & Forecast

The gene therapy market was valued at approximately USD 9.2 billion in 2024 and is expected to grow at a substantial CAGR of around 30.1% during the forecast period (2025-2033), owing to the growing gene therapy pipeline.

Gene Therapy Market Analysis

The gene therapy market is constantly growing, and its growth rates are constantly increasing due to the constant developments in genetic science and biotechnology that affect the treatment of diseases. There is an agreed-upon idea of gene therapy where defective genes are either repaired or replaced to offer a long-term cure to the diseases. This is being applied across oncology, rare genetic disorders, neurological, and ocular diseases, among others, due to its effectiveness.

The market is mainly driven by factors such as preparations by biopharmaceutical industries, approval of gene-regulated treatments, and patronage from state authorities. The advances in vector technologies, manufacturing techniques, and delivery systems are ever-increasing and are also enhancing the clinical uses of the vectors. Precise partnerships, operations, and research studies are steadily driving the development of gene therapy as one of the most significant fields in the contemporary world.

Gene Therapy Market Trends

This section discusses the key market trends influencing the various segments of the Gene Therapy Market as identified by our research experts.

Expanding Applications Beyond Rare Diseases

One significant trend in the structure of the gene therapy market globally is the transition from the treatment of rare or congenital diseases to the intervention in more common and multifactorial diseases. The first application of gene therapy was to treat some monogenic disorders since their targets are clear genetic markers and medical needs were unmet; however, it is now used in cancer, cardiovascular, and neurological diseases. As such, there has been progress in vector technologies, better gene editing tools, and a trial success rate in clinical research. They foresaw that this trend would contribute to the extensive growth of the patient population and the market prospects of gene therapies all over the world as science develops.

Gene Therapy Industry Segmentation

This section provides an analysis of the key trends in each segment of the global gene therapy report, along with forecasts at the global, regional, and country levels for 2025-2033.

The Viral Vector Segment Holds the Largest Share of the Gene Therapy Market.

Based on vector, the market is segmented into viral vector (retroviral and adeno-associated) and non-viral vector (oligonucleotides). The viral vector held a dominant share of the market in 2022. Lentiviral vectors, derived from the human immunodeficiency virus, have been extensively investigated and optimized over the past two decades. Third-generation, self-inactivating lentiviral vectors have recently been used in multiple clinical trials to introduce genes into hematopoietic stem cells to correct primary immunodeficiencies and hemoglobinopathies. CAR T-cell therapies engineered using lentiviral vectors have demonstrated noteworthy clinical success in patients with B-cell malignancies, leading to regulatory approval of the first genetically engineered cellular therapy using lentiviral vectors.

The Ex Vivo Segment is Expected to Witness a Higher CAGR than the Gene Therapy Market.

By delivery method, the market is bifurcated into in vivo and ex vivo. The ex vivo segment is anticipated to grow with a high CAGR during the forecast period. The expression of the gene and health of the transfected cells can be verified before it is introduced to the patient via ex vivo delivery. This method can be useful for therapies where compounds should be released or expressed by specific cell types that can be selected on the culture dish before transplant. In ex vivo, cells are modified outside the patient’s body, and the corrected version is transplanted back into the patient. The cells are treated with either a viral or nonviral gene therapy vector carrying the corrected copy of the gene.

North America has a significant share of the market in 2024.

The North American gene therapy market holds the position of the most progressive in the global market regarding innovation and growth due to the increased focus on R&D, supportive legislation, and a high level of investments in biotechnology. It so enjoys a favourable environment that agencies such as the FDA approve novel gene therapies for faster commercialization. The region has a massive number of outstanding key pharmaceutical and biotechnology firms, as well as increasing attention being paid to the treatment of such diseases as genetic disorders, cancer, and other orphan diseases. Some of them include high treatment costs and a complex regulatory environment, but the growth is steady due to the integration of gene therapy in clinical and commercial applications.

U.S. Dominates the North American Gene Therapy Market

The market share of gene therapy is growing in the U.S. about increased numbers in expenditure for R&D activities, favorable regulation, and a recent increase in FDA approvals of novel therapy for genetic and other rare or chronic conditions. Gene therapy is emerging as a new paradigm for treatment as it finds its uses in oncology, neurology, and genetic disorders, and many more. Although the market looks promising, such issues as high development costs, complicated manufacturing processes, and access to treatment can be considered as the main factors affecting the development of the market in the future.

Gene Therapy Industry Competitive Landscape  

The gene therapy market is competitive, with several global and international players. The key players are adopting different growth strategies to enhance their market presence, such as partnerships, agreements, collaborations, new product launches, geographical expansions, and mergers and acquisitions.

Top Gene Therapy Companies

Some of the major players operating in the market are Bluebird Bio (Carlyle and SK Capital), Biogen, BioMarin Pharmaceuticals, Celgene Corporation (Bristol Myers Squibb), Gilead Sciences, Novartis, Orchard Therapeutics (Kyowa Kirin company), Regenxbio, Spark Therapeutics (Roche), and uniQure.

Recent Developments in the Gene Therapy Market

• In January 2024, Biogen and Ginkgo Bioworks announced the completion of their gene therapy collaboration involving AAV-based vectors. This is expected to fuel the demand for gene therapies in the coming years.

• In December 2023, the Swiss Agency for Therapeutic Products granted approval to Libmeldy for the treatment of early-onset metachromatic leukodystrophy.

• In May 2023, Krystal Biotech was approved for VYJUVEK gene therapy for Dystrophic Epidermolysis Bullosa treatment

Gene Therapy Market Report Coverage

Reasons to Buy the Gene Therapy Market Report:

• The study includes market sizing and forecasting analysis validated by authenticated key industry experts.

• The report presents a quick review of overall industry performance at a glance.

• The report covers an in-depth analysis of prominent industry peers with a primary focus on key business financials, product portfolios, expansion strategies, and recent developments.

• Detailed examination of drivers, restraints, key trends, and opportunities prevailing in the industry.

• The study comprehensively covers the market across different segments.

• Deep dive regional-level analysis of the industry.

Customization Options:

The global gene therapy market can be customized further as per the requirements or any other market segment. Besides this, UnivDatos understands that you may have your own business needs; hence, feel free to contact us to get a report that completely suits your requirements.