Gene Therapy Market Size & Share Analysis - Key Trends, Future Opportunities, Growth Strategies, and Forecasts (2025 - 2032)

Gene Therapy Market Emerging Trends & Growth Drivers

Next-Gen Therapies and Global Approvals Reshaping the Gene Therapy Landscape Is the Key Trend

• Gene therapy products are gaining a rising number of regulatory approvals throughout global markets, which improves market commercialization while expanding patient access worldwide, including potential future approvals.
• In vivo gene therapy approach represents a rising market trend because researchers direct therapeutic genes to patients in-body cells which demonstrates benefits compared to ex vivo procedures through scalability and patient convenience.
• Scientists have discovered major breakthroughs by creating safer and more efficient viral vectors like adeno-associated viruses, AAVs, which improve both the delivery efficacy and safety of gene treatments.
• The research and development in non-viral delivery systems such as lipid nanoparticles and CRISPR-Cas9 delivery systems to address limitations related with the viral vector, such as immunogenicity and manufacturing complexities.
• Scientists are working hard to create allogeneic gene therapies as manufactured off-the-shelf tools for multiple patients because these treatments could be both more available and less expensive than autologous therapies.

Rising Demand and Funding Fueling Gene Therapy Expansion Are the Major Growth Driver

• The large genetic disorder population along with the extensive cancer cases worldwide along with inherited disorders in form an urgent medical requirement that gene therapies attempt to solve.
• With an increase in public and private funding, which includes venture capital and pharmaceutical investments drives innovation and clinical development pipeline for gene therapies.
• The market demonstrates optimistic potential since the number of gene therapy candidates in clinical development continues to expand through different stages.
• The approval process for innovative therapies will gain speed because countries globally are developing government initiatives and modern regulatory frameworks.
• Market adoption of gene therapy will grow because healthcare professionals and patients gain both knowledge and understanding about gene therapy's curative qualities. The acceptance rate for gene therapy treatment is gradually becoming more widespread across the globe.

Gene Therapy Market Segmentation Analysis

Type Analysis

The in-vivo category held the larger share, over 70%, in 2024. This is because this method eliminates the requirement for removing the cells from the patient’s body, manipulating them outside the body, and then returning them to their original place. Moreover, in vivo gene therapy is useful to modify the genetic repertoire of target cells for therapeutic goals. With in-vivo gene therapy, new genetic material is created in a lab and inserted into your cells directly. A vector usually carries the new genetic material into the body through an injection in the blood, or directly to the target organ.

This therapy is particularly effective for systematic conditions, such as cancer and genetic disorders since these methods facilitate thorough cell distribution and rapid cell uptake. The widespread use of gene therapy in different medical applications globally has been made possible through improvements in vector design particularly through the adoption of adeno-associated viruses (AAVs).

The type categories covered in this report are:

• In Vivo (Larger Category)
• Ex Vivo (Faster-Growing Category)

Vector Type Analysis

The viral category held the larger share, over 75%, in 2024. This is because viral vectors are exhibiting high levels of genomic expression and incorporation in clinical trials and high efficiency in gene delivery and widespread use in approved therapies. Moreover, viral vectors are more efficient than non-viral variants and widely being adopted all over the world.

The popularity of viral vectors essentially stems from the fact that they can deliver genetic material to cells effectively. Due to their incredible capacity to transport several copies of therapeutic genes to host cells, viral vectors are the most often used agents in gene therapy, which is expected to fuel the market growth in coming years.

The vector types analyzed here are:

• Viral (Larger Category)
  • Adeno-Associated Virus (AAV)
  • Adenovirus
  • Lentivirus
  • Retrovirus
• Non-Viral (Faster-Growing Category)

Application Analysis

The oncology category held the largest share, over 20%, in 2024, due to the increasing prevalence of various cancers worldwide and the growing adoption of gene therapies as targeted treatment options. Oncology gene therapy modifies genes through replacement operations with the goal to inhibit tumor development while improving immune responses and allowing medication delivery to cancer cell targets. The advent of CAR-T cell therapy produces transformative impacts on cancer therapy especially when treating patients with leukemia and lymphoma.

Significant research investments along with market-ready cancer gene treatments have made this market segment dominant. For instance, as per the WHO report, it is predicting that more than 9 million people die from cancer each year. Moreover, given that gene therapy is very selective in destroying tumor cells, over 60% of the clinical studies that are now being conducted in the field are for the treatment of cancer.

Moreover, several gene therapy techniques have been applied to the treatment of cancer, such as oncolytic virotherapy, immune modulation based on gene therapy, anti-angiogenic gene therapy, treatment of gene defects, tumor invasion pathways, RNAi strategies, and genetic manipulation of apoptotic and antisense. Furthermore, lung, brain, pancreatic, liver, prostate, colorectal, bladder, ovarian, and renal cancer are among the cancer types that have been treated by gene therapy.

Here are the applications studied in the report:

• Oncology (Largest Category)
• Rare Diseases (Fastest-Growing Category)
• Neurology
• Ophthalmology
• Hematology
• Infectious Diseases
• Cardiology

End User Analysis

The pharmaceutical and biotechnology category held the larger share, over 50%, in 2024. This is due to the existence of multiple industry participants who contracted out the vector production process to broaden the use of gene therapy and enable it to treat many more ailments than it now is.

Furthermore, key companies are exploring a range of market entry strategies, including the growth of biopharmaceutical facilities, partnerships with other significant companies, and partnerships with contract manufacturing organizations.

End users analyzed in the report:

• Pharmaceutical and Biotechnology Companies (Larger Category)
• Academic Institutes and Research Centers (Faster-Growing Category)

Gene Therapy Market Regional Market Performance

The North America held the largest share, of around 55%, in the market in 2024, and it is expected to sustain its dominance over the next few years. This is ascribed to the increasing prevalence of cancer and the presence of several leading industry players in the region. In addition, regulatory authorities are rapidly approving gene therapy products, thereby fueling the expansion of the market. Within the region, the U.S. is the larger user of gene therapy because of its advanced healthcare sector and most of the market players have offices and laboratories here.

The Asia-Pacific will grow at the highest CAGR, of 17.5%, during the forecast period, throughout the projection period due to the low labor costs and the emergence of group investors. In addition, regional nations are putting rules into effect to control the procedures for developing and approving gene therapy items. The Asia Pacific region is expected to have significant growth in the commercial usage of genetic therapies throughout the projection period because of the easy access to resources, the presence of large firms locally, and the increase in government financing across the region.

On the other hand, Europe is expected to hold significant growth in the coming years. The presence of contemporary healthcare infrastructure, reimbursement availability, and the presence of important industry players are all essential factors driving the growth of the European region.

The regions analyzed here are:

• North America (Largest Category)
  • U.S.
  • Canada
• Europe
  • U.K.
  • Germany
  • France
  • Italy
  • Spain
• Asia-Pacific (Fastest-Growing Category)
  • China
  • Japan
  • Australia
  • India
  • South Korea
• Latin America
  • Brazil
  • Mexico
• Middle East & Africa
  • U.A.E.
  • Saudi Arabia
  • South Africa

Gene Therapy Market Share Analysis

The gene therapy market is fragmented in nature because with the presence of numerous biotechnology startups, mid-sized firms, and large pharmaceutical companies that are actively involved in developing gene therapy products across a variety of therapeutic fields. Numerous market participants choose to operate in special disease areas or platform delivery technology development.

The market shows a growing consolidation of power among major companies because more gene therapies achieve regulatory acceptance and enter commercial operations resulting in heightened strategic partnerships and mergers. The upcoming years will witness this market trend strengthening further as corporations work on growing their product lines together with production facilities and worldwide market penetration.

Gene Therapy Market Developments

• In November 2024, Novartis purchased gene therapy startup Kate Therapeutics for up to $1.1 billion.
• In May 2023, Krystal Biotech announced a private placement equity financing of approximately $160 million. The funds were intended to support the launch of VYJUVEK and advance the company's pipeline through the end of 2026.