Visiongain has published a new report entitled Gene Therapy R&D Market Report 2025-2035 (Including Impact of U.S. Trade Tariffs): Forecasts by Disease (Cancer, Rare Diseases (Oncologic, Non-oncologic), Cardiovascular Diseases, Ophthalmic Diseases, Haematology, Neurological, Diabetes Mellitus, Other Diseases)), Vector (Viral (Retrovirus, Adenovirus, AAV, Lentivirus, Others), Non-viral (Naked DNA, Gene Gun, Electroporation, Lipofection)), Techniques (Gene Augmentation Therapy, Gene Replacement Therapy), Participants (Small/Medium Pharma & Biotech, Universities & Research Institutes, Hospitals, Government & Public Bodies, Big Pharma) AND Regional and Leading National Market Analysis PLUS Analysis of Leading Companies.

The global Gene Therapy R&D market is estimated at US$3,696.1 million in 2025 and is projected to grow at a CAGR of 30.9% during the forecast period 2025-2035.

Regulatory Scenarios to Challenge Industry Growth

The regulatory landscape for gene therapy remains another critical barrier, characterised by complexity, fragmentation, and lack of global harmonisation. Developing, testing, and commercialising a gene therapy requires adherence to rigorous safety and quality standards, but regulatory expectations vary considerably between jurisdictions. In the United States, the FDA has adopted a more proactive stance, by issuing guidance and creating fast-track designations for high-need therapies. By contrast, in Europe, regulatory authorities have pursued a more cautious approach, aiming to avoid over-specification that might stifle innovation but creating uncertainty in the absence of standardised guidelines. In Asia, divergent policies across countries add further complexity for multinational developers.

On the manufacturing side, regulatory demands are heightened by the inherent complexity of working with biological materials. Every stage—vector design, plasmid preparation, transduction, purification, and quality control—requires cutting-edge technology and costly infrastructure. Many smaller companies struggle to meet these requirements, forcing them to rely heavily on third-party contract development and manufacturing organisations (CDMOs). The lack of standardised processes means that scaling up production from clinical to commercial stages often necessitates extensive revalidation, delaying market entry.

Communication gaps between researchers, regulators, and policymakers exacerbate the issue. Regulatory staff often lack the technical expertise to engage fully with frontier scientific debates, while scientists and developers may underestimate regulatory constraints, leading to misalignment in expectations. This disconnect slows approval cycles and increases compliance costs. While progress has been made—such as the creation of product-specific registries and adaptive approval frameworks—the absence of universally accepted standards remains a bottleneck for global growth.

How will this Report Benefit you?

Visiongain’s 357-page report provides 155 tables and 185 charts/graphs. Our new study is suitable for anyone requiring commercial, in-depth analyses for the Gene Therapy R&D market, along with detailed segment analysis in the market. Our new study will help you evaluate the overall global and regional market for Gene Therapy R&D. Get financial analysis of the overall market and different segments including type, process, upstream, downstream, and company size and capture higher market share. We believe that there are strong opportunities in this fast-growing Gene Therapy R&D market. See how to use the existing and upcoming opportunities in this market to gain revenue benefits in the near future. Moreover, the report will help you to improve your strategic decision-making, allowing you to frame growth strategies, reinforce the analysis of other market players, and maximise the productivity of the company.

What are the Current Market Drivers?

Growing Prevalence of Targeted Diseases Treatment

The rising prevalence of cancers and other chronic conditions is a major driver of demand for gene therapy. These diseases represent large patient populations with limited treatment options, creating strong incentives for investment in advanced therapies.

In the United States, cancer remains the second leading cause of death, with an estimated 618,120 deaths projected in 2025. Lung cancer alone accounts for nearly 2.5 times more deaths than colorectal and pancreatic cancers combined, with smoking remaining the dominant risk factor despite decades of prevention efforts. Breast cancer is also a significant burden, with 316,950 new invasive cases and 59,080 cases of DCIS expected in 2025. Despite progress in screening and treatment, breast cancer is still projected to cause over 42,000 deaths this year.

Gene-based approaches are reshaping oncology by enabling precise, personalised interventions. In late 2024, a DNA-based vaccine for triple-negative breast cancer demonstrated strong clinical promise, with 16 of 18 patients remaining cancer-free after three years, compared with historical survival rates of ~50%. Similarly, breakthroughs in CRISPR/Cas9 gene editing and oncolytic virotherapy (OVT) are allowing researchers to directly target oncogenes such as HER2 and MYC, addressing tumour growth and immune evasion at the genetic level.

Gene therapy is increasingly integrated into cancer care, with strategies ranging from cancer vaccines and synthetic delivery vectors to cell-based approaches such as CAR T-cell therapy, which is already FDA-approved for certain haematological cancers. These developments demonstrate how gene therapy is moving beyond experimental research to become a mainstream driver of innovation in cancer treatment.

Role of Artificial Intelligence and Machine Learning in Gene Therapy

Artificial intelligence (AI) and machine learning (ML) are increasingly being applied to accelerate gene therapy research and development. These technologies help identify viable gene-editing targets, predict potential off-target effects, and design more efficient delivery systems. By improving accuracy and reducing development timelines, AI and ML are strengthening both the safety and scalability of advanced therapies.

In practice, Deep Genomics uses AI to design RNA-based therapeutics for rare genetic diseases, directly linking computational insights to gene and RNA therapy pipelines. Exscientia applies AI-driven drug design platforms to precision medicine, including genetic-based approaches that can inform future gene therapy strategies.  

Together, these initiatives highlight how AI is moving from a supportive tool to a core enabler of innovation in gene therapy, particularly in rare and genetic diseases where speed and precision are critical.

Where are the Market Opportunities?

Facility Expansion Anticipated to Offer Lucrative Growth Prospects

The surge in clinical-stage start-ups has created a shortage of viral vector manufacturing capacity among contract manufacturers, on which many new gene and cell therapy companies depend for early development. As these firms progress towards commercialisation, they increasingly seek full control of manufacturing to avoid outsourcing risks. This has prompted the formation of internal teams, engagement with site consultants, and searches for suitable lab space or, in competitive real estate markets, entirely new construction projects.

Building in-house facilities enables rapid scale-up from clinical to commercial batches, smooth technology transfer, and co-location with R&D operations. However, the urgency of “time-to-market” has intensified demand for existing buildings in established biotech hubs, where access to specialised talent, academic partnerships, and peer networks create competitive clusters. Cost sensitivity remains a key factor, particularly for venture-funded businesses keen to minimise both upfront and recurring expenses.

Recent developments illustrate the scale of investment in facility expansion. On 5 August 2025, Sumitomo Chemical completed its third regenerative medicine and cell therapy plant (CRAFT) through subsidiary S-RACMO, with a fourth planned by 2028. On 10 June 2025, Cellex Cell Professionals expanded its GMP facilities in Cologne, Germany, strengthening its CDMO leadership in CGT manufacturing. On 4 June 2025, Cellares and Mitsui Fudosan announced plans for a “smart” automated cell therapy facility in Chiba Prefecture, Japan, scheduled to open in 2026 with capacity for 350 staff. On 20 March 2025, Bharat Biotech inaugurated India’s first integrated CGT facility at Genome Valley, Hyderabad, featuring a viral vector production unit for blood disorders and malignancies. Finally, on 12 March 2025, Johnson & Johnson and Legend Biotech committed an additional US$150 million to expand their Ghent, Belgium, site, aiming to double Carvykti (ciltacabtagene autoleucel) output, with commercial operations slated for 2028.

Expansion of Clinical Trials and CAR-T Therapies

The continued expansion of clinical trials in gene and cell therapy represents one of the strongest market opportunities. Growing numbers of candidates are advancing through preclinical and clinical phases, signalling rising confidence in safety, efficacy, and commercial potential. This expanding pipeline strengthens the evidence base, encourages regulatory dialogue, and attracts sustained investment, offering companies opportunities to secure partnerships and accelerate time-to-market.

Within this broader trend, CAR-T cell therapies stand out as a particularly significant opportunity. CAR-T currently represents around 32% of the total CGT pipeline, reflecting the transformative efficacy these therapies have demonstrated in refractory haematological malignancies. Oncology remains the dominant area, accounting for approximately 45% of all marketed and pipeline CGTs. At the same time, diversification of CAR-T research into CNS and cardiovascular indications is broadening the therapeutic scope, highlighting the multi-dimensional opportunity landscape for developers and investors.

Together, the rising number of clinical trials and the prominence of CAR-T therapies underscore the industry’s commitment to advancing treatment options, reshaping standards of care, and capitalising on the strong growth trajectory of the global gene therapy R&D market.

Competitive Landscape

The major players operating in the Gene Therapy R&D market are American Gene Technologies, Applied Genetic, Astellas Pharma Inc., Bayer, Benitec BioPharma, Biogen, Bluebird Bio, Bristol Myers Squibb, Calimmune, Inc. (CSL Behiring), Cellectis, GenSight Biologics, Gilead Lifesciences, Inc., GQ Bio Therapeutics GmbH (Pacira Pharmaceuticals, Inc.), Novartis AG, OCUGEN, INC., Orchard Therapeutics, Oxford Biomedica, Pfizer, Inc., REGENXBIO Inc., Sangamo Therapeutics, Inc., Sanofi, Sarepta Therapeutics, Inc., Spark Therapeutics (Subsidiary of Roche), Takeda Pharmaceutical Company Limited, Taysha GTx, Transgene, UniQure N. V., VeonGen Therapeutics, and Voyager Therapeutics Inc. These major players operating in this market have adopted various strategies comprising M&A, investment in R&D, collaborations, partnerships, regional business expansion, and new product launch.

Recent Developments

• On 16^th June 2025, Ocugen, Inc. announced that the FDA had cleared its IND amendment for OCU410ST, a gene therapy candidate for Stargardt disease, a rare inherited eye disorder causing progressive vision loss. The therapy, based on Ocugen’s modifier gene platform and delivered via an AAV vector, introduces a functional copy of the RORA gene into retinal cells. With this clearance, Ocugen will launch the Phase 3 GARDian trial to assess its safety and efficacy.
• On 5^th June 2025, VeonGen Therapeutics received Rare Pediatric Disease Designation (RPDD) from the FDA for its lead gene therapy VG801, targeting ABCA4 mutation-related retinal dystrophy (Stargardt disease). VG801 is a dual AAV-based therapy that leverages VeonGen’s proprietary vgAAV capsid and vgRNA REVeRT (mRNA trans-splicing) technology to deliver the full-length ABCA4 gene, overcoming traditional AAV size constraints.
• On 23^rd April 2025, Ray Therapeutics, received an US$8 million grant from the California Institute for Regenerative Medicine (CIRM) to advance its gene therapy candidate, RTx-015, for treating retinitis pigmentosa. The funding will support a pioneering Phase‚ÄØ1 clinical trial designed to evaluate the safety and preliminary efficacy of RTx-015, delivered via subretinal injection to patients with genetically confirmed forms of the disease. RTx-015 leverages an adeno-associated virus (AAV) vector to deliver a functional copy of a gene that can slow or halt retinal degeneration.
• On 8^th January 2025, Columbia University scientists led by Raul Rabadan, PhD, unveiled a powerful AI model that can accurately predict gene expression across any human cell type by “learning the grammar” of gene regulation. Trained on data from over 1.3 million cells, the model forecasts gene.

Notes for Editors

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