The global cell and gene therapy (CGT) pipeline looks enormous on the surface, but the real story is concentration. Thousands of programs are being developed, yet a relatively small number of regions, therapeutic areas and modalities account for a disproportionate share of activity.
As of March 2025, the global CGT pipeline contained 4,418 gene, cell and RNA therapies across clinical and preclinical development: 2,154 gene therapies, 966 non-genetically modified cell therapies and 1,298 RNA therapies.
United States and China – 67% of Global Cell-Therapy Trials
The geographic concentration is particularly clear in cell therapy. A global analysis identified 10,373 cell-therapy clinical trials through October 2025.
The United States accounted for 3,563 trials and China for 3,365, together representing almost 67% of the global total. Europe followed with 1,584 trials.
| Region | Cell-therapy clinical trials | Approx. global share |
|---|---|---|
| United States | 3,563 | 34.3% |
| China | 3,365 | 32.4% |
| Europe | 1,584 | 15.3% |
| Rest of world | 1,861 | 17.9% |
This creates an important strategic divide. The U.S. remains a major center for advanced clinical development, biotech innovation and commercialization, while China has built extraordinary trial volume and a rapidly expanding domestic CGT ecosystem.
Oncology – 56.1% of Cell-Therapy Research
The strongest concentration is not only geographic; it is therapeutic.
Oncology represented 56.1% of global cell-therapy clinical trials, making cancer by far the largest disease area for cell-based development. Immune-system diseases accounted for another 9.3%.
CAR-T remains central to this concentration, with 2,409 trials identified globally. The field is now moving beyond the first generation of CD19 and BCMA programs toward solid tumors, next-generation CAR designs, allogeneic platforms and combination approaches.
CAR-T and Stem Cells Dominate the Modality Mix
Two technology platforms account for much of the cell-therapy pipeline.
The global analysis identified 5,167 immune-cell therapy trials and 4,796 stem-cell therapy trials. Within these categories, CAR-T accounted for 2,409 trials, while mesenchymal stem cells and hematopoietic stem cells accounted for 1,904 and 1,550 respectively.
This matters for investors and technology providers because pipeline concentration translates directly into manufacturing demand, vector requirements, clinical infrastructure and specialized talent.
Gene Therapy Is Smaller Than Cell Therapy – But Strategically Different
Gene therapy represents a different opportunity profile. Of the 2,154 gene therapies in the March 2025 pipeline, 710 were already in clinical trials while 1,432 remained preclinical.
The pipeline is also diversifying beyond rare genetic disorders. Neurology, ophthalmology, hematology and oncology are becoming increasingly important, while RNA-based approaches are widening the definition of what constitutes the next generation of genetic medicines.
The Pipeline Is Still Early-Stage Heavy
The concentration story becomes even more important when development stage is considered.
ISCT’s 2025 Global Regulatory Report identified 322 active clinical trials in its global CGT pipeline, with activity concentrated in Phase I and Phase II. Genetically modified cell therapies represented 53% of clinical trials, while gene therapies represented another 25%.
That creates both opportunity and risk: the pipeline is deep, but a substantial proportion still needs to demonstrate clinical efficacy, durability, safety and scalable manufacturing.
China Is Becoming a Critical CGT Development Hub
China deserves particular attention because its CGT ecosystem is no longer simply an emerging market.
Recent research found that China has achieved substantial progress in CGT approvals and clinical development, although its approved products remain heavily concentrated in oncology and primarily serve the domestic market.
The strategic implication is clear: companies seeking global CGT opportunities need to track China not only for competitive intelligence, but also for clinical innovation, licensing opportunities and manufacturing capabilities.
Where the Real Opportunity Sits
The headline number of thousands of CGT programs can be misleading. The real pipeline is concentrated around three major geographic engines – the U.S., China and Europe, and a handful of technology and therapeutic clusters led by oncology, CAR-T, stem-cell therapies and gene-based medicines.
For pharma, biotech and investors, the next question should therefore not be “How large is the CGT pipeline?” but “Where is pipeline density converting into late-stage assets, approvals, manufacturing capacity and commercial partnerships?”
That is where the competitive landscape is likely to separate between 2026 and 2030.
For strategic planning, CGT opportunity mapping should combine pipeline concentration, Phase III assets, therapeutic-area density, modality mix, manufacturing capacity, funding, licensing activity and geographic expansion.
A region with 1,000 early-stage programs may ultimately be less commercially attractive than a region with 200 programs but significantly higher Phase II/III progression and manufacturing readiness.
The next CGT leaders will therefore not necessarily come from the regions with the most programs. They will come from the ecosystems that can convert pipeline volume into repeatable clinical, regulatory and commercial success.
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