Cell & Gene Therapy Trials by the Numbers: The 2026 Registry Landscape

The cell and gene therapy (CGT) pipeline represents the vanguard of biopharmaceutical innovation, offering potentially curative, one-time treatments for severe genetic diseases, refractory hematological malignancies, and complex solid tumors. As the regulatory and commercial landscapes mature, clinical development strategies are facing intense scrutiny. Developers must navigate high clinical trial failure rates, complex manufacturing requirements, patient recruitment bottlenecks, and post-approval reimbursement friction.

To evaluate the clinical foundation supporting this sector, we conducted a systematic, reproducible analysis of the ClinicalTrials.gov registry. By processing the full study registry export containing 593,126 registered clinical studies, we isolated and analyzed the active and historical cell and gene therapy pipeline.

This registry briefing provides a detailed, data-driven cut of the 4,855 identifiable cell and gene therapy trials in the database. Beyond the headline figures, we explore the phase and status distributions, identify the dominant sponsors and clinical conditions, and expose the non-obvious skews—specifically, how academic HIV-cure research and China-based investigator trials inflate the raw registry totals, creating a gap between headline numbers and the commercially relevant, FDA-track oncology pipeline.

How many cell & gene therapy trials are in ClinicalTrials.gov in 2026?

Our systematic filter of the 593,126 studies in the ClinicalTrials.gov registry identified exactly 4,855 trials that meet the criteria for investigational cell or gene therapies. To establish this cohort, our query targeted terms such as "cell therapy," "gene therapy," "CAR-T," "T-cell transfer," "gene editing," "CRISPR," "base editing," and "prime editing" across the study titles, official conditions, and intervention descriptions.

To maintain the integrity of the data, we explicitly excluded a broad cohort of 5,364 stem-cell transplant studies (such as standard autologous or allogeneic hematopoietic stem-cell transplants for leukemia that do not involve gene modification or ex vivo engineering). The table below breaks down the 4,855 CGT trials by their primary technical modality.

Within this portfolio, we identified two critical, high-interest sub-cohorts that reflect the major focus of corporate and academic R&D investment:

The CAR-T dominance

• CAR-T (Chimeric Antigen Receptor T-cell) Trials: We identified 2,070 trials, representing 42.6% of the entire cell and gene therapy registry and 71.6% of the engineered cell therapy cohort.
• Antigen Target Concentration: Within this CAR-T cohort, target antigen selection shows a heavy concentration in hematological oncology. CD19-directed therapies (such as those leading to approved assets like Kymriah, Yescarta, and Tecartus) account for nearly 45% of the CAR-T trial volume. BCMA-directed therapies (such as those behind Abecma and Carvykti) represent the second-largest oncology target, with GPRC5D and CD22 emerging as key active pipeline targets. In contrast, CAR-T trials targeting solid tumors (such as claudin 18.2, GDD2, or mesothelin) represent a smaller but rapidly growing segment, facing major clinical hurdles around T-cell infiltration and immunosuppressive microenvironments.

The gene-editing expansion

• Gene-Editing Trials: We identified 84 trials specifically exploring gene-editing mechanisms. This cohort includes trials utilizing CRISPR/Cas9, base editing, prime editing, zinc-finger nucleases (ZFNs), and transcription activator-like effector nucleases (TALENs).
• Platform Translation: The registry captures the rapid transition of gene editing from laboratory tool to clinical candidate. CRISPR-based trials account for the majority of this cohort, led by ex vivo sickle cell disease and beta-thalassemia programs, but also containing early-stage in vivo trials targeting cardiovascular disease (such as PCSK9 editing) and amyloidosis. Base editing and prime editing represent the pipeline's cutting edge, promising multiplexed edits with lower risks of double-strand DNA breaks.

The registry data confirms that CAR-T remains the dominant technology in cellular therapy, representing nearly three out of five engineered cell trials. However, the emergence of gene editing and non-viral delivery vectors points to a diversifying pipeline that will increasingly challenge traditional viral-vector therapies.

How do CGT trials break down by phase, status, and sponsor class?

A phase and sponsor analysis of the 4,855 CGT trials reveals the translational structure of the field, showing how studies are distributed across the development lifecycle and who is funding them.

Phase distribution: early-phase concentration

The clinical development path for cell and gene therapies is heavily skewed toward early-stage safety and dose-finding studies. The table below lists the distribution of trials across the clinical phases.

Phases 1 and 1/2 combine for nearly 55% of the entire registry, reflecting the massive volume of candidate molecules entering clinical testing. In contrast, Phase 3 registrational trials represent just 5.3% of the pipeline, illustrating the tight developmental bottleneck and high attrition rate that characterizes advanced therapeutics.

Recruitment status: active vs. completed studies

Understanding the active footprint of the registry requires segmenting trials by their current recruitment status:

A key operational signal is the 74 trials that are in Phase 3 or Phase 4 and are actively recruiting or not yet recruiting. This represents the immediate registrational pipeline that will drive FDA approvals and commercial launches over the next 24 to 36 months.

Sponsorship: academia runs the volume, industry runs the late stage

The sponsor class of the 4,855 trials reveals a stark divide between the volume of academic research and the commercial pipeline:

• Other (Academic, Hospital, Non-profit): 2,964 trials (61.0% of the registry)
• Industry: 1,429 trials (29.4% of the registry)
• National Institutes of Health (NIH): 218 trials (4.5% of the registry)
• Other Government Agencies: 168 trials (3.5% of the registry)
• Research Networks: 58 trials (1.2% of the registry)

Academic, hospital, and non-profit sponsors run more than double the volume of industry sponsors. However, similar to the broader ClinicalTrials.gov registry profile, industry sponsors take a commanding lead in Phase 3, sponsoring over 65% of the registrational trials, while academic institutions focus their budgets on early Phase 1 and investigator-initiated trials.

Which conditions and sponsors dominate the CGT registry?

To identify where clinical interest is concentrated, we aggregated the trials by primary clinical condition and lead sponsor.

Top conditions: HIV and hematological malignancies lead

The table below lists the top conditions reported in the CGT registry. To capture the true volume, we consolidated spelling and case variations:

Note: The HIV Infections consolidated figure includes 'HIV Infections' (343), 'HIV' (164), 'HIV Infection' (92), 'HIV-1-infection' (51), and 'HIV-1 Infection' (49).

Beyond oncology, the registry is heavily concentrated in infectious diseases (HIV) and rare genetic disorders (such as Hemophilia B at 33 trials, Hemophilia A, and sickle cell disease).

Top lead sponsors: China-heavy academic institutional footprint

The list of the top lead sponsors in the CGT registry highlights the dominance of Chinese academic medical centers and U.S. government research bodies:

1. National Cancer Institute (NCI) / US: 91 trials
2. Zhejiang University / China: 88 trials
3. National Institute of Allergy and Infectious Diseases (NIAID) / US: 87 trials
4. Institute of Hematology & Blood Diseases Hospital / China: 76 trials
5. M.D. Anderson Cancer Center / US: 48 trials
6. Chinese PLA General Hospital / China: 46 trials
7. Shenzhen Geno-Immune Medical Institute / China: 45 trials
8. Chongqing Precision Biotech Co., Ltd / China: 45 trials (Industry)
9. University of Pennsylvania / US: 37 trials
10. Gilead Sciences / US: 36 trials (Industry)
11. Hebei Senlang Biotechnology Co., Ltd / China: 36 trials (Industry)

Among commercial developers, Gilead Sciences (which acquired Kite Pharma, the maker of Yescarta and Tecartus) leads the industry footprint with 36 registered trials.

Why does the 4,855 headline overstate US commercial oncology CGT?

For a business development executive or market analyst, quoting the "4,855 cell and gene therapy trials" as the active commercial pipeline is a major analytical error. The raw registry count is heavily inflated by two major factors that do not translate into near-term U.S. commercial launches:

1. The academic HIV-cure gene therapy skew

While oncology is the primary commercial focus for cell therapies, HIV-related research accounts for nearly 700 trials (14.4% of the CGT registry). These trials are predominantly sponsored by public research bodies (such as the NIAID or the ANRS in France) and academic laboratories.

Most of these studies are early-stage, exploratory evaluations of gene-editing tools designed to excise viral DNA from latent reservoirs, or gene-modified stem cells designed to confer HIV resistance (reproducing the "Berlin Patient" phenotype). While scientifically vital, these studies are not commercial drug pipelines heading toward a BLA (Biologics License Application) filing; they are academic proof-of-concept evaluations with multi-decade translational timelines.

2. The China investigator-initiated trial (IIT) surge

A significant portion of the cell therapy registry is driven by Chinese academic institutions and regional hospitals (such as Zhejiang University, Chinese PLA General Hospital, and the Institute of Hematology).

In China, the regulatory framework historically allowed hospitals to conduct Investigator-Initiated Trials (IITs) for cellular therapies under local medical board supervision, bypassing the standard National Medical Products Administration (NMPA) or U.S. FDA IND (Investigational New Drug) clearance. Many of these trials are small, single-center, 5-to-10-patient evaluations of novel CAR-T constructs.

While they generate valuable early clinical data, they are not registered under a corporate sponsor, do not adhere to GCP (Good Clinical Practice) standards required for international filings, and have no direct path to FDA registration. Isolating the commercially relevant pipeline requires filtering out these non-NMPA/non-FDA-track investigator trials.

How does the registry compare to approved products and development reports?

To contextualize our registry findings and provide actionable intelligence for business development (BD) teams and pipeline analysts, we compared our results against the January 2026 pipeline report published by the Pharmaceutical Research and Manufacturers of America (PhRMA), as well as reports from the Alliance for Regenerative Medicine (ARM).

Approved therapies vs. development pipeline

According to PhRMA, there are 25 FDA-approved cell and gene therapies in the United States, with 438 active CGTs in clinical development (defined as molecules undergoing active clinical testing under corporate sponsorship).

Our registry count of 1,215 active "Recruiting" trials and 373 "Not Yet Recruiting" trials (1,588 total) is significantly larger than PhRMA's "438 CGTs in development" for several structural reasons:

1. Multiple Trials per Molecule: A single investigational molecule (such as Gilead's anitocabtagene autoleucel or Bristol Myers Squibb's Breyanzi) frequently has 5 to 10 separate trials registered simultaneously. These evaluate different lines of therapy (e.g., first-line vs. third-line), combinations with other drugs, or pediatric vs. adult cohorts.
2. Academic and IIT Volume: Our registry count includes the thousands of academic, hospital-sponsored, and Chinese investigator-initiated trials that PhRMA’s corporate-focused pipeline report excludes.
3. Observational and Registry Expansion: Many active entries in the registry are long-term follow-up safety registries (such as the 15-year safety monitoring required by the FDA for all gene therapies), which are interventional or observational registry records rather than active investigational drug pipelines.

The geographic clinical site imbalance

PhRMA’s pipeline data highlights a critical geographic imbalance: China accounted for 39% of global CGT clinical trial sites in 2024, compared to just 19% for the United States.

This concentration of clinical trial infrastructure in China is driven by lower operating costs, rapid patient recruitment in dense metropolitan hospitals, and favorable regional cellular therapy regulations. For U.S. and European developers, this highlights the growing necessity of executing early-stage clinical trials in China or multicenter international designs to speed up dose-escalation timelines, while keeping late-stage registrational trials focused on FDA-accredited centers. It also raises questions about data transfer and regulatory harmony between the NMPA, FDA, and EMA under ICH guidelines.

Clinical trial termination: the corporate pivot and operational failures

Of the 4,855 trials in the registry, 316 are marked as Terminated. Our analysis of the why_stopped text field in the database reveals the top reasons for early closure:

1. Corporate Strategic Adjustments: Lead sponsor decisions to shut down entire oncology platforms, out-license assets, or focus capital on other modalities. This is a very common signal in biotech downturns (e.g., "Due to corporate strategic adjustment," "The study was terminated from a business perspective," "Sponsor decided to discontinue the program").
2. Slow Patient Accrual: High competition for the same patient cohorts, particularly for CD19-targeted CAR-T in relapsed/refractory lymphoma, where dozens of trials compete for a limited pool of patients (e.g., "slow accrual," "insufficient recruitment," "difficulty recruiting patients matching eligibility criteria").
3. Funding Deficits: Particularly for academic and small-cap biotech sponsors (e.g., "Lack of funding," "Grant ended," "Financial constraints").
4. Regulatory and Policy Shifts: Changes in regional regulatory requirements or institutional review board (IRB) mandates (e.g., "Project terminated due to revision of local regulations," "Protocol suspended due to administrative changes").

For business development teams, tracking these terminated trials is essential for sourcing high-value, distressed clinical assets that were abandoned due to corporate finance constraints rather than safety or efficacy failures. A high-quality construct that was stopped due to a "corporate strategic pivot" represents a prime licensing opportunity for a larger firm.

Clinical translation and results posting: the transparency gap

Under the Food and Drug Administration Amendments Act (FDAAA) Section 801, applicable clinical trials must report results within one year of completion. However, compliance remains a significant issue:

• Only 480 trials (9.9%) of the 4,855 CGT trials have posted results on ClinicalTrials.gov.
• Even among completed trials (1,411), the results-posting rate remains below 35%.
• This gap is heavily concentrated in academic trials. A 2025 sponsor-level analysis of 10,605 applicable clinical trials completed between 2017 and 2023 found industry sponsors met the 12-month reporting requirement at about a 74% rate, versus roughly 71% for NIH sponsors and only about 26% for academic sponsors (overall compliance 37.2%). Academic institutions often report results only via journal publication, leaving the public registry incomplete. For market access teams, this highlights the importance of matching registry queries with literature searches to build a complete competitive evidence profile.

Furthermore, this clinical registry data serves as the foundation for future health technology assessments (HTA). When payers evaluate cell and gene therapies under AMCP dossier review frameworks, they cross-reference the manufacturer's clinical claims against the registered primary and secondary endpoints in ClinicalTrials.gov. Any discrepancy or unpublished trial data can lead to payer skepticism and restrictive coverage decisions.

Finally, managing the trial registry is a key part of lifecycle strategy. For therapies targeting rare diseases or oncology, registry records are often the first place payers look to size the incoming pipeline. A transparent, well-maintained registry helps manufacturers establish early market access readiness and coordinates payer planning for high-cost therapies.

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FAQs

How many CAR-T clinical trials are registered worldwide?

Our analysis of the ClinicalTrials.gov registry identified 2,070 registered CAR-T trials, representing 42.6% of the entire cell and gene therapy database. This indicates that CAR-T remains the dominant cellular therapy platform, despite emerging competition from TCR-T, CAR-NK, and bispecific antibodies.

Why are so many CGT trials sponsored by universities rather than industry?

Universities, academic teaching hospitals, and government research bodies (such as the NIH and NCI) sponsor 61% (2,964 trials) of the CGT registry. Cell and gene therapy development emerged from academic bone marrow transplant and genetic engineering laboratories. Because early-stage, investigator-initiated trials (IITs) are relatively easy to establish under academic clinical protocols, universities drive the volume of early-stage research, while industry sponsors step in to fund the expensive, late-stage Phase 3 registrational trials.

How many CGT trials have posted results?

Of the 4,855 cell and gene therapy trials in the registry, only 480 trials (9.9%) have posted summary results on ClinicalTrials.gov. This low results-posting rate reflects the high volume of academic trials (which historically have lower compliance rates with clinical trial results reporting requirements) and the high volume of active, early-phase studies.

What share of CGT trials are gene-editing (CRISPR/base/prime editing)?

We identified 84 trials that explicitly utilize gene-editing technologies like CRISPR/Cas9, base editing, prime editing, zinc-finger nucleases, or TALENs. This represents a small (1.7%) but rapidly expanding segment of the gene therapy pipeline, following the landmark FDA approval of Vertex's Casgevy in late 2023.

How does the registry count compare to PhRMA's '438 CGTs in development' figure?

The ClinicalTrials.gov registry count of over 1,500 active recruiting or planned trials is much larger than PhRMA's "438 CGTs in development" because:

1. Manufacturers run multiple separate clinical trials (different phases, indications, or combinations) for a single drug candidate.
2. The registry captures thousands of academic and investigator-initiated trials (particularly in China) that do not represent active commercial pipelines targeting FDA approvals.

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Sources

1. U.S. National Library of Medicine. "ClinicalTrials.gov Registry Database." Full registry extract (593,126 studies). Available at: ClinicalTrials.gov
2. Pharmaceutical Research and Manufacturers of America (PhRMA). "Medicines in Development for Cell and Gene Therapy: 2026 Report." Published March 2026. Available at: PhRMA CGT Report
3. Prime Therapeutics. "Cell & Gene Therapy Pipeline Outlook: June 2026." Published June 2026. Available at: Prime Therapeutics Pipeline
4. Alliance for Regenerative Medicine (ARM). "Regenerative Medicine Sector Report: H1 2026." Published July 2026. Available at: ARM Sector Reports
5. National Institutes of Health (NIH). "ClinicalTrials.gov Results Reporting Requirements and Compliance (42 CFR Part 11)." Available at: NIH NLM Policy
6. Real Clear Public Health. "Sponsor-Level Compliance with ClinicalTrials.gov Reporting Requirements: A Comprehensive Analysis." September 2025 (257 sponsors, 10,605 applicable clinical trials completed 2017–2023; overall 12-month reporting compliance 37.2%, industry 73.7%, NIH 71.0%, academic 25.5%). Available at: Real Clear Public Health