U.S. Cell and Gene Therapy Clinical Trials Market Expanding with Biotech Demand

Revenue, 2025

USD 5.41 Billion

Forecast, 2035

USD 23.14 Billion

CAGR, 2026-2035

15.64%

Report Coverage

United States

U.S. Cell and Gene Therapy Clinical Trials Market Size, Key Players with Insights and Growth

The U.S. cell and gene therapy clinical trials market size was estimated at USD 5.41 billion in 2025 and is predicted to increase from USD 6.26 billion in 2026 to approximately USD 23.14 billion by 2035, expanding at a CAGR of 15.64% from 2026 to 2035.

The market is experiencing strong growth due to rising investment in advanced biologics increasing prevalence of cancer and rare genetic disorders, and an active biotechnology ecosystem. Supportive FDA regulatory pathways, growing academic–industry collaborations, and advances in viral vectors and gene-editing technologies continue to accelerate clinical trial activity across the country.

Key Takeaways

• The U.S. cell and gene therapy clinical trials market will likely exceed USD 6.26 billion by 2026.
• The global cell and gene therapy clinical trials market is projected to grow to USD 45.31 billion by 2034, at a CAGR of 15.43%.
• Valuation is projected to hit USD 23.14 billion by 2035.
• Estimated to grow at a CAGR of 15.64% starting from 2026 to 2035.
• By phase, the phase III segment held the largest market share in 2025.
• By phase, the phase I segment is expected to grow at the fastest CAGR in the market during the forecast period.
• By indication, the oncology segment led the market in 2025.
• By indication, the cardiology segment is expected to grow at the fastest CAGR in the market during the forecast period.

Quick Facts Table

Driving the Future of Medicine: Rapid Growth of U.S. Cell and Gene Therapy Clinical Trials

U.S. cell and gene therapy clinical trials are FDA-regulated studies conducted in the United States to test the safety and effectiveness of therapies that use genes or living cells to treat or prevent diseases. The U.S. cell and gene therapy clinical trials market is growing due to rising demand for treatments targeting cancer and rare diseases, strong biotech innovation, increased R&D investment, supportive FDA regulatory frameworks, and advancements in gene-editing and cell-based technologies that accelerate trial development and commercialization.

How AI Can Revolutionize the Market?

AI can revolutionize the U.S. cell and gene therapy clinical trials market by accelerating patient recruitment, optimizing trial design, and predicting treatment responses through advanced data analytics. Machine learning enables faster biomarker identification, real-time monitoring of patient safety, and improved trial efficiency. These capabilities reduce development timelines, lower costs, and increase the success rate of complex cell and gene therapy trials.

Trend and Future Outlook of the U.S. Cell and Gene Therapy Clinical Trials Market

• Expansion of Rare Disease Trials: Increasing focus on rare and genetic disorders will drive new clinical programs. This trend offers personalized therapies, expanded patient access, and potential breakthroughs for previously untreatable conditions, supported by advanced genomic technologies and precision medicine approaches.
• Integration of Digital & AI Technologies: Adoption of AI-powered tools and digital data platforms will streamline trial design, patient matching, and real-time monitoring. This offers faster decision-making, higher efficiency, reduced costs, and improved safety oversight throughout the clinical development lifecycle.
• Regulatory & Collaborative Innovation: Enhanced FDA support, expedited pathways, and public-private partnerships will fuel innovation and trial approvals. This offers accelerated development timelines, shared resources among stakeholders, and broader commercialization opportunities for cutting-edge cell and gene therapies.

Major Investments in the U.S. Cell and Gene Therapy Clinical Trials Market

• In June 2025, Big Pharma is actively investing via acquisitions to strengthen trial portfolios. Eli Lilly agreed to acquire Verve Therapeutics for up to $1.3 B in 2025, enhancing its gene-editing pipeline.
• In September 2024, ArsenalBio raised $325 M, Capstan Therapeutics secured $175 M, and Obsidian Therapeutics closed a $160.5 M round to support mid- to late-stage clinical development.

Segmental Insights

By Phase Insights

Why did the phase III Segment Dominate in the Market in 2025?

The phase III segment held the largest U.S. cell and gene therapy clinical trials market share because these trials are essential for validating safety, efficacy, and optimal dosing before regulatory approval. They involve large patient populations, extended study durations, and complex trial design, leading to significantly higher costs and resource requirements. Additionally, the increasing number of cell and gene therapy candidates progressing into late-stage development and nearing commercialization further strengthens the dominance of phase III trials.

For instance,

• In August 2025, Opus Genetics secured FDA clearance to launch a Phase 1/2 clinical trial for OPGx-BEST1, a gene therapy targeting Best disease and other retinal conditions caused by BEST1 mutations. The dose-escalation study, known as BIRD-1, is currently underway at two U.S. sites with initial efficacy data expected in early 2026. Complete enrollment details and study objectives are available via ClinicalTrials.gov.

Phase I

The phase I segment is expected to grow at the fastest CAGR during the forecast period due to the rising number of early-stage cell and gene therapy candidates entering clinical development. Increased biotech startup activity, strong venture funding, and advancements in gene editing and cell engineering are accelerating first-in-human studies. Additionally, growing research focus on novel indications and rare diseases is driving demand for early-phase safety and dosing trials.

By Indication Insights

How Does the Oncology Segment Dominate the Market in 2025?

The oncology led the U.S. cell and gene therapy clinical trials market due to the high prevalence of cancer and strong demand for targeted, long-lasting treatments. Advances in CAR-T cell therapies, gene-editing technologies, and tumor-specific biomarkers have driven extensive clinical research. Additionally, significant funding, robust clinical pipelines, and expedited FDA pathways for cancer therapies continue to support the dominance of oncology-focused trials. The National Cancer Institute (NCI) estimates that in 2025, the U.S. will see around 2.04 million new cancer cases and approximately 618,000 cancer-related deaths. 

In September 2024, Poseida Therapeutics received the FDA’s RMAT designation for P-BCMA-ALLO1, an allogeneic CAR-T therapy using stem cell memory T cells for relapsed/refractory multiple myeloma. The therapy also earned Orphan Drug Designation in March 2024.

Cardiology

The cardiology segment is expected to grow at the fastest CAGR during the forecast period due to the rising burden of cardiovascular diseases and the limited effectiveness of conventional treatments for advanced heart conditions. Growing research into gene therapies for inherited cardiac disorders and regenerative cell therapies for heart failure is accelerating clinical trial activity. Additionally, increasing investment, technological advances in cardiac gene delivery, and expanding precision medicine approaches are supporting rapid growth in cardiology-focused trials.

Cell And Gene Therapy Clinical Trials Market Growth

The global cell and gene therapy clinical trials market size reached USD 10.8 billion in 2024, grew to USD 12.47 billion in 2025, and is projected to hit around USD 45.31 billion by 2034, expanding at a CAGR of 15.43% during the forecast period from 2025 to 2034.

Value Chain Analysis

Regulatory Approvals

• U.S. cell and gene therapy trials require FDA CBER oversight and IND approval.
• Sponsors must follow strict manufacturing and quality standards.
• RMAT or Breakthrough Therapy pathways can be used for faster review.
• Key players: Novartis, Gilead Sciences, Bristol-Myers Squibb, Kite Pharma, and Juno Therapeutics.

Patient Support and Services

• Patient support in U.S. CGT trials emphasizes coordinating travel, scheduling, and treatment logistics for complex, long-term studies.
• Participants receive clear education and counseling to understand procedures, risks, and long-term follow-up requirements.
• Personalized concierge-style assistance helps reduce financial, emotional, and practical burdens on patients and caregivers.
• Key players: Bluebird Bio, Spark Therapeutics, Novartis, AveXis, and CRISPR Therapeutics.

Top Vendors in the Market & Their Offerings

SWOT Analysis

Strengths

• Advanced biotechnology infrastructure and strong R&D ecosystem.
• Supportive FDA regulatory pathways, including accelerated approvals.
• Large pool of experienced clinical research organizations and specialized talent.
• Robust funding from private investors, venture capital, and government programs.

Weaknesses

• High cost and long duration of clinical trials.
• Complex manufacturing and supply chain requirements for cell and gene therapies.
• Limited patient availability for rare disease trials.

Opportunities

• Growing prevalence of cancer, rare genetic disorders, and chronic diseases.
• Advancements in gene-editing, CAR-T, and regenerative therapies.
• Increasing collaborations between biotech firms, academia, and pharma.
• Expansion of digital and AI tools for trial optimization.

Threats

• Stringent regulatory requirements and potential trial delays.
• Safety concerns and adverse events impacting therapy approval.
• Competition from global markets and alternative therapeutic approaches.
• High failure rates in early-stage clinical trials.

What are the Recent Developments in the U.S. Cell and Gene Therapy Clinical Trials Market?

• In May 2025, Ensoma announced FDA clearance of its IND application for EN-374, targeting X-linked chronic granulomatous disease. This approval enables the development of their in vivo HSC engineering platform, advancing potential treatments for X-CGD and other genetic disorders.
• In April 2025, Labcorp broadened its oncology portfolio to enhance support for clinical research and assist biopharma companies in accelerating drug development programs.

Segments Covered in the Report

By Phase

• Phase I
• Phase II
• Phase III
• Phase IV

By Indication

• Oncology
• Cardiology
• CNS
• Musculoskeletal
• Infectious diseases
• Dermatology
• Endocrine, metabolic, genetic
• Immunology & inflammation
• Ophthalmology
• Hematology
• Gastroenterology
• Others