U.S. Cell and Gene Therapy Clinical Trials Market Size, Share, Trend, Industry Analysis Report By Phase (Phase I, Phase II, Phase III, Phase IV), By Indication – Market Forecast, 2025–2034

The U.S. cell and gene therapy clinical trials market size is expected to reach USD 22.25 billion by 2034, according to a new study by Polaris Market Research. The report “U.S. Cell and Gene Therapy Clinical Trials Market Size, Share, Trend, Industry Analysis Report By Phase (Phase I, Phase II, Phase III, Phase IV), By Indication – Market Forecast, 2025–2034” gives a detailed insight into current market dynamics and provides analysis on future market growth.

The U.S. cell and gene therapy clinical trials market focuses on the development and testing of advanced therapies designed to modify or replace genetic material or manipulate cellular function to treat diseases. The market is gaining momentum due to increasing demand for personalized and curative treatments, particularly for conditions that have limited therapeutic options. Strong regulatory support, including fast-track designations and orphan drug incentives, is encouraging clinical activity and reducing time-to-market. Research funding from both public and private sectors continues to flow into the space, allowing trial sponsors to explore novel platforms, including CRISPR-based gene editing and next-generation viral vectors.

A shift toward decentralized and adaptive trial designs is helping improve patient recruitment and retention while also addressing logistical and compliance hurdles. Integration of digital technologies is enhancing real-time data capture and trial monitoring, improving safety assessments and trial efficiency. Collaborations among research institutions, biotech firms, and contract research organizations are becoming more strategic, aiming to speed up protocol execution and streamline trial phases. The market is also experiencing rising trial volumes in rare and ultra-rare disease indications, supported by active patient advocacy networks and registries. Opportunities are emerging in therapeutic areas such as neurodegenerative diseases, oncology, and cardiovascular conditions, where traditional treatments fall short. Rapid scientific progress, aligned regulatory frameworks, and robust trial infrastructure continue to position this market for sustained growth over the next decade.

U.S. Cell and Gene Therapy Clinical Trials Market Report Highlights

By phase, the phase III segment dominated the market in 2024 due to the sharp rise in late-stage programs targeting high-burden conditions such as cancer and rare genetic diseases.

Based on indication, the oncology segment held the largest revenue share in 2024 due to the strong demand for cell and gene therapies that overcome resistance to conventional treatments such as chemotherapy and radiation.

A few key players in the U.S. cell and gene therapy clinical trials market include Charles River Laboratories; ICON Plc; IQVIA; LabCorp; Medpace; Novotech; PAREXEL International Corp.; Syneos Health; Thermo Fisher Scientific, Inc.; and Veristat, LLC.

Polaris Market Research has segmented the U.S. cell and gene therapy clinical trials market report on the basis of phase and indication:

By Phase Outlook (Revenue, USD Billion, 2020–2034)

Phase I

Phase II

Phase III

Phase IV

By Indication Outlook (Revenue, USD Billion, 2020–2034)

Oncology

Cardiology

CNS

Musculoskeletal

Infectious diseases

Dermatology

Endocrine, metabolic, genetic

Immunology & inflammation

Ophthalmology

Hematology

Gastroenterology

Others Show more