Country Level Analysis:

Increasing approved gene therapy treatments
available in the United States drive the growth of the market. Leading research
and developing a safe and effective gene therapy is expensive, and it can be
challenging and inefficient to produce a gene therapy on a large scale.  More
than 4,000 gene, cell, and RNA therapies in expansion. Early–phase clinical
trials are significant for evaluating novel medicine safety, tolerability, and
pharmacokinetics.

They offer appreciated insights in the
safety of potential therapeutic agents before they are progressive to
larger–scale research. The cell and gene therapy areas are increasing
exponentially, with novel players developing daily and much development being
made both in and out of the laboratory.

⬥︎ For Instance, In April 2025, Abeona
Therapeutics Inc. announced the U.S. Food and Drug Administration (FDA) has
approved ZEVASKYN gene-modified cellular sheets, also known as pz-cel, as the
first and only autologous cell-based gene therapy for the treatment of wounds in
adult and pediatric patients with recessive dystrophic epidermolysis bullosa
(RDEB), a serious and debilitating genetic skin disease.

U.S. Cell and Gene Therapy Clinical
Trials Market Segmentation Analysis:

By Phase Analysis:

The phase III segment dominates in the U.S.
cell and gene therapy clinical trials market, as phase III trial continues with
the emphasis on safety and efficacy, but increases the standards than the Phase
II by growing the size and refining the endpoints essential for approval. A
drug in Phase III can be under investigation for many years and potentially
enrol thousands of subjects in a randomized, double-blind, placebo-controlled
trial. The NDA covers all the scientific information that the business has
gathered during the phases in all trials.

On the other hand, the phase I segment is
expected to grow exponentially during the forecast period as phase I trials are
done to discover the highest dose of a novel treatment that can be given safely
deprived of causing serious adverse effects. These studies help to decide on
the greatest way to give the novel treatment. Phase I trials look at what the
treatment does to the body and what the body does with the treatment. Phase one
in most clinical trials tests if the therapy is safe for a small number of well
volunteers. In gene and cell therapy clinical trials, the conduct can be far
more challenging and very precise to a disease, so strong participants are not
generally used.

By Indication Analysis:

The oncology
segment generated the highest market revenue in 2024, as clinical
trials are the novel ways to prevent, find, and treat cancer. They support
physician to improve the quality of life for patients with cancer by testing
ways to manage the adverse effects of cancer and its treatment. Most cancer
clinical trials are conduct studies that contain people who have cancer. These
trials test novel treatments or new ways of using present treatments. The main
aim of cancer screening trials is to test ways to find cancer before it causes
symptoms, when it may be easier to manage.

On the other hand, the cardiology segment
is expected to grow exponentially during the forecast period as therapeutic use
of cell therapies has the probable to reverse myocardial injury and enhance
cardiac function, in contrast to most present medical therapies. Cell-based
therapy enhanced quality of life for patients with progressive heart failure,
Mayo Clinic scientists and international partners discovered in a late-stage
international clinical trial.

Some of the Prominent Players in the
U.S. Cell and Gene Therapy Clinical Trials Market

• Charles
River Laboratories

• ICON
Plc

• IQVIA

• LabCorp

• Medpace

• Novotech

• PAREXEL International Corp.

• Syneos
Health

• Thermo Fisher
Scientific, Inc.

• Veristat, LLC

What is Going Around the Globe? 

⬥︎ In April 2025, INmune Bio Inc. a clinical-stage
biotechnology company targeting inflammation and immunology through the innate
immune system has partnered with the Cell and Gene Therapy Catapult (CGT
Catapult) to establish large-scale, commercial-ready manufacturing for its cell
therapy platforms. CGT Catapult is an independent technology and innovation
organization specializing in the advancement of the cell and gene therapy
industry. 

⬥︎ In April 2025, AGC Biologics launched
New Dedicated Cell and Gene Business Division. The new Cell and Gene
Technologies Division will focus on elevating existing AGC Biologics
capabilities and supporting developers in need of capacity, scientific
capabilities, and technically qualified cell and gene CDMO operators

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Related Report –

⬥︎ Cell And Gene Therapy Clinical Trials Market – https://www.novaoneadvisor.com/report/cell-and-gene-therapy-clinical-trials-market

⬥︎ Cell and Gene Therapy Market – https://www.novaoneadvisor.com/report/cell-and-gene-therapy-market

⬥︎ U.S. Cell And Gene Therapy Clinical Trial Services Market – https://www.novaoneadvisor.com/report/us-cell-gene-therapy-clinical-trial-services-market

⬥︎ Cell & Gene Therapy Contract Research Organizations Market – https://www.novaoneadvisor.com/report/cell-and-gene-therapy-contract-research-organizations-market

⬥︎ Cell And Gene Therapy Bioanalytical Testing Services Market – https://www.novaoneadvisor.com/report/cell-and-gene-therapy-bioanalytical-testing-services-market

⬥︎ Cancer Gene Therapy Market – https://www.novaoneadvisor.com/report/cancer-gene-therapy-market

⬥︎ Cell And Gene Therapy Manufacturing Market – https://www.novaoneadvisor.com/report/cell-and-gene-therapy-manufacturing-market

⬥︎ U.S. Gene Therapy Market – https://www.novaoneadvisor.com/report/us-gene-therapy-market

⬥︎ U.S. Cell And Gene Therapy Manufacturing Market – https://www.novaoneadvisor.com/report/us-cell-and-gene-therapy-manufacturing-market

Segments Covered in the Report

This report forecasts revenue growth at
country levels and provides an analysis of the latest industry trends in each
of the sub-segments from 2021 to 2034. For this study, Nova one advisor, Inc.
has segmented the U.S. cell and gene therapy clinical trials market.

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

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