The United States' Pioneering Role in Cell and Gene Therapy
By Joseph Pategou
With the rapid globalization and strong momentum of the cell and gene therapy (CGT) market, it is easy to overlook the pioneering role the United States has played in its development. This raises two key questions: What was the U.S.’ role in the birth of CGT, and what is its contribution today?
Contributions of Individual Scientists and the NIH to Cell and Gene Therapy
The idea of gene therapy first emerged in the late 1970s following the development of recombinant DNA technology.
American researchers played a crucial role in shaping gene therapy during the 1970s and 1980s. Notably, Professor Theodore Friedmann was among the first to conceptualize gene therapy, while Martin J. Cline made history with the first attempt at gene therapy. These pioneering efforts set the stage for future breakthroughs.
In response to growing scientific interest, the National Institutes of Health (NIH) established the Recombinant DNA Advisory Committee (RAC) and published federal guidelines for recombinant DNA research. This laid the foundation for safe and regulated advancements in the field.
A major breakthrough occurred in 1990 when NIH researchers conducted the first-ever human gene therapy trial. A four-year-old girl with adenosine deaminase deficiency (ADA)—a rare genetic disorder causing severe immunodeficiency—was treated using gene transfer technology. This historic milestone demonstrated the therapeutic potential of gene therapy and paved the way for further innovations.
Beyond regulatory oversight, the NIH has been instrumental in funding and advancing CGT research. For instance, the Somatic Cell Genome Editing program, part of the NIH Common Fund, is dedicated to reducing the burden of diseases caused by genetic mutations. More recently, in 2023, NIH awarded Yale School of Medicine a $40 million grant to support CRISPR-based gene therapy development for brain diseases, including Angelman syndrome and H1-4 (HIST1H1E) syndrome.
U.S. Dominance in Clinical Trials, Approvals, and Regulation
The United States continues to grow and dominate the cell and gene therapy (CGT) sector.
In 2020, there were 512 companies developing cell and gene therapies in the U.S. with 505 ongoing clinical trials conducted at U.S. sites. By 2024, the number of developers in North America had surged to 1,230, with 981 ongoing clinical trials.
This dominance in clinical trials has translated into significant regulatory approvals by the FDA. In 2023 alone, the agency approved five gene therapies for rare genetic diseases and nine CGTs, including the first two adoptive cell therapies for solid tumors. Notable approvals include Aucatzyl for acute lymphocytic leukemia and Tryngolza for lipoprotein lipase deficiency.
Beyond clinical trials and approvals, efforts have been made to streamline regulatory processes and expand patient access to these advanced therapies. In 2021, the Bespoke Gene Therapy Consortium (BGTC) was established as a research initiative by the U.S. Foundation for the National Institutes of Health (FNIH). This initiative brings together the FDA, NIH, five nonprofit organizations, and ten pharmaceutical companies. Its primary goal is to develop standardized platforms that accelerate the creation and delivery of customized gene therapies. Many of these therapies target rare diseases that may otherwise be overlooked due to a lack of commercial viability.
Further advancements were announced in early 2024 with the introduction of the Cell and Gene Therapy Access Model. This initiative allows the Centers for Medicare and Medicaid Services (CMS) to negotiate outcomes-based agreements with manufacturers. The model is expected to launch in January 2025, with state and territory enrollment occurring on a rolling basis. By implementing this system, policymakers aim to make transformative treatments more accessible to patients suffering from rare and severe diseases.
U.S. Dominance in Funding, Revenue, and Deals
Funding for U.S.-based startups in gene, cell, and RNA therapy remains strong with several notable financing rounds in late 2024.
In October 2024, Judo Bio launched with $100 million in financing, while City Therapeutics secured $135 million in Series A funding, both focusing on RNAi modalities. During the same month, March Biosciences raised $28.4 million in an oversubscribed Series A round for CAR-T therapy. Leal Therapeutics also closed a $45 million financing round for antisense oligonucleotide therapy.
In November 2024, Trace Neuroscience launched with $101 million in Series A funding to advance antisense oligonucleotide therapies. The momentum continued into December 2024, with Portal Biotechnologies raising $7 million in seed financing for intracellular delivery in gene editing and gene expression modulation. Tolerance Bio secured $20.2 million in seed funding for iPSC therapy, while GEMMABio raised $34 million in seed funding for gene therapy.
In addition to startup funding, the U.S. remains dominant in dealmaking within the gene, cell, and RNA therapy sector. Strategic alliances, acquisitions, and public financings continue to shape the market. Poseida Therapeutics was acquired for $1.5 billion, while Kate Therapeutics secured a $1.1 billion deal in up-front and milestone payments. IPOs and follow-on financings have further contributed to the sector’s growth. According to the Alliance for Regenerative Medicine, total investment in North America surpassed $11.8 billion in 2024.
On the commercial side, the U.S. accounted for 65% of global CGT revenue in 2024. While market size estimates vary across different firms, there is a clear consensus that the U.S. cell and gene therapy market has been expanding and is expected to continue its growth trajectory in the coming years.
Conclusion
The United States has been a pioneer in cell and gene therapy and continues to dominate it, driven by its public and private institutions, research advancements, and strong investor support.
With ongoing innovation in research, clinical development, and regulatory frameworks, the U.S. remains at the forefront of the CGT industry. The combination of scientific progress and policy initiatives is shaping the future of these transformative therapies.
At the same time, the globalization of the CGT sector is accelerating, bringing new innovations, increased financing, and broader access to advanced therapies for patients worldwide. This international growth benefits not only individuals but also society as a whole.
Acknowledgment
I want to express my gratitude to the Alliance for Regenerative Medicine for giving me access to data link to Cell and Gene Therapy landscape. The Alliance for Regenerative Medicine (ARM) is the leading international advocacy organization dedicated to realizing the promise of regenerative medicines and advanced therapies.
About the Author
Joseph Pategou has authored over 30 articles in esteemed journals such as Cell and Gene Therapy, The Indian Economist, Labotech.eu, Drug Discovery & Development and others. A former consultant at Boston Consulting Group in New York. He has held and continues to hold operational roles in biopharma companies. The views expressed are his own. He holds an MBA from New York University and a master’s degree from HEC Paris. You can connect with and follow him on LinkedIn, Instagram, or Twitter.
Sources:
- American Society of Gene & Cell Therapy (ASGCT). Website
- European Society of Gene and Cell Therapy (ESGCT). Website
- PubMed – National Library of Medicine. "Adenosine Deaminase Deficiency." Abstract
- Yale Medicine. "Large NIH Grant Supports CRISPR-Based Gene Therapy Development for Brain Diseases." Article
- National Center for Advancing Translational Sciences (NCATS). Website
- Foundation for the NIH (FNIH). Website
- Centers for Medicare & Medicaid Services (CMS). "Cell and Gene Therapy Access Model." Model Information
- Alliance for Regenerative Medicine (ARM). Website