China Biotech Innovation: Can US Regain Lead in Cell & Gene Therapy?

The United States may need to recalibrate its approach to biotechnology innovation if it wants to maintain its global leadership, according to a leading cell therapy expert. While CAR-T cell therapy originated in the U.S., China is now conducting more CAR-T cell trials than America, rapidly generating data and accelerating the development of these potentially life-saving treatments.

Michel Sadelain, director of the Columbia Initiative in Cell Engineering and Therapy, highlighted this shift at the STAT Breakthrough Summit East. He noted that China is swiftly building upon research initially published in the U.S., quickly launching trials and collecting patient data while the American system grapples with funding and logistical hurdles. This dynamic raises questions about the U.S.’s long-held dominance in drug discovery and development.

A Dual-Track System Accelerates Progress in China

A key factor driving China’s progress is its “dual-track” regulatory system. This system allows for “investigator-initiated trials” (IITs) – studies run by clinical researchers in hospitals – to begin quickly with less administrative burden than traditional, centrally-approved drug trials. According to a 2025 analysis, IITs for cell and gene therapy products have grown rapidly since 2018, enrolling over 30,000 participants – double the number in typical, nationally-overseen trials.

This approach effectively “de-risks” new experimental therapies, Sadelain explained, providing valuable early insights into their efficacy. He expressed hope that the U.S. Could adapt its system to regain some of its competitive edge, particularly by empowering academic centers to manufacture these therapies, which often originate in academic research.

Growing Concerns About U.S. Leadership in Drug Discovery

Sadelain’s remarks reflect a broader anxiety about China’s growing strength in the pharmaceutical sector. Fueled by national ambitions, substantial government investment, and a more flexible regulatory environment, China’s biotechnology industry has experienced an explosive expansion in recent years. One analysis found that China’s output of new drug discoveries tripled since 2018, while American output remained relatively flat. (Spot related STAT News report)

Historically, the U.S. Has been the dominant force in biomedical research and development since the end of World War II. This leadership has not only benefited Americans with earlier access to innovative therapies but has also given the U.S. Significant control over the global pharmaceutical market and the ability to shape international regulatory standards. However, this position is now being challenged.

Declining U.S. Investment and its Consequences

A trend of declining federal support for biomedical research over the past few decades, compounded by cuts to the federal science workforce and a disruption of the government’s research partnership with academia during the second Trump administration, is contributing to the erosion of U.S. Leadership. A recent forecast from researchers at the University of California, San Diego, predicts that China’s public spending on research will surpass that of the United States within the next two to three years. (Nature report on research spending forecasts)

Industry Perspectives: Collaboration, Not Competition

While academics like Sadelain express concern, pharmaceutical research and development executives appear more optimistic. Jane Grogan, head of research at Biogen, suggested viewing China’s rise as an opportunity for collaboration. “We’re out in China, we’re talking to VCs, academics, startups as well to see how can we actually partner with them to develop quickly and agilely drugs,” she said during a recent session.

Robert Plenge, chief research officer of Bristol Myers Squibb, echoed this sentiment, emphasizing that the ultimate goal is to deliver the best possible medicines to patients, regardless of their origin. “If you’re a patient wanting to receive the best possible medicine as quickly as possible, I feel having innovation across the globe is the best possible outcome,” Plenge stated. Stelios Papadopoulos, board chair of Exelixis, also downplayed the idea of China as a threat, noting the presence of “a lot of smart people” there.

CAR-T Cell Therapy: A Brief Overview

CAR-T cell therapy, or chimeric antigen receptor T-cell therapy, is a type of immunotherapy that reprograms a patient’s own immune cells to recognize and attack cancer cells. It has shown remarkable success in treating certain blood cancers, but has faced challenges in treating solid tumors, which represent the vast majority of cancer cases. HIT cells, a newer type of cell therapy developed by Sadelain’s team at Columbia, are designed to overcome some of these challenges by enhancing the sensitivity of the immune cells. A recent study published in Science showed that HIT cells could completely eliminate kidney, pancreatic, and ovarian cancers in mice. (Columbia University News report on HIT cell therapy)

What’s Next for Cell and Gene Therapy?

The evolving landscape of cell and gene therapy development highlights the need for ongoing evaluation and adaptation. The U.S. Food and Drug Administration (FDA) continues to refine its regulatory pathways for these innovative therapies, balancing the need for safety and efficacy with the desire to accelerate access for patients. Further research is needed to address the challenges of treating solid tumors and to improve the affordability and accessibility of these potentially life-changing treatments. The success of in vivo CAR T cell therapies, which modify T cells directly within the patient’s body, is also being closely watched, with several companies currently running Phase 1 clinical trials. (Drug Discovery News report on in vivo CAR T cell trials)

the global fight against cancer will likely benefit from a collaborative approach, leveraging the strengths of researchers and innovators from around the world. As Plenge emphasized, the focus should remain on delivering the best possible medicines to patients, wherever those medicines may originate.