Duy-Cuong Le, MD, Ph.D.
Laboratory, Vinmec Riverside, Vinmec Healthcare System, Vietnam
College of Health Sciences, VinUniversity, Hanoi, Vietnam

Introduction

Cell and gene therapy (CGT) is reshaping modern medicine by enabling interventions at the level of cellular function and genetic programming. From hematologic malignancies to rare inherited disorders, these therapies have shifted from experimental promise to clinical reality in many parts of the world.

In Vietnam, CGT is emerging within a distinctive context: a system with growing scientific capability, early clinical successes, and an evolving regulatory framework that is still catching up with technological advances. Understanding this landscape requires examining not only clinical progress, but also the legal instruments that govern how such therapies are developed, tested, and applied.

Scientific Foundations and the Scope of Application

The biological basis of CGT lies in the unique properties of stem cells, particularly their ability to self-renew and differentiate into specialized cell types. These characteristics enable applications in regenerative medicine and targeted therapies, but they also introduce significant biological risks if not properly controlled.

In Vietnam, the most mature application remains hematopoietic stem cell transplantation, widely used in the treatment of hematologic diseases at institutions such as Vietnam National Children’s Hospital and National Institute of Hematology and Blood Transfusion (NIHBT). This procedure reflects a high degree of clinical standardization and regulatory acceptance.

Beyond this domain, stem cell–based interventions have been explored in neurology, orthopedics, cardiology, and aesthetic medicine. However, most of these applications remain experimental, lacking sufficient clinical evidence for routine use.

The 2020 National Guideline: Defining Cell Therapy as a High-Risk Biomedical Intervention

A defining feature of Vietnam’s regulatory approach is the issuance of the national guideline on research and application of cell-based products by the Ministry of Health in 2020. This document provides the first systematic framework for understanding and managing cell therapy within the country.

The guideline explicitly defines cell therapy as the use of living cells administered into the human body for therapeutic purposes. More importantly, it establishes that such interventions must be treated as high-risk biomedical technologies, requiring rigorous control across all stages of development and application. Rather than being categorized as routine medical techniques, cell-based therapies are framed as interventions that demand structured evaluation of safety and efficacy.

A key principle embedded in the guideline is the classification of cell products according to their level of manipulation and associated risk. This approach aligns with international regulatory thinking and underscores that minimally manipulated cells and highly engineered products should not be treated equivalently. The document also emphasizes that all cell-based products must be manufactured under conditions consistent with Good Manufacturing Practice (GMP), ensuring traceability, sterility, and quality control.

Equally important is the requirement that clinical use must be preceded by appropriate research and evaluation. The guideline reinforces that any application of cell therapy must be grounded in scientifically validated protocols and subject to ethical oversight. In effect, it institutionalizes a “research-first” model, in which clinical deployment is contingent upon prior evidence rather than market demand.

This framework has significant implications. It formally distinguishes legitimate clinical innovation from unregulated practice, and it provides a legal and technical basis for restricting the use of cell therapies outside controlled settings.

Clinical Research Governance: The Expanding Role of Circular 50/2025

The introduction of Circula 50/2025 from Vietnam Ministry of Health marks a significant step in strengthening the legal infrastructure for clinical research in Vietnam. While originally designed for drug trials, its scope and principles are highly relevant to CGT, particularly for advanced therapies that function as biological products.

This regulation reinforces key requirements for clinical trials, including ethical approval, protocol standardization, safety monitoring, and data integrity. It also aligns Vietnam more closely with international clinical research standards, thereby facilitating more rigorous evaluation of emerging therapies such as CAR-T and gene-based interventions.

When viewed alongside the 2020 national guideline, Circular 50 contributes to a more coherent regulatory pathway: from laboratory research to clinical testing, and eventually to controlled therapeutic application. Together, these frameworks strengthen the legitimacy of CGT research while setting clear boundaries against premature or unauthorized clinical use.

Research Capacity and the Limits of Translation

Vietnam has demonstrated notable capacity in CGT-related research. According to Vinmec, several areas of stem cell research, particularly mesenchymal stem cells and regenerative medicine, were initiated relatively early and have progressed alongside global developments.

Despite this progress, a significant gap persists between research output and clinical implementation. This reflects structural challenges, including limited GMP-compliant manufacturing infrastructure and the absence of fully developed regulatory pathways for advanced therapies.

As a result, CGT in Vietnam remains largely research-driven, with only a limited number of applications transitioning into standardized clinical practice.

CAR-T Therapy and the Emergence of Domestic Innovation

The introduction of CAR-T therapy represents a defining moment in Vietnam’s CGT landscape. This advanced immunotherapy, which involves engineering a patient’s T cells to target cancer, has already transformed oncology globally.

In Vietnam, early clinical applications have been pioneered by Vinmec Healthcare System. Reported cases, including successful treatment in pediatric leukemia, demonstrate that highly complex therapies can be implemented domestically when supported by appropriate infrastructure and expertise.

These achievements highlight the emergence of a localized translational ecosystem, where research, manufacturing, and clinical care are integrated. At the same time, they underscore the uneven distribution of such capabilities across the national healthcare system.

Regulatory Structure: Between Control and Incompleteness

Vietnam’s regulatory framework for CGT combines general healthcare law, technical classifications, and specialized guidance. The Law on Medical Examination and Treatment (2023) and Circular 23/2019/TT-BYT provide a legal basis for certain cell-based procedures.

However, it is the combination of the 2020 national guideline and Circular 50/2025 that most clearly defines the current regulatory approach. Together, they establish a continuum from research to clinical application, grounded in risk assessment, quality control, and ethical oversight.

Nevertheless, a comprehensive legal framework specifically tailored to CGT—particularly gene therapy and engineered cell therapies—has yet to be fully developed.

The Persistent Challenge of Misuse

Despite a structured regulatory philosophy, enforcement challenges remain. Reports from mainstream media indicate the proliferation of clinics offering so-called stem cell therapies without scientific validation or regulatory approval.

Such practices directly contradict established principles, including GMP requirements and mandatory clinical evaluation. They also expose patients to serious risks, ranging from immune reactions to infections and tumor formation.

This disconnect between regulatory intent and real-world practice remains one of the most pressing issues in Vietnam’s CGT landscape.

Conclusion

Cell and gene therapy in Vietnam is at a critical stage of development. The country has established a strong scientific foundation and demonstrated early success in advanced therapies such as CAR-T. At the same time, its regulatory framework—anchored by the 2020 national guideline and reinforced by Circular 50/2025—reflects a cautious, research-centered approach.

Yet, the system remains incomplete. Bridging the gap between research and clinical practice, strengthening enforcement, and developing a dedicated legal framework for advanced therapies will be essential steps moving forward.

Vietnam’s experience illustrates a broader global challenge: how to translate cutting-edge biomedical innovation into safe, effective, and accessible healthcare—without losing control of its risks.