China CDE Defines Classification Rules for Cell & Gene Therapy Drugs

On July 3, 2026, the Center for Drug Evaluation under China’s National Medical Products Administration published the “Technical Guideline for the Scope and Classification of Cell Therapy Medicinal Products (2026 Edition)” and the “Technical Guideline for the Scope and Classification of Gene Therapy Medicinal Products (2026 Edition).” Both took effect upon publication.

The guidelines close a gap that has grown more pressing as cell and gene therapy platforms have converged in recent years. CAR-T, in vivo gene editing, and hybrid cell-delivery products increasingly blur the line between the two categories. These documents establish China’s formal framework for categorizing advanced therapies as either Cell Therapy Medicinal Products (CTMPs) or Gene Therapy Medicinal Products (GTMPs). This classification is a critical milestone, as it dictates the technical standards, evidence packages, manufacturing controls, regulatory strategies, and specialist capabilities required for development within China.

How the CDE Classifies Advanced Therapies

The guidelines address technologies with increasingly overlapping scientific characteristics, including:

• Engineered immune cells
• In vivo cell programming
• Nucleic acid delivery
• Genome editing
• Stem-cell-derived products
• Oncolytic microorganisms
• Cell-based delivery systems

Rather than classifying products according to platform labels, the CDE applies a three-part assessment based on:

1. Material basis and active ingredient (weighted as the primary factor)
2. Manufacturing process
3. Mechanism of action

The governing principle is simple: classification follows the form and function of the active substance administered to the patient, not the label chosen by the developer. For global companies, this means classifications used in other jurisdictions may not translate directly into China's regulatory framework. 

The CTMP Guideline: Cell as the Active Ingredient

Under the cell therapy guideline, a CTMP uses human cells, tissues, or body fluids as starting materials, undergoes ex vivo processing, and contains cells as the active ingredient.

The cells achieve the intended medical purpose through mechanisms that may include:

• Modulation of biological activity, immune characteristics, or metabolic state
• Cell replacement
• Tissue repair or regeneration
• Functional reconstruction
• Homing, cell-to-cell interactions, or paracrine activity

The category includes stem cells, stem-cell-derived cells, immune cells, and other somatic cell therapies.

The guideline excludes:

• Blood components collected for transfusion
• Directly donated and unprocessed human cells, tissues, or organs used for transplantation
• Artificial embryos, and reproductive cells used in reproductive technologies

These exclusions confirm that the CTMP framework applies to products developed and regulated through the pharmaceutical pathway. It does not extend medicinal-product classification to established transfusion, transplantation, or reproductive procedures.

Two Forms of CTMPs

CTMPs fall into two working categories. Non-genetically modified CTMPs (tumor-infiltrating lymphocytes, natural killer cells,dendritic cells,non-engineered T-cell products) undergo ex vivo isolation, expansion, activation, or physical/chemical processing without alerting their genetic material. Ex vivo genetically modified CTMPs, including

ex vivo CAR-T, ex vivo TCR-T, and genetically modified hematopoietic stem cells or  CAR-NK cells derived from human pluripotent stem cells, involve introducing nucleic acid into cells outside the body, via viral vectors, nonviral vectors, nonviral systems, or other methods, to insert, express, remove, suppress, replace, compensate for, block, or correct a gene. The determining feature across both: the final active substance administered to the patient is a cell, and any genetic modification happens before administration.

The determining feature is that genetic modification occurs before administration and the final active substance administered to the patient is a cell.

The GTMP Guideline: In Vivo Genetic Action

The gene therapy guideline defines GTMPs as products that use DNA, RNA, viral or nonviral vectors, or nucleases and proteins acting on specific genes to introduce genetic sequences, alter human gene sequences, or regulate gene expression in the body.

GTMPs may act by replacing, supplementing, blocking, correcting, or silencing specific genes. Their mechanism of action depends directly on an in vivo nucleic acid sequence, genetic modification, or gene-expression product.

The framework excludes:

• Prophylactic vaccines
• Chemically synthesized oligonucleotide drugs

Antisense oligonucleotides, small interfering RNA products, and related modalities do not automatically fall within the GTMP category when their active substances are chemically synthesized oligonucleotides.

Two Forms of GTMPs

GTMPs also split into two working categories. Nonmicrobial vector-based GTMPs, such as plasmid DNA,messenger RNA,mRNA delivered by lipid nanoparticles, TALEN proteins, CRISPR-Cas ribonucleoprotein complexes, base and prime editors, ADAR-based editing systems, andnucleic acid or editing components delivered through virus-like particles or extracellular vesicles, use physical, chemical, or nonmicrobial carries. Microbial vector based GTMPs, including Adeno-associated virus and adenovirus, Lentivirus, Herpesvirus, Salmonella, Listeria-based products, use genetically engineered viral or nonviral microorganisms to deliver functional nucleic acids into the body. Engineered oncolytic viruses and bacteria also fall into this second category whenever the microorganism acts both as an active component and as a carrier for an introduced genetic sequence.

The In Vivo vs. Ex Vivo Boundary

The most significant commercial distinction lies between ex vivo engineered cells and in vivo cell programming. Even if the clinical outcome is similar, the classification differs based on the administered material and the location of genetic modification.

• Ex vivo CAR-T products are CTMPs because the cells are genetically modified outside the body and the administered active ingredient is the engineered cell.
• In vivo CAR-T products are GTMPs because the administered product is a vector or delivery system containing nucleic acid that generates CAR-expressing cells inside the patient.

This distinction has practical consequences. Companies comparing conventional autologous or allogeneic CAR-T platforms with in vivo immune-cell engineering technologies should expect their development programs require different CMC strategies, biodistribution packages, vector controls, shedding assessments, nonclinical models, and long-term risk considerations.

Regulatory assumptions from an ex vivo CAR-T program should not be carried over to an in vivo CAR-T candidate simply because both generate functionally similar immune cells.

How the CDE Treats Complex and Borderline Products

The CDE’s framework becomes most consequential where a product does not fit neatly within a single category. In these cases, classification depends on the interaction among the active ingredient, carrier, manufacturing history, payload, and principal mechanism of action.

Cell-Based Delivery Systems

The guidelines provide additional rules for products that use cells as delivery vehicles. Two products using the same cellular carrier could follow different regulatory pathways depending on the payload and primary mechanism:

• A cell carrying a non-nucleic-acid or non-nuclease active substance will generally fall within the CTMP category when the product’s function depends substantially on the cell. This may include enucleated cells, such as red blood cells, used to transport another active substance.
• A cell carrying nucleic acid or a nuclease may fall outside the CTMP category when the product acts mainly by changing a gene sequence or regulating gene expression. Such a product may qualify as a nonmicrobial vector-based GTMP.

This presents a material due-diligence issue when assessing delivery-platform acquisitions or partnerships. Therefore, product teams should document:

• The identity of the administered active substance
• The contribution of the cell or carrier to efficacy
• Whether the payload is independently active
• Whether the principal effect depends on gene alteration or gene-expression control
• Where the relevant biological transformation occurs

The answer should be reflected consistently across the target product profile, quality documentation, nonclinical rationale, clinical protocol, and regulatory correspondence.

Stem-Cell-Derived Products

Products derived from human pluripotent stem cells may fall within different CTMP subcategories depending on their manufacturing history:

• A human pluripotent stem-cell-derived islet-cell product: May qualify as a stem-cell-derived CTMP even when its active substance contains multiple cell types, such as alpha and beta cells. 
• A stem-cell-derived product: May qualify as an ex vivo genetically modified CTMP when genetic modification occurs during reprogramming, differentiation, or preparation of the final active cell component.

For induced pluripotent stem-cell platforms, the classification assessment must therefore cover the complete process rather than only the final cell phenotype.

Use of viral vectors, plasmids, or other genetic modification systems during reprogramming or differentiation may affect the product’s subcategory even when the final therapeutic cell is not conventionally described as engineered.

This creates a clear diligence requirement for platform licensing and cross-border technology transfer. Sponsors should secure access to:

• Early cell-line history
• Reprogramming methods
• Vector-clearance information
• Clone-selection records
• Genomic characterization
• Master cell bank provenance

Gaps in the historical package may become regulatory risks later in development.

Multicomponent and Borderline Products

Both guidelines acknowledge that some products may display characteristics of multiple categories. The CDE encourages developers to discuss complex or novel cases with the review authority.

Several examples illustrate how this reasoning applies in practice.

1. A platelet precursor product that generates functional platelets after administration may be classified as a CTMP because the administered active ingredient is cellular and the therapeutic effect depends on its biological transformation.
2. A neoantigen product containing cells loaded with neoantigen peptides may qualify as a non-genetically modified CTMP because the cell is the principal functional component and the product works through modulation of immune activity.
3. A neoantigen DNA or mRNA product, or a viral vector carrying a neoantigen nucleic acid sequence, is classified as a GTMP because the active substance and mechanism depend on nucleic acid delivery and expression.
4. An extracellular vesicle carrying a CRISPR-Cas complex may qualify as a nonmicrobial vector-based GTMP because the payload modifies genetic material, even though the carrier is biologically derived.

These examples show that the CDE examines the complete functional relationship among the carrier, payload, active ingredient, manufacturing process, and mechanism of action.

Classification Affects More Than the Regulatory Label

The guidelines do not change China’s existing drug registration categories. However, CTMP or GTMP classification will materially influence development planning.

CMC and Control Strategy

For a CTMP, key CMC concerns include cell identity, purity, potency, viability, differentiation state, genetic stability, , and comparability following process changes.

For a GTMP, the focus shifts to nucleic acid identity, vector characterization, transduction/transfection performance, replication competence, genome-editing specificity, and biodistribution..

An incorrect or delayed classification can misalign assays,  specifications, and reference standards and on the clinical side, it can affect animal-model selection, biodistribution study design, tumorigenicity assessment, and first-in-human dose justification, since a cell dose and a vector genome does require different translational reasoning.

Nonclinical and Clinical Development

Classification may affect animal-model selection, biodistribution study design, tumorigenicity or insertional-risk evaluation, off-target editing assessment, persistence monitoring, and long-term follow-up planning.

The distinction also matters for first-in-human dose justification. A cell dose, vector genome dose, nucleic acid dose, editing-component dose, or combined measure may require different translational reasoning.

Facility and Compliance Planning

CTMP and GTMP platforms can create different manufacturing and containment needs - viable-cell processing, viral-vector production, genetically modified organisms, aseptic filling, cryogenic logistics orpatient-specific chain of identity – which should inform facility strategy and CMO selection..

Classification also intersects with China’s human genetic resources, ethics, and biosafety requirements, which overseas sponsors should plan for in parallel, particularly for autologous products, donor-derived platforms, and products developed using Chinese clinical samples. Actions for Advanced Therapy Developers

The immediate priority is not to rename portfolio assets. Companies should test whether their current development assumptions align with the CDE’s framework.

Reassess the Classification of Each China-Facing Asset

Sponsors should prepare a concise classification memorandum identifying:

• The administered active ingredient
• The carrier and payload
• Where any genetic modification occurs
• The principal mechanism of action
• Why alternative classifications are less appropriate

This is especially important for hybrid platforms and products described differently across jurisdictions.

Map Classification to the Evidence Package

Teams should determine whether the proposed classification is consistently reflected in:

• CMC development plans
• Analytical methods
• Release specifications
• Nonclinical studies
• Clinical dose expression
• Risk-management plans
• Long-term follow-up proposals
• Regulatory briefing documents

Any mismatch should be resolved before a formal China submission or major development milestone.

Seek CDE Engagement for Borderline Products

Early communication is particularly important for in vivo cell engineering, extracellular-vesicle delivery, cell-loaded payloads, mixed-cell structures, advanced genome editors, engineered microorganisms, and other emerging technologies.

A well-supported briefing package should explain the product’s material basis, manufacturing process, mechanism of action, proposed category, alternative interpretations, and the development consequences of each classification.

Final Thoughts

The CDE’s new guidelines represent a transition toward a more functionally focused, substance-based regulatory environment in China. By defining products based on their material basis and primary mechanism of action rather than legacy platform labels, the CDE is forcing developers to move beyond "global boilerplate" strategies.

In practice:

• CTMP Framework: Generally applies when the final active substance is a cell, even if it has undergone ex vivo genetic modification.
• GTMP Framework: Generally applies when the delivery of genetic material, via viral or non-viral vectors, is the primary driver of therapeutic effect in vivo.
• Hybrid Products: Complex carriers and multicomponent therapies require a deeper, evidence-based assessment of the active ingredient and dominant mechanism to determine the appropriate classification.

For global biotech and pharmaceutical firms, early classification is now a critical strategic filter. A misstep here can lead to costly delays in clinical initiation and CMC misalignment. Conversely, developers who proactively map their portfolios to these specific CDE criteria enable leaner development programs, more efficient CMC investments, and more predictable submission timelines.

With deep expertise in China’s regulatory landscape, Cisema is positioned to help you navigate these nuances. We partner with advanced-therapy developers to refine classification assessments, structure CDE communication, and optimize clinical development for both pharmaceutical and biotech pipelines

To evaluate how China’s 2026 cell and gene therapy classification guidelines affect your development program and long-term pathway to market, contact Cisema today.

Further Information

• Explore Cisema’s pharmaceutical consulting services.

References

• Announcement from the Center for Drug Evaluation of the National Medical Products Administration on the Release of the "Technical Guidelines for the Scope and Classification of Cell Therapy Drugs (2026 Edition)" (2026 No. 39).
• Announcement from the Center for Drug Evaluation of the National Medical Products Administration on the Release of the 'Technical Guidelines for the Scope and Classification of Gene Therapy Drugs (2026 Edition)' (2026 No. 40)

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