National Day Special Six: Pipeline Development, Multi-Field Progress, China's CGT Drugs Enter a Critical Period for Commercialization

Recently, ten departments including the Ministry of Industry and Information Technology jointly issued the "15th Five-Year Plan for the Development of the Pharmaceutical Industry" (hereinafter referred to as the "Plan"). The "Outline of the 15th Five-Year Plan for National Economic and Social Development of the People's Republic of China" issued in March this year listed biomedicine as a national emerging pillar industry. The release of this special plan represents further refinement and implementation of the Outline's arrangements. The Plan lists Cell and Gene Therapy (CGT) drugs as a key innovation direction in the biopharmaceutical field, clearly proposing to focus on developing new CAR-T cell therapy products, general CAR-T, in vivo gene-edited CAR-T, multi-functional induced stem cell (iPSC) therapy products, etc., and focus on developing AAV carriers and LNP delivery systems. It is worth noting that the Plan includes general cell therapy in the list of key technologies to be broken through, indicating that CGT has moved from an industry exploration level to a national strategic technology breakthrough level."15th Five-Year" period, CGT in China will show the following core characteristics: The development pattern will evolve from a single track to multiple indications simultaneously; the technical route will shift from autologous CAR-T to general and in vivo CAR-T.

In 2025, there were 149 registered clinical trials of CGT drugs in China, involving 131 varieties. Among them, there were 81 cell therapy varieties and 50 gene therapy varieties, accounting for 61.8% and 38.2% respectively. In terms of the number of trials, 97 cell therapy trials were registered, accounting for 65.1%; 52 gene therapy trials were registered, accounting for 34.9%. In terms of trial phases, Phase I was the main type, with 82 trials registered, accounting for 55.0%; 25 Phase II trials, accounting for 16.8%; 4 Phase III trials, accounting for 2.7%; and 38 other trials, accounting for 25.5%.

The CGT pipeline reserve in China has reached a considerable scale. Cell therapy remains the main track, while gene therapy is accelerating its pursuit, and the overall R&D activity is continuously rising. At the same time, clinical trials are mainly early-stage, and the proportion of Phase III is low, indicating that the industry is still in a critical stage of transitioning from technical verification to commercialization. During the "15th Five-Year" period, with continuous breakthroughs in advanced directions such as general CAR-T, in vivo CAR-T, and iPSC-derived cell therapies, as well as diversified payment system innovations and large-scale expansion of CDMO capacity, the CGT industry in China is expected to achieve a substantial leap from the advantage in the number of clinical pipelines to the ability to achieve commercialization.

The first line is technological innovation.The "Plan" includes general cell therapy in the list of key technologies to be broken through, and clearly supports advanced directions such as in vivo gene-edited CAR-T and iPSC therapy products. General CAR-T and in vivo CAR-T are considered key paths to solve the problems of personalized preparation of autologous CAR-T and high pricing of millions, and are expected to shift cell therapy to the era of large-scale preparation.

The differentiation of this technical route is accelerating. General CAR-T has evolved from the first generation solution that requires CD52 monoclonal antibody preprocessing to the third generation platform based on SPPL3 single gene knockout to regulate TCR/MHC function dependence of glycosylation. The technical feasibility of multi-gene target editing has been verified, but scaling up production and capacity improvement still face challenges in the process, and the overall maturity is still not high. At the same time, the attention of in vivo CAR-T is rapidly exceeding the general route. In vivo CAR-T directly delivers CAR genes to the patient's body through a single intravenous injection to complete T cell transformation, skipping the cumbersome in vitro manufacturing process of traditional CAR-T, and is expected to significantly reduce costs and shorten the treatment cycle. At the 2026 ASCO annual meeting, several in vivo CAR-T pipelines presented positive results, achieving a crucial leap from concept to clinical feasibility.

The second line is industrial chain security.The "Plan" proposes key advanced technologies in the industrial maturation stage, such as breaking through the technology of large-scale preparation of gene therapy virus carriers and human induced pluripotent stem cell-derived cell drug technologies. In the CGT field, the localization replacement of upstream links such as virus carriers, cell processing equipment, and automated preparation systems is accelerating.

Currently, the bottleneck problems in the upstream of the CGT industry chain are still prominent. Core culture medium, key reagents, and automated closed production lines still rely heavily on imports. In the field of virus carriers, the signal of industrialization upgrading is particularly clear. As more pipelines move towards clinical and commercialization, the key to AAV development is whether high-quality carriers can be prepared stably, precisely, scalable, and eligible for application. The competition in AAV is entering a more complex stage: it is necessary to prove the usefulness of the carrier, as well as that it can be "made, stable, and good enough".

The third line is industrial ecosystem.Among the 25 key tasks deployed in the "Plan", "developing specialized production service industry for CDMO" is included, clearly proposing to strengthen the construction of pharmaceutical R&D and production service platforms, and accelerate the verification, transformation, and industrialization application of key technologies for new modalities of drugs. Local CGT-CDMO enterprises are accelerating capacity expansion and qualification breakthroughs.

The CGT-CDMO track is in a stage where order reduction and capacity upgrade are combined. Leading enterprises added more than 150 million yuan in CDMO orders in the first half of the year, undertaking more than 790 projects in total, including 6 Phase III clinical projects, and obtained the "Drug Production License (Class C)", becoming service providers with both large-scale capacity and commercial production licenses. The significance of qualification breakthroughs lies not only in compliance but also in the transformation of CDMO enterprises from clinical stage service providers to commercial production partners. The pace of internationalization is also accelerating. China's CGT CDMO cost advantages and process capabilities are gaining substantial recognition in the international market, and global service capabilities are beginning to take shape. In addition, local infrastructure construction for the industry is also accelerating. Cities such as Shenzhen have started to build CGT industry public service platforms, focusing on three pain points: difficulty in large-scale production, high commercialization costs, and weak alignment with international standards, and planning to build an integrated R&D, preparation, and quality control service system covering plasmid DNA, virus carriers, and cell drugs.

The fourth line is diagnosis and treatment integration.The "Plan" proposes supporting pharmaceutical industrial enterprises to extend to the entire "testing—diagnosis—treatment" chain, establish a precise treatment product portfolio based on targets and types, and accelerate the R&D and industrialization of precise treatment products such as targeted drugs, CGT drugs, innovative traditional Chinese medicine preparations, and minimally invasive interventional devices in major disease areas. The coordinated development of CGT products and precise diagnosis will become an important trend.

The implementation of this trend is first attributed to the clarity of the policy framework. The "Regulations on the Management of Clinical Research and Clinical Transformation Applications of Biomedical New Technologies" implemented on May 1, 2026, is the first national administrative regulation on biomedical new technologies, clarifying the dual routes of technical approval by the National Health Commission and drug approval by the National Medical Products Administration, and setting a roadmap for the standardized development of the CGT industry. The clarity of policies makes the path for CGT products from R&D to clinical application more predictable, and provides a institutional foundation for the collaborative development of companion diagnostics and treatment products.

At the technical level, companion diagnostics, as a pre-step in CGT precision treatment, are evolving from auxiliary tools to a core process. Some enterprises have built a complete technical platform covering target verification, methodology development, clinical trial sample testing, and minimal residual disease monitoring. The industry is forming an integrated model of "companion diagnostics + targeted treatment", and the R&D cycle and cost are expected to be significantly reduced. For CGT products, precise diagnosis is particularly important. The efficacy of cell therapy products highly depends on the expression level of targets and patient screening, and the accuracy of diagnosis directly determines the response rate and safety of treatment. Therefore, diagnosis and treatment integration is not just a simple industrial extension, but an inherent requirement for realizing the clinical value of CGT products. With the deep application of AI and multi-omics technologies, patient stratification and efficacy prediction based on biomarkers are expected to become important supports for CGT product registration and commercialization.

In the field of anti-tumorare the most concentrated indications for CGT drugs. The "Plan" clearly focuses on key areas such as cancer in "actively cultivating and strengthening the innovative medicine and device industry", and proposes to focus on developing new CAR-T cell therapy products, general CAR-T, in vivo gene-edited CAR-T, etc.

In the field of neurological diseases. The "Plan" includes neurological diseases in the key focus areas of "actively cultivating and strengthening the innovative medicine and device industry", and clearly proposes to deepen the application of advanced technologies such as brain-machine interfaces and break through key technologies such as general cell therapy.

In the field of blood system diseasesare the third major indication area for cell therapy, with mature CGT commercialization. The "Plan" includes general cell therapy in the list of key technologies to be broken through. Blood system diseases, as the first indication area where general technology was verified, are expected to first achieve a paradigm shift from personalized customization to standardized mass production.

In the field of skin and facial organ diseasesare the second major indication area for gene therapy. The "Plan" clearly lists AAV carriers and LNP delivery systems as key development directions, and proposes to promote the deep integration of genetic testing, biogenetic technology, and disease prevention systems, and strengthen early screening and prevention of major diseases such as genetic diseases and birth defects.

In the field of gynecologyare the third major indication area for gene therapy CGT. In gynecological tumors, ovarian cancer lacks effective treatment methods due to strong immune microenvironment suppression and low response rate to immune checkpoint blockade, and cell therapy provides a new intervention path for it.

"15th Five-Year" is a critical period for CGT in China to move from technical verification to commercialization. The "Plan" includes general cell therapy in the list of key technologies to be broken through, and includes iPSC therapy products, AAV carriers, and LNP delivery systems in key innovation directions, providing top-level design guidance for industrial technical innovation and industrialization implementation.

Looking to the future, CGT in China will open new paths through original innovation, strengthen the foundation with an independent industrial chain, and take accessibility as the value goal. It will work hard in directions such as overcoming solid tumors, breaking through general technologies, and international cooperation, injecting Chinese momentum into global treatment of major diseases.