Policy , Clinical , Capital Empowerment : The CSGCT Conference Unleashes China’s Strength - Witnessing the Transition from Follower to Leader

Grounded in regulation, pooling wisdom, connecting globally, and creating a shared future. China's cell and gene therapy sector welcomes its annual premier event.

From September 12 to 13, 2025, the China Stem Cell and Gene Therapy Conference (CSGCT) was grandly held at the Zhongguancun Exhibition and Trading Center in Haidian District, Beijing. Under the guidance of the Haidian District People's Government of Beijing, the conference was jointly organized by the Zhongguancun Science City Management Committee and the CSGCT China Stem Cell and Gene Therapy Alliance, with the Beijing Medical Health Technology Development Center, the HIEA Medical Innovation Ecosystem Alliance, and Arterial Network serving as the hosts, and the Beijing Juneyu Culture Technology Development Co., Ltd. and the Hainewei Urban Renewal Group as the co-hosts.

At the main forum of the aforementioned conference, leaders from the Beijing Municipal Science and Technology Commission, Zhongguancun Administrative Committee, the People's Government of Haidian District, Beijing, and the Zhongguancun Science City Administrative Committee also provided a detailed introduction to the current development status of the medical and health industry in Beijing and Haidian District.

Over the past three years, Beijing has ranked first in the country in the number of medical device products approved for market launch. In 2024, the scale of the medical and health industry reached 1.06 trillion yuan, marking an 8.7% year-on-year increase, making Beijing the first city in the nation to achieve a trillion-yuan medical and health industry. Tang Jian, a second-level inspector from the Beijing Municipal Science and Technology Commission and Zhongguancun Administrative Committee, stated at the meeting, "We believe that with the support of city-region collaboration, Beijing's CGT industry will undoubtedly achieve remarkable results.". ”

Cui Ying, a member of the Party Leadership Group of the Haidian District People's Government and Deputy District Chief, noted that Haidian leverages the profound expertise of 37 higher education institutions, including Tsinghua University and Peking University, as well as 57 secondary and above hospitals such as Peking University Third Hospital and Peking University Cancer Hospital, in the field of CGT. This has fostered a "talent hub" where innovation, research, and clinical resources converge. Additionally, Haidian has attracted a cluster of innovative enterprises dedicated to the development of cell and gene therapy drugs, such as Biotech, Yimiao Shengke, Innorivon, and Northland, providing sustained momentum for cutting-edge fields like CGT. Haidian will collaborate with various sectors through policy openness, data openness, spatial openness, and institutional openness to jointly drive China's CGT industry from "following" to "leading.".

As a pilot zone for institutional innovation, Haidian District is not only strengthening its role as a source of original innovation and creating a world-class business environment but also fully driving the development of the CGT industry across its entire chain. Yuan Xueqin, Deputy Director of the Third Division of the Zhongguancun Science City Management Committee's Industry Promotion Department, explained that on the policy front, action plans and measures have been released to chart the direction for industrial development. Spatially, specialized CGT industrial clusters are being established to provide enterprises with high-quality development platforms. In terms of services, a global future pharmaceutical and health industry incubation platform is being cultivated, along with the establishment of the Beijing Pharmaceutical and Medical Device Innovation Service Station (Haidian Station) to offer one-stop services. Additionally, efforts are being made to actively open up medical application scenarios to foster deep collaboration between medical institutions and enterprises. Notably, the construction of the nation's first trustworthy data space for pharmaceutical and health industries is underway, integrating diverse data sources to inject strong momentum into industrial innovation and propel the CGT industry to new heights.

As one of the most influential international conferences in the field of cell and gene therapy in China, the CSGCT Conference has always been committed to promoting the full chain collaboration of basic research, technology development, clinical trials, industrial transformation, and global cooperation. The successful convening of the 2025 conference once again highlights China's increasing influence and innovative vitality in the global CGT field.

The conference, guided by the core philosophy of "Rooted in Regulation, Harnessing Wisdom, Connecting Globally, and Creating a Shared Future," features one main forum, 15 sub-forums, and two project roadshows. It has attracted over 2,000 participants from various sectors, including more than 10 internationally renowned CGT leaders, scientists, clinicians, entrepreneurs, and investors, who gathered together to discuss the development strategies for cell and gene therapy.

On the first day of the conference, several major industry announcements and ceremonies were successfully held. The China Cell and Gene Therapy Alliance (CSGCT Board) was officially established. The Alliance will carry out comprehensive planning across multiple areas, including policy support, academic research, clinical trials, industrial implementation, international cooperation, talent cultivation, and public education. It is committed to addressing the common challenges currently facing China’s CGT industry, accelerating the integration of Chinese CGT products into the global consumer market, and driving China’s scientific and technological innovation and industrial development in cell and gene therapy to globally advanced levels.

"Since Legend Biotech embarked on China’s global innovation journey in CGT in 2017, with the continuous reduction in process costs, the accumulation of clinical data, and the CDE’s clear definition of advanced therapy medicinal products, China is rapidly transforming from a follower to a parallel runner or even a leader in the global CGT landscape. A vibrant and highly promising CGT ecosystem is taking shape in China, contributing Chinese solutions and wisdom to global health. Science knows no borders, and neither does innovation," said Qi Fei, Co-founder of the China Cell and Gene Therapy Alliance (CSGCT) and Executive Director of Legend Capital. He noted that it is against this backdrop that the 2025 China Cell and Gene Therapy Conference came into being.

Wu Zhenhua, Founder and Chairman of the China Cell and Gene Therapy Alliance (CSGCT) and Founder & CEO of Hangzhou Jiayin Biotech, added: "Scientifically, we hope to promote global innovation to take root and flourish in China. Commercially, we aim to facilitate international cooperation and exchange. Clinically, we will leverage China’s abundant clinical resources to accelerate validation. On the regulatory front, we will work with drug regulatory agencies from various countries to drive the introduction of innovative policies."

The Blue Book on the Development and Regulatory Interpretation of China’s Cell and Gene Therapy Industry was released for the first time at the conference, providing an authoritative reference for dialogue between the industry and regulators. "The CGT industry is currently in a phase of clinical exploration and regulatory framework construction, while also moving toward manufacturing innovation and exploring payment models," said Li Datao, Founder of Arterial Network (VBDATA). "We believe that in such a complex field that heavily relies on systemic support, a scientific and agile regulatory system is crucial. We hope that this Blue Book will help gather consensus and voices from the industry, and promote faster and better iteration of regulatory policies."

The conference also featured two dedicated project roadshows, where participating companies showcased their cutting-edge achievements and breakthroughs in various sub‑fields. The 21 projects covered gene therapy, gene editing, immune cell therapy, stem cell therapy, and nucleic acid drugs, creating an efficient bridge between innovative companies and capital players focused on this sector. Multiple types of investment institutions and financial organizations, including Hankang Capital, Taikang Investment, Fortune Capital, Cathay Capital, Shengjing Jiacheng Ventures, Sino‑US Green Fund, CASSTAR, Buchang Asset Management Center, Beijing Zhongguancun Bank, and Yuansi Capital, participated in matchmaking activities to help companies expand financing channels and accelerate their international expansion.

Today, cell and gene therapy has become an important treatment modality on par with small molecules, antibodies, and protein therapeutics. It is reported that there are approximately 2,000 CGT clinical trials worldwide, with China accounting for more than 50% of them—and in the field of cell therapy specifically, China’s share exceeds 60%. Notably, China’s IIT (Investigator‑Initiated Trial) pathway is very active, representing about 50% of all clinical trials. In cell therapy alone, the number of IITs in China has surpassed 500, on par with the United States.

Looking ahead, the CSGCT will continue to build an open, authoritative, and efficient global dialogue mechanism, helping China’s cell and gene therapy industry deeply engage in global innovation and standard‑setting, and contributing Chinese wisdom and solutions to the advancement of human health.

The main forum on the morning of September 12, themed “Global Market, China’s Power,” invited leading domestic and international experts to share their insights.

Among them, Professor Richard Jude Samulski, one of the founding figures in the field of AAV gene therapy, delivered a virtual keynote speech titled “Gene Therapy: The Evolutionary Path of a Revolution.” In his address, Professor Samulski reviewed the development of adeno-associated virus (AAV) as a gene therapy vector since 1978.

At the same time, he acknowledged that although AAV vectors still face challenges in clinical applications—particularly in cross‑species differences in transduction efficiency and potential toxicity—significant global progress has been made in the field since AAV was first discovered in 1965, offering new therapeutic modalities for modern medicine. To date, 48 related products have been approved, fully demonstrating the immense potential and real‑world value of gene therapy.

Professor Samulski also specifically noted that his laboratory has trained many Chinese scholars, who, after returning to China, have actively engaged in research and entrepreneurship, thereby driving the rapid development of the gene therapy field in China.

Mark A. Kay, head of the Human Gene Therapy division at Stanford University and professor of pediatrics and genetics, shared research findings on the AAV transduction mechanism and its variability among individuals. He emphasized that understanding the molecular mechanisms of AAV capsid–host interactions will help develop more efficient and safer gene therapy vectors, and may also optimize therapeutic outcomes through small‑molecule drugs or genetic screening approaches.

Winston X. Yan, co‑founder of Arbor Biotechnologies, shared the latest advances in gene editing. Gene editing offers advantages such as one‑time cures, precise correction of mutations, and platform scalability, making it an important direction for future genetic disease treatment. China's strength in investigator‑initiated trials (IITs) provides a valuable opportunity for rapidly validating new technologies. He called for global cooperation, especially in the field of rare diseases, to break down national boundaries and enable the sharing of patient resources and interoperability of clinical data.

Frank L. Graham, discoverer of the HEK293 cell line and Professor Emeritus at McMaster University, reviewed the history and impact of this cell line. HEK293 cells have played an irreplaceable role in gene therapy, viral vector production, protein expression, and particularly in the production of adenoviral and AAV vectors. He noted that although he originally developed HEK293 to study the mechanism of adenovirus transformation, he never expected it to become one of the core tools in global biopharmaceutical R&D, for which he feels immensely gratified and proud.

In the roundtable discussion titled "China's CGT Ecological Practice and Global Competitiveness Building," guests including Fu Sheng, Director of the Administration of Hainan Boao Lecheng International Medical Tourism Pilot Zone; Li Ning, Vice President of the Cancer Hospital, Chinese Academy of Medical Sciences; Li Zonghai, Founder of CARsgen Therapeutics; and He Ting, CEO of Imunopharm, engaged in an in‑depth dialogue to jointly explore new strategies for the global development of China's CGT industry.

Xiao Xiao, Co‑founder, Chairman, and Chief Scientist of Believa Medicine, pointed out that the core innovation in gene therapy lies in the delivery system, particularly the tissue‑targeting optimization of AAV capsids.He emphasized that China has achieved a transition from following to running alongside global leaders in gene therapy, but still faces challenges in the commercialization stage, especially regarding payment systems. Li Zonghai shared his experience of transitioning from gene therapy to cell therapy, stressing that China is already globally competitive in areas such as CAR‑T. He Ting added that the close connection between innovation in the CGT field and universities and research institutes is a unique advantage. The proportion of scientists starting businesses in China is much higher than in other fields, which provides a sustained impetus for innovation in the industry.

Experts at the conference noted that the dual‑track system (IIT + IND) is not unique to China; it is also implemented in Europe, Japan, and elsewhere. China's advantage lies in its high clinical execution efficiency, which is key to the rapid development of China's CGT industry. In particular, the Boao Lecheng Pilot Zone, through legislative guarantees and strict screening of companies and medical teams, provides a "fast track" for the rapid translation of CGT products.

As an emerging direction, in vivo CAR‑T technology has attracted significant attention due to its advantages, such as eliminating the need for ex vivo modification and offering convenient treatment.

Hamideh Parhiz, Co‑founder of Capstan, and Lai Caida, Co‑founder and CEO of Metis Pharmaceuticals, shared the technical principles and development progress of in vivo CAR‑T from international and local perspectives, respectively.

Zhang Dan, Co‑founder and Chief Strategy Officer of Quanlin Pharma, and Co‑founder and Co‑Chairman of Pubsin Bio, emphasized in his speech that China is already globally competitive in the field of cell and gene therapy. In particular, data from investigator‑initiated trials (IITs) in China are gradually being recognized by international regulatory agencies. He called on Chinese companies to focus on the global market, fully leverage China's advantage of rapid clinical advancement, and align with international regulatory standards to achieve "leapfrog development."

In vivo CAR‑T represents not only a revolution in delivery technology but also an upgrade in molecular design. Yang Hanshuo, Founder of Zhenyu Biotech and Professor at West China Hospital of Sichuan University, pointed out that different CAR structures (chimeric antigen receptor architectures) should be designed for different indications. For example, safety and efficacy are equally important for hematological malignancies; efficacy takes priority for solid tumors; and safety is paramount for autoimmune diseases.

Mei Heng, Chief Physician and Professor in the Department of Hematology at Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, noted that compared to ex vivo CAR‑T, in vivo CAR‑T offers advantages such as higher accessibility, lower cost, and shorter waiting times, making it particularly suitable for patients with rapidly progressing diseases. However, he also emphasized that in vivo CAR‑T still faces challenges including safety, immunogenicity, dosing design, and long‑term follow‑up, which must be carefully considered in clinical trial design.

Song Yuqin, Vice President of Peking University Cancer Hospital, shared practical experience in IIT research from the perspectives of clinical management and regulation. She stressed that IIT research must ensure data quality and ethical compliance in order to gain international recognition.

Many experts at the conference believe that in vivo CAR‑T represents the future direction of cell therapy, but it is still in its early stages. The year 2025 will be critical for data readouts, and different technological pathways will gradually diverge. Chinese companies should leverage their clinical efficiency advantages while strengthening original innovation and international cooperation, striving for a dominant position in the global market.

An advanced therapy forum held during the conference brought together leading experts from research institutions, medical centers, innovative biopharma companies, and patient organizations. Focusing on areas such as central nervous system diseases, rare diseases, and cardiometabolic disorders, participants engaged in in‑depth discussions on the R&D progress, technical bottlenecks, and clinical translation pathways of advanced therapies. Multiple high‑level presentations and roundtable discussions showed that next‑generation biotechnologies, represented by gene therapy, are continuously expanding their application boundaries, bringing hope to previously intractable diseases.

Xiao Bin, Co‑founder and Head of Science at Uni‑Pioneers BioMed Inc., focused on the core of gene therapy—the design and optimization of delivery vectors. His team has innovatively modified AAV vectors to significantly enhance their binding affinity and specificity to target cells.

The sessions were rich in substance, showcasing the "China speed" and global vision of gene therapy as it moves from the lab to the clinic and from concept to product. Xiao Xiao shared the complete journey of "Xinjiuning" (Bopendakibine injection), the first gene therapy drug for hemophilia B approved in China, from R&D to market launch. Xiao Xiao acknowledged that the gene therapy industry is currently in a "glacial" low period, but he firmly believes that spring will come as long as we persist. He reviewed the development of gene therapy vectors, noting that AAV has become mainstream due to its natural ability to infect cells, but immunogenicity and production costs remain common challenges for the industry.

Dong Biao, Founder of Zhishan Weixin, further pointed out that although China started late in gene therapy, it has developed rapidly. Currently, nearly 30 institutions in China have the capability to develop rAAV gene drugs.

Looking ahead, many speakers candidly acknowledged ongoing commercialization challenges. Despite the remarkable efficacy of these products, if a single‑dose treatment is priced above one million yuan, how to improve patient accessibility through payment mechanisms such as medical insurance is an urgent issue for the industry.

During a roundtable discussion, the panel reached a consensus: the future of gene therapy lies not only in rare diseases but will also expand into Alzheimer’s disease, other age‑related conditions, and major diseases. Although the road ahead is full of challenges—including technical optimization, cost reduction in manufacturing, and innovative payment models—gene therapy represents a medical revolution with disruptive potential. Only through sustained long‑term investment in innovation and close collaboration among industry, academia, and research institutions can this technology truly benefit more patients.

Small nucleic acid drugs have already become the "third wave" of modern pharmaceuticals, and the related sub‑forum was full of highlights. Experts at the conference pointed out that breakthroughs in extrahepatic delivery technology will greatly expand the therapeutic horizons of small nucleic acid drugs, with cardiovascular, metabolic, and neurological diseases becoming the next major battlegrounds.

Participants in multiple thematic forums, through multi‑dimensional and multi‑level sharing and debate, comprehensively showcased the latest developments and trends in advanced therapies such as gene therapy, cell drugs, small nucleic acids, and exosomes.

The biopharmaceutical industry, including cell and gene therapy, has its own unique characteristics, with high barriers to entry. It cannot be accommodated by just any office building.

During the conference, Xiao Jiayin, Deputy General Manager of Biological Increment at Haixinyu Urban Renewal Group, stated that urban renewal deeply empowers the upgrading of the medical and health industry. By integrating spatial restructuring, technological convergence, and ecological synergy, it builds a full-chain pharmaceutical and health ecosystem covering R&D, translation, production, and services.

Currently, the CGT industry is moving from technological breakthroughs to the critical stage of industrialization and commercialization. Whole-ecosystem collaboration has become the core driving force for the realization of therapies.

At the CGT Product Quality Control and Testing sub‑forum, experts focused on the latest regulatory requirements and technological advances in various countries, emphasizing that strict quality control is the lifeline for product approval and patient safety.

The concurrent "AI + Drug Discovery" forum showcased the immense potential of artificial intelligence in accelerating innovation in target discovery, molecular optimization, and preclinical research. AI is gradually becoming a key tool for improving R&D efficiency and success rates. Speakers included Li Ziqing, Chief Scientist (AI Large Model) at BioMap; Ma Jianzhu, Associate Research Professor and Associate Professor at the Institute for AI Industry Research, Tsinghua University; Ma Lijia, Distinguished Research Fellow and Doctoral Supervisor at Westlake University, and Scientific Founder of Westlake Yunzhi Medicine; Alan Wong (Huang Zhaolin), Associate Professor at the School of Biomedical Sciences, University of Hong Kong; and Lai Caida, Co‑founder of Metis Pharmaceuticals. They shared views on cutting‑edge directions such as AI virtual cells, full‑atom generative models, AI‑assisted vector design for in vivo CAR‑T development, gene editor optimization, and nano‑delivery.

Representatives including Jing Xutian, Partner at Wuyuan Capital; Alan Wong; Li Linxian, Founder of深信生物 (Shenxin Bio); Wang Gang, Founder of Buyang Gene; and Guo Pengbo, R&D VP at Xingchen Zhiyao, discussed the future of cell and gene therapy driven by AI. Delivery systems are widely recognized as both a technical bottleneck and a breakthrough focus in the CGT field. Many corporate representatives agreed that achieving the "critical leap" from lab to clinic requires interdisciplinary collaboration and continuous process innovation.

It is worth noting that commercial manufacturing capability is a core guarantee for the successful launch of CGT products. A robust process and production system must be established to lay the foundation for the scale‑up and standardization of cell therapy products.

Investment, financing, and industrial cooperation are important engines driving CGT from R&D to market. Participants noted that early‑stage technological barriers and clinical differentiation have become key focal points for capital. Investors are more rational and favor companies with genuine global innovation capabilities. With the deep integration of government‑guided funds and corporate venture capital (CVC), full‑cycle support is being provided to CGT enterprises, from R&D assistance to industrial implementation.

In the discussion titled "From a Global Perspective to China: How Innovative Assets Become BD Targets," heads of external innovation from several multinational companies stated that China‑originated innovative drug assets are attracting increasing global attention. Products with high clinical value and a clear IP strategy are more likely to gain favor from multinational pharmaceutical companies.

More participants from the financial and legal sectors analyzed professional services and risk management in BD transactions and financing processes from the perspectives of law, investment banking, and consulting, emphasizing that a mature professional intermediary ecosystem acts as a "catalyst" for the marriage of capital and industry.

Multiple high‑level technical sharing sessions and strategic dialogues comprehensively reviewed the current status and challenges of the CGT industry in quality control, manufacturing processes, delivery technologies, capital cooperation, and commercialization.

A consensus emerged: China's CGT industry is undergoing a critical transformation from scientific innovation to industrial maturity. Only through close collaboration across the entire ecosystem of "industry, academia, research, medicine, capital, and policy" can bottlenecks be broken, allowing more innovative therapies to truly benefit patients worldwide.