As a new treatment, cell therapy has shown good results in the treatment of many diseases, especially cancer and genetic diseases. In recent years, driven by technology, policy and market, China's cell therapy industry has shown a trend of vigorous development.

On May 22, as an important part of the 2024 Shanghai International Symposium on Biotechnology and Medicine, from basic research and development to clinically available products at the Forum on Cell Therapy Product Research and Development and Clinical Transformation, a number of domestic and foreign scholars, industry figures and regulatory leaders jointly discussed the latest development trends and technological breakthroughs in the field of cell therapy. The research and development, production, supervision and clinical application of cell therapy products were exchanged to explore the development of cell therapy in China.

The struggle between Innovation, Regulation and Market: the way to risk Control of Cell Therapy products

"We can't do research all day long. When we have achievements, we have to transform them." Gao Shaorong, academician of the Chinese Academy of Sciences and dean of the School of Life Science and Technology of Tongji University, shared the practical application of pluripotent stem cells and regenerative medicine in clinical transformation. "if there is something wrong with the cells in the human body, in theory, as long as pluripotent stem cells are differentiated in a very standardized manner, they may be able to differentiate into the cell types we need." In his view, pluripotent stem cells are ideal seed cells for cell therapy in the future because of their great potential for differentiation. "by using iPS technology, we can do gene editing and obtain high-quality cells to treat Parkinson's disease, diabetes, etc., as well as for drug screening. I think organ-like organs are a better way for us to do this kind of drug screening."

"due to the heterogeneity and technological innovation of cell therapy products, the regulatory challenge is to keep up with the advanced technologies and latest developments in the industry." Zhang Dong, a pharmaceutical assessor of the Changjiang Delta Sub-Center of Drug Evaluation and Inspection of the State Drug Administration, introduced the current status of registration and supervision of cell therapy products. He said that from 2020 to 2025, China's cell and gene market will achieve a compound annual growth rate of 276%. Three of the five CAR-T products approved in China are in Shanghai. "at present, our regulatory focus is to emphasize the concept of risk control, focusing on the sources of risk in five aspects: raw materials, production processes, quality control, stability and packaging systems."

Liu Fen, director of the Shanghai Drug Evaluation and Verification Center, introduced the key points that should be paid attention to in the production and manufacturing process of cell therapy products, especially from the perspective of supervision and verification. "Cell therapy products are going to the international market, but global regulators have different definitions, categories and technical requirements for such products. China is actively exploring the realization of mutual trust in supervision. Liu Fen said, for example, that our country joined the PIC/S (international drug regulatory agency) mainly to establish an international regulatory trust framework to ensure the supply of high-quality, safe and effective cell therapy products. "

Yang Fan, chief operating officer of Shanghai Esar Biotechnology Co., Ltd., is one of the leading companies in the field of cell therapy. Yang Fan, chief operating officer, delivered a keynote speech entitled "Biological potency verification strategy related to mesenchymal stem cell drugs during the third clinical period". The current production process of knee osteoarthritis cell products was introduced, and the possible titer determination methods of MSC products and the problems faced by CGT products were analyzed. The verification strategy is described in detail, and valuable suggestions are put forward for the titer determination of MSC products for colleagues in the field of cell therapy. It can be seen that in the field of cell therapy, the world's leading production and preparation capacity is the core competitiveness of cell enterprises.

Tang Fan, marketing director of Shanghai Golden Inspection Co., Ltd., also mentioned that the current field of cell therapy has put forward new requirements for safety and regulation. Tang Fan said that as far as enterprises are concerned, they also need to establish personalized testing standards and methods according to their personalized products. "in the whole process of drug research and development to clinical application, as product producers, they focus on the effectiveness and innovation of the product. The regulatory authorities must focus their attention on safety and risk. "

From Laboratory to Clinic: a duet of Speed and Safety

At present, with the rapid development of biological cell technology and related industries around the world, contemporary medicine is gradually shifting from the traditional treatment mode to the field of cell therapy.

"Stem cell therapy in China is ushering in a new stage, which depends on who runs fast and who is the first to bring cell therapy products to the hospital application scene, who will have an advantage in this competition." Hao Junwei, vice president of Beijing Xuanwu Hospital of Capital Medical University, said at the forum. 

As a clinician, Hao Junwei is most concerned about the safety of patients, followed by the effectiveness of treatment. He introduced the progress of stem cell therapy in stroke sequelae. "after ischemic stroke, there are some redeemable areas in the brain tissue, what is this part of the rescue?" medication? Cell therapy? This is a scientific question to answer. "

"CGT CDMO industry is an upgraded version of the traditional CMO industry, breaking away from the traditional single business model of technology transfer and customized production." Ding Xiaoling, general manager of Shanghai Shenyaozhi Life Technology Co., Ltd., stressed the importance of CGT CDMO. "what can CGT CDMO do? In a word, it is to help enterprises solve many problems in the development of CGT. It can't be said to be all the problems, but it can help enterprises solve many problems from R & D to production at low cost. " For startups, laboratory production conditions can meet demand at an early stage, but as pipelines advance, the production environment must be transferred to plants that meet GMP standards, she says. CDMO can provide pharmaceutical companies with a variety of process options to avoid high conversion costs caused by technological changes.

"All innovations are carried out to meet clinical needs." Li Zonghai, chairman, CEO and chief scientific officer of Keji Pharmaceutical Holdings Co., Ltd., said, "T cells are a very good tumor killing tool, and the key lies in how to transform and apply it." He stressed the importance of universal CAR-T cell therapy. "Universal CAR-T is a must for military personnel, and it is also the 'battle of Tianwang Mountain' that we have to take down." Li Zonghai joked, "I once said that I retired after doing two things, one is to have a certain proportion of patients cured, and the other is a general-purpose CAR-T." We can do a test in five to seven years to see if I can retire. "