Original Title: Policy Interpretation of the “Shanghai Action Plan for Promoting Innovation and Industrial Development in Gene Therapy (2023–2025)” To implement the “14th Five-Year Plan for Building Shanghai into a Globally Influential Center for Science and Technology Innovation” and accelerate the creation of a “breakthrough hub” for biomedical innovation and an “industrial cluster,”with the approval of the Municipal Government, the Municipal Science and Technology Commission, the Municipal Commission of Economy and Information Technology, and the Municipal Health Commission have jointly issued the "Shanghai Action Plan for Promoting Innovation and Industrial Development in Gene Therapy (2023–2025)" (hereinafter referred to as the "Action Plan"). The interpretation is as follows.
I. Background
Cell and gene therapy represent a crucial “new frontier” in global scientific, technological, and industrial competition. Shanghai is one of the most dynamic industrial clusters for cell and gene therapy in China, boasting a solid foundation for innovation and strong corporate innovation capabilities. To seize the window of opportunity for technological innovation and industrial development in cell and gene therapy,the Municipal Science and Technology Commission, the Municipal Commission of Economy and Information Technology, and the Municipal Health Commission have formulated the "Action Plan." Building upon the "Shanghai Action Plan for Promoting Scientific and Technological Innovation and Industrial Development in Cell Therapy (2022–2024)," this initiative aims to strengthen Shanghai’s capacity for innovation in the field of gene therapy, enhance clinical research and translational application capabilities, optimize the industrial ecosystem, and promote the coordinated development of gene therapy and cell therapy, thereby providing support for the construction of a biomedical industry innovation hub with international influence.
II. Development Approach
Centered on Shanghai’s gene therapy innovation chain, industrial chain, capital chain, and talent chain, and focusing on key issues such as basic research, technological innovation, clinical research, public services, industrial development, and supporting measures, this plan adopts a global perspective and holistic approach to systematically outline the city’s vision and objectives for promoting innovation and industrial development in gene therapy over the next three years. It identifies key tasks and policy measures to enhance innovation origination, clinical research translation, and public service capabilities, thereby driving the high-quality development of the gene therapy industry.
III. Main Content
The Action Plan addresses the characteristics of gene therapy—including its broad interdisciplinary scope, high barriers to technological development, numerous challenges in clinical application, and significant potential for industrial growth—by proposing a development strategy that emphasizes both research and development (R&D) and translation, as well as innovation and service. This approach aims to comprehensively enhance Shanghai’s capacity for generating gene therapy innovation and elevate the level of industrial development.The Action Plan comprises 12 major tasks across four areas and eight supporting measures, aimed at strengthening innovation origination and clinical research translation capabilities in the gene therapy field, while improving the accessibility of gene therapy products and elevating the level of industrial development.
First, strengthen the capacity for innovation in the gene therapy field. Underlying technologies such as gene editing tools and delivery vectors are core technologies for the R&D of gene therapy products. The Action Plan establishes the Shanghai Major Special Project for Gene Therapy Science and Technology Innovation and builds the Shanghai Institute of Advanced Biotechnology. It explores and implements new research paradigms such as AI-enabled approaches and Trial Ready Cohorts (TRC), strengthens basic research, technological breakthroughs, and the development of equipment and materials in the gene therapy field, and drives the development of gene therapy products.
Second, the enabling role of high-level clinical research will be enhanced. As a disruptive technology that directly manipulates genes, gene therapy imposes new requirements on clinical research facilities, research capabilities, and ethical review mechanisms.The Action Plan will explore the establishment of specialized research hospitals and model research wards dedicated to gene therapy to facilitate clinical trials of gene therapy drugs and product development; increase support for municipal medical institutions undertaking new drug clinical trials and investigator-initiated trials (IITs), providing funding to eligible institutions at a rate of 50,000 yuan per person, with a total funding cap of 500,000 yuan per project and an annual cumulative funding limit of 5 million yuan per institution;Develop working rules and standards for expedited ethics review, and establish a mechanism for mutual recognition of ethics review results among municipal-level hospitals; organize and carry out research, consultation, services, and training related to ethics in the field of gene therapy to enhance the ethics review capabilities of medical institutions.
Third, promote the development of public service platforms. Gene therapy products are highly innovative and require significant upfront investment; service platforms with engineering-level R&D and production capabilities can facilitate a fast track connecting basic research, applied research, and industrialization, thereby promoting the commercialization of innovative achievements.The Action Plan will focus on building and upgrading a group of high-quality specialized incubators, establishing a service system for engineering-oriented functional platforms in gene therapy and related fields that complies with Good Manufacturing Practice (GMP) standards, accelerating proof-of-concept, product testing, and pilot-scale production, and shortening the R&D, clinical, and production cycles.
Fourth, optimize the business development ecosystem. Shanghai has already attracted a cluster of innovative enterprises specializing in the R&D of gene therapy products, the R&D and production of raw materials, and service outsourcing. These enterprises cover virtually every link in the gene therapy industrial chain, having preliminarily formed Shanghai’s gene therapy industrial ecosystem.The Action Plan will guide the clustering and development of upstream and downstream enterprises in the industrial chain—including raw materials and reagents, delivery vectors, facilities and equipment, and R&D services—to create a comprehensive and convenient industrial supply system; it will strengthen the spatial layout of industrial zones such as the Zhangjiang Cell and Gene Industry Park in Pudong and the Pujiang Gene Future Valley in Minhang, and promote the development of supporting public facilities and comprehensive service systems within these industrial bases.
Fifth, financial support will be strengthened. The Action Plan will provide substantial financial support to gene therapy product R&D enterprises.For gene therapy products that have already undergone clinical trials in China, obtained marketing authorization through a Shanghai-registered enterprise, and are manufactured in Shanghai, financial support of up to 30 million yuan will be provided, with a cumulative annual support limit of no more than 100 million yuan per entity; for gene therapy enterprises that successfully secure equity financing of 50 million yuan or more (calculated based on the actual amount received), a subsidy of up to 2% of the actual equity financing amount will be granted, with an annual limit of no more than 10 million yuan per enterprise.
Sixth, innovative payment models will be introduced. Gene therapy products are expensive, and individuals’ ability to pay has, to some extent, limited their application.The Action Plan further refines insurance support measures, actively recommends eligible innovative gene therapy products for inclusion in the National Medical Insurance Drug Directory, and explores diversified and composite payment methods; it supports the inclusion of gene therapy drugs for rare diseases and other fields in the coverage of public-benefit commercial health insurance programs such as “Shanghai Huibao”; it leverages the role of multi-tiered commercial health insurance to encourage local enterprises, commercial insurance companies, and medical institutions to explore innovative payment models such as installment payments and outcome-based payments, thereby enhancing the accessibility of gene therapy products from multiple dimensions.
Seventh, strengthen talent recruitment and cultivation. Competition in science and technology is fundamentally a competition for talent, and professionals with specialized expertise in gene therapy are a vital pillar of this emerging industry. The Action Plan will pilot engineering master’s and doctoral training programs in the gene therapy sector and explore new models for training these professionals within enterprises; it will encourage enterprises and institutions in the gene therapy sector to establish postdoctoral research stations or join postdoctoral innovation practice bases; and it will leverage relevant industrial platforms in the gene therapy sector to strengthen talent training and improve the development of professional technical teams within enterprises.
Eighth, improve policy safeguards and service provision. The research, development, and market launch of gene therapy products rely on measures such as market approval review and evaluation, intellectual property protection, and customs clearance. The Action Plan will fully leverage the role of the Yangtze River Delta Branch of the Drug Evaluation and Inspection Center under the National Medical Products Administration to provide pre- and during-process guidance and services for the registration applications of key gene therapy products under development in the city;subsidize official fees—such as registration and examination fees—for PCT international invention patents in the gene therapy sector; support key enterprises in applying to become registered entities for the Shanghai Intellectual Property Protection Center’s fast-track preliminary examination service to improve patent application quality and expedite review; and streamline approval processes for key raw materials and supplies used in gene therapy R&D and production, while accelerating customs clearance procedures.
