Source: Shanghai Science and Technology Commission

In order to promote the coordinated development of gene therapy and cell therapy, and accelerate the creation of a "nuclear explosive point" and a "cluster area" for biomedical science and technology innovation and industrial development, the "Action Plan of Shanghai to Promote gene therapy science and Technology Innovation and Industrial Development (2023-2025)" (hereinafter referred to as the "Action Plan") has been officially issued recently.

Cell and gene therapy is an important "new track" for global technology and industry competition. Gene therapy refers to the method of introducing foreign genes or gene editing tools into target cells to treat diseases caused by gene abnormalities (usually gene deletion or gene mutation). It has the characteristics of good druggability, strong specificity and remarkable curative effect. Shanghai is one of the innovation highlands of China's gene therapy industry, which has strong advantages in the research and development of gene therapy products such as viral vectors and nucleic acids, and has a good foundation of innovation sources and a strong enterprise innovation strength. As of the end of June this year, 79 gene therapy drugs in China have entered the clinical trial stage, including 19 in Shanghai, the number of which ranks among the top in the country.

In order to promote the coordinated development of gene therapy and cell therapy, the Action Plan of Shanghai to Promote Technological Innovation and Industrial Development of Gene Therapy (2023-2025) was officially issued. The "Action Plan" inherits the "Shanghai Action Plan for Promoting Cell Therapy Technological Innovation and Industrial Development (2022-2024)" issued in November 2022, centering on Shanghai's gene therapy innovation chain, industrial chain, capital chain and talent chain, adhering to the source of innovation, strengthening the strategic layout, adhering to the whole chain development, and strengthening the four-chain integration. Adhere to the principle of industry orientation and strengthening policy guarantee, systematically plan the next three years in our city to promote gene therapy technological innovation and industrial development path, condense key tasks, formulate policies and measures, and promote the high-quality development of gene therapy industry.

The goals set by the Action Plan include: By 2025, Shanghai gene therapy technology innovation source capacity will be further enhanced, clinical research and transformation application capacity will be further improved, innovation system will be further improved, and industrial ecology will be further optimized; To build more than 5 clinical medical research centers and demonstration research wards in gene therapy and related fields; Introduce and cultivate more than 15 leading and backbone enterprises in the gene therapy industry; 1-2 innovative products applied for listing.

The Action Plan contains 12 major tasks and 8 safeguard measures in 4 aspects, aiming to enhance the innovation source and clinical research transformation capacity in the field of gene therapy, and improve the accessibility of gene therapy products and the level of industrial development.

1. Strengthen the ability to innovate in the field of gene therapy

Breakthroughs in underlying technologies such as gene editing tools and delivery carriers are the core key technologies for gene therapy product development. The Action Plan establishes a major project for Gene therapy scientific and technological innovation in Shanghai, establishes the Shanghai Institute of Frontier Biotechnology, explores new research paradigms such as artificial intelligence enabling and Clinical Preparation Cohort (TRC), and strengthen basic research, technical research and equipment and material development in the field of gene therapy. Promote gene therapy product development.

2. Enhance the enabling effect of high-level clinical research

As a disruptive technology that directly manipulates genes, gene therapy places new demands on clinical research facilities, research capacity, and ethical review mechanisms. The "action Plan" will explore the construction of gene therapy characteristic research hospitals and demonstration research wards, and promote gene therapy drug clinical trials and product development; Increase the support for the municipal medical institutions to undertake new drug clinical trials and investigator-initiated clinical research (IIT) and other projects, according to the standard of 50,000 yuan per person to give preferential medical institutions funding, the total amount of funding for each project is not more than 500,000 yuan, and the annual cumulative funding for each medical institution is not more than 5 million yuan; To study and formulate working rules and standards such as rapid ethical review, and establish a mutual recognition mechanism of municipal hospital ethical review results; To organize and carry out ethical research, consultation, service and training in the field of gene therapy, and improve the ethical review capacity of medical institutions.

3. Promote the construction of public service platforms

Gene therapy products have strong technological innovation, large initial investment, and a service platform with engineering research and development production capacity can dreg the fast track of two-way links between basic research, applied research and industrialization, and promote the transformation of innovative achievements. The "action Plan" will build and enhance a number of high-quality professional incubators, establish an engineered functional platform service system for gene therapy and related fields in line with production quality management practices (GMP), accelerate proof of concept, product testing and pilot scale up, and shorten research and development, clinical and production cycles.

4, Optimize the enterprise development ecology

Shanghai has gathered a group of innovative enterprises focusing on gene therapy innovative product research and development, raw material research and development and production, and service outsourcing, basically covering all links of the gene therapy industry chain, and initially forming the Shanghai gene therapy industry ecology. The Action Plan will guide upstream and downstream industrial chain enterprises such as raw materials and reagents, delivery carriers, facilities and equipment, and research and development services to gather and develop, and create a comprehensive and convenient industrial chain supply system; Strengthen the spatial layout of industries such as Pudong Zhangjiang Cell and Gene Industrial Park and Minhang Pujiang Gene Future Valley, and promote the construction of public facilities and comprehensive service systems for industrial bases.

5. Increase financial support

《行动方案》将给基因治疗产品研发企业带来“真金白银”。对已在国内开展临床试验，由本市注册企业获得上市许可并在本市生产的基因治疗产品，给予最高3000万元的资金支持，每个单位每年累计支持额度不超过1亿元；对成功引入股权融资5000万元人民币（以实际到位金额计算）以上的基因治疗企业，按照实际获得股权融资额给予不超过2%的资助，每个企业每年不超过1000万元。

6. Innovate payment models

The price of gene therapy products is expensive, and the ability of individuals to pay limits the application of gene therapy products to a certain extent. The Action Plan further improves insurance support measures, actively recommends eligible listed gene therapy innovative products to enter the national medical insurance drug catalog, and explores multiple and complex payment methods; Support gene therapy drugs in rare diseases and other fields to be included in the scope of commercial medical insurance such as "Shanghai Huibao"; Give play to the role of multi-level commercial medical insurance, promote the city's enterprises, commercial insurance companies and medical institutions to explore innovative payment models such as installment payment and payment according to curative effect, and improve the accessibility of gene therapy products from multiple dimensions.

7. Strengthen talent education

Scientific and technological competition is talent competition, gene therapy-related professional and technical personnel are an important support for emerging industries. "Action Plan" in the field of gene therapy pilot set up engineering master and doctor training projects to explore a new model of training engineering master and doctor in enterprises; Encourage enterprises and institutions in the field of gene therapy to set up post-doctoral research stations or settle post-doctoral innovation practice bases; Relying on the related industry platform in the field of gene therapy, strengthen personnel training and improve the construction of professional and technical personnel.

8. Improve policy support and service supply

The research and development of gene therapy products can not be separated from the market review and approval, intellectual property protection, goods customs clearance and other measures. The Action Plan will give full play to the role of the Yangtze River Delta Branch Center for Drug Evaluation and Inspection of the State Drug Administration to provide guidance and services in advance for the registration and application of key gene therapy products in the city; Fund registration fees, examination fees and other official fees related to PCT international invention patents in the field of gene therapy, support key enterprises to apply for the Shanghai Intellectual Property Protection Center rapid pre-examination service filing subject, improve the quality of patent applications and review speed; Optimize the approval process of key raw materials and materials related to gene therapy research and development and production, and speed up customs clearance procedures.

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