—— Adhering to industry orientation and strengthening policy guarantees. Innovative institutions and mechanisms to promote innovative policy sources and industrial development; We will improve the policy guarantee system, promote the organic integration of effective markets and productive governments, accelerate the gathering of innovative factors such as talent, technology and capital, and foster a sound industrial development environment.

Ⅲ. Main objective

By 2025, Shanghai will further enhance its ability of gene therapy scientific and technological innovation, clinical research and translational application, further improve its innovation system, and further optimize its industrial ecology, so as to become an important source of gene therapy scientific and technological innovation and provide new impetus for high-quality industrial development.

—— Continuous improvement in our ability to innovate in policy sources.Build and upgrade more than 20 gene therapy related innovation bases and platforms (key laboratories, technology innovation centers, etc.), basically form an independent and controllable gene therapy technology innovation system; A number of innovative achievements have emerged, and a number of key core technologies with independent intellectual property rights have been formed in fields such as gene editing and drug delivery.

—— The ability of clinical translation continues to increase.More than 5 clinical medical research centers and demonstration research wards in gene therapy and related fields should be built; To explore the construction of a special research hospital for gene therapy; More than 5 new clinical trials were approved.

—— Industrial vitality continues to rise.Building public service platforms for gene therapy research and development, manufacturing, testing, pilot testing and production; More than 15 leading and backbone enterprises of gene therapy industry were introduced and cultivated; One or two innovative products have been applied for marketing.

Ⅳ. Major Tasks

(1) We will enhance our capacity for innovation

1.1. Carry out the prospective layout of basic research

To strengthen the basic research of disease genetics and pathogenesis, gene editing, drug delivery and other fields, explore new targets of gene therapy, explore new pathways of gene expression and regulation, study new strategies of targeted delivery, develop new methods of gene therapy for complex polygenic diseases such as tumors, nervous system diseases, cardiovascular diseases, and consolidate the scientific foundation of gene therapy drug innovation.

2. Carry out research on key core technologies

We will strengthen research in the fields of nucleic acid sequence design and synthesis, gene editing and other new tools for gene manipulation, and new vectors for drug delivery, promote the cross-integration of cutting-edge technologies such as artificial intelligence and synthetic biology with gene therapy related technologies, break through core key technologies, build an independent and controllable innovation system for gene therapy technology, and support high-quality development of the industry.

3. Carry out independent development of key equipment materials

To strengthen the research and development of core equipment such as tangential flow filtration system, liposome high-pressure homogenizer, and disposable bioreactor, key raw materials and materials such as virus, nuclease, and small guide RNA, and key consumables such as serum-free medium and filter membrane, so as to promote the independent control of gene therapy research and development and production supply chain.

(2) improving the translational capacity of clinical research

4. Strengthen the capacity building of clinical research

According to the international first-class standards, a number of clinical medical research centers in gene therapy and related fields should be built to accelerate clinical research and translation. To explore the construction of characteristic research hospitals and demonstration research wards for gene therapy, and promote clinical trials and product development of gene therapy drugs. The special disease cohorts of cancer, nervous system diseases, cardiovascular diseases and other major diseases were established, and the cohort open and sharing platform was constructed to promote the development of gene therapy products and technology research. Medical institutions should be encouraged to formulate relevant rules and regulations for standardized clinical research.

5. Encourage medical institutions to carry out clinical research

Encourage medical institutions in this city to undertake clinical trials of new drugs of local enterprises in the field of gene therapy and independently carry out investigator-initiated clinical trials (IIT) in accordance with relevant regulations. After the completion of relevant research work, according to the number of participants in the project, medical institutions will be given funding according to the standard of 50,000 yuan per person, and the total amount of funding for each project will not exceed 500,000 yuan. Each medical institution's cumulative subsidy should not exceed 5 million yuan per year.

6. Optimize ethical review and human genetic resources approval services

To play the role of the municipal hospital ethics committee alliance, study and formulate working rules and standards such as rapid ethical review, and establish a mutual recognition mechanism for ethical review results of municipal hospitals; Give full play to the role of Shanghai Medical Ethics Expert Committee and Shanghai Clinical Research Ethics Committee to organize and carry out research, consultation, service and training related to ethics in the field of gene therapy, and improve the ethical review capacity of medical institutions. To give full play to the role of Shanghai Pudong Human Genetic Resources Management Service Station, strengthen the publicity and implementation of the Implementation Rules of the Regulations on the Management of Human Genetic Resources, promote gene therapy related enterprises to strengthen the construction of human genetic resources specialists, and improve the quality of service.

(3) Improving public service capacity

7. Strengthen the construction of scientific and technological innovation base system

The establishment of Shanghai Frontier Biotechnology Research Institute, strengthening frontier biological basic research and technical research, and exploring new research paradigms such as practical artificial intelligence empowerment and Trial Ready Cohort (TRC); To build innovation bases such as key laboratories, technology innovation centers and enterprise technology centers in the field of gene therapy, and to improve the ability of innovation sources and the level of transformation and application of achievements in gene therapy and other fields.

8. Strengthen the construction of professional incubators and innovation platforms

Building and upgrading a number of high-quality professional incubators to incubate new technologies and new enterprises; To build an intelligent process research and development platform, an automated detection platform, a modular pilot scale up platform, and other functional platforms. To establish a good manufacturing practice (GMP) -compliant functional platform service system for gene therapy and related fields, accelerate proof-of-concept, product testing, and pilot scale up, shorten the R&D, clinical and production cycles, and further optimize the innovation and entrepreneurship ecology.

9. Strengthen industrial development service capacity building

Encourage CROs/CDmos and other market players to build and optimize third-party service platforms such as vector library screening, preparation filling, and product evaluation based on the needs of gene therapy drug enterprises, provide relevant units with services such as virus bank establishment and storage, preparation development and production, and quality evaluation of gene therapy products, and improve industrial service capabilities.

(4) Improving the level of industrial development

10. Strengthen the spatial layout of "1+1" industries

With Pudong Zhangjiang Cell and Gene Industry Park as the core, we will build an international gene industry landmark and create a gathering area for gene therapy technology innovation and industrial development. To build a gene therapy industry chain demonstration area integrating research and development, testing and manufacturing based on Pujiang Gene Future Valley in Minhang City; Combined with the needs of gene therapy industry, the construction of public facilities and comprehensive service system of the industrial base should be strengthened.

11. Optimize the enterprise development ecology

Guide the agglomeration and development of upstream and downstream industrial chain enterprises such as raw materials and reagents, delivery carriers, facilities and equipment, and R&D services, and create a comprehensive and convenient industrial chain supply system; Establishing an industry-university-research-investment alliance; We will accelerate the development of standards for key raw materials, production and preparation, quality testing, storage and transportation, and actively promote their use to improve the standardization level of the industrial chain. Promote the construction of Shanghai innovative biological product quality inspection and testing center, improve the detection ability of related products.

12. Promote the internationalization of enterprises

Under the premise of controllable risks, we will support qualified diversified investment entities to carry out research and development of human cells and gene technology and promote the industrialization process in accordance with relevant laws and regulations, and promote the coordinated development and deep integration of industrial chain and capital chain. To support the development of international exchanges and cooperation, deeply integrate into the international advanced technology and regulatory system, and improve the international development level of gene therapy enterprises.

V. Safeguard Measures

(1) Establishment of "Shanghai Major Project for Gene Therapy Technology Innovation"

In cutting-edge biotechnology fields such as gene therapy, we have organized and implemented innovation projects, promoted basic research, clinical translation and product development, innovated scientific theories and methods, and formed a number of key core technologies and innovative products with independent intellectual property rights.

(2) Speed up product evaluation and application

Give full play to the role of the Yangtze River Delta sub-center of Drug Evaluation and Inspection of the National Medical Products Administration to provide pre-and in-process guidance and services for the registration and application of key gene therapy products under research in this city; To explore the establishment of a scientific research base for gene therapy regulation to provide support for improving the level of product regulation; Encourage the development of real-world research to explore the application of real-world data in the clinical evaluation of gene therapy drugs for rare diseases.

(3) Strengthening the protection and application of intellectual property rights

For the international invention patents in the field of gene therapy that have been applied abroad through PCT and verified by the relevant patent review institutions, 50% of the registration fee, examination fee and other official fees will be funded, no more than 20 000 yuan per case, and no more than 5 countries or regions will be funded for each PCT international application. Support key enterprises to apply for rapid pre-examination service of Shanghai Intellectual Property Protection Center, improve the quality of patent applications and the speed of review, and enhance enterprises' intellectual property protection and risk response capabilities.

(4) Strengthen fiscal and tax policy support

For gene therapy products that have carried out clinical trials in China and have been produced in this city by local registered enterprises, a maximum of 30 million yuan of financial support will be given, and the annual cumulative support amount of each unit will not exceed 100 million yuan; Promoting gene therapy products in this city to be included in the "list of anti-cancer drugs and rare disease drugs subject to value-added tax policy" to effectively ensure the needs of patients.

(5) Improving insurance support measures

Actively recommend eligible marketed innovative gene therapy products to enter the national medical insurance drug list, and explore multiple payment methods; Supporting the inclusion of gene therapy drugs in rare diseases and other fields in the coverage of people-friendly commercial medical insurance such as "Shanghai Huibao"; Give full play to the role of multi-level commercial medical insurance, and encourage enterprises, commercial insurance companies and medical institutions in this city to explore innovative payment models such as payment by installments and payment by effectiveness; Support medical institutions and enterprises to insure human clinical trial-related liability insurance, and provide financial subsidies of 50% of the premium of eligible subjects, with a subsidy of no more than 500,000 yuan for a single policy.

(6) Increase financial support

We will coordinate the use of government guidance funds and equity investment funds in the biomedical industry to guide social capital to increase support for gene therapy enterprises in their infancy and growth periods. For gene therapy enterprises that have successfully introduced equity financing of more than 50 million yuan (based on the actual amount in place), no more than 2% of the actual equity financing will be funded, and no more than 10 million yuan per enterprise per year; Encourage the launch of more financial service products in Shanghai banks, and support the application of "new drug loan" in the field of gene therapy; We will deepen the "Light of Pujiang" initiative and support gene therapy-related enterprises to list on both domestic and foreign capital markets such as the Science and Technology Innovation Board. Strengthen the listing services of securities firms and other financial institutions for high-quality gene therapy enterprises; To promote overseas listed technology companies such as Youxiu Gene Therapy to return to the Shanghai Science and Technology Innovation Board.

(7) Strengthen the training of innovative talents

Continue to play the "sea of talents" brand influence, vigorously introduce high-end talents; Implement the evaluation project of high-end technical talents in gene therapy industry, and recommend key institutions in the field of gene therapy to be included in the list of key institutions for talent introduction. Increase the support for gene therapy related talents from Qimingxing, academic/technical leaders, and industry elites. In the field of gene therapy, a pilot engineering master and doctor training program has been set up 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 postdoctoral research workstations or settle in postdoctoral innovation practice bases, support postdoctoral personnel to apply for Shanghai "Super postdoctoral" projects, and cultivate scientific research talents that meet the needs of industrial development. Relying on the relevant industrial platforms in the field of gene therapy, we should strengthen talent training and improve the construction of professional and technical personnel in enterprises.

(8) Promoting customs clearance facilitation for research and development goods and special goods

Promote the pilot work of importing research and development items in the field of gene therapy, and support related enterprises and research and development items to be included in the "white list"; We will promote the pilot work of the inbound and outbound joint supervision mechanism for special goods in the field of gene therapy, support key enterprises to apply for the pilot units of the joint supervision mechanism, 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.

Source: Shanghai People's Government