I can walk out of my house alone at night now, even if it's far away, I can still see the words on the store signs! "Xiao Yi, who is a patient with congenital melanoma (LCA) and also a beneficiary of China's first clinical study on gene therapy for LCA, said. It was a group of ophthalmologists from Shanghai First People's Hospital who helped her. Up to now, dozens of patients have completed phase III clinical trials, and the follow-up data is very good. The vision of LCA patients has improved to varying degrees Professor Sun Xiaodong, the head of China's first clinical study on LCA gene therapy and director of ophthalmology at Shanghai First People's Hospital, revealed that after all enrolled patients complete a one-year follow-up, they can submit a new drug application. The Third Plenary Session of the 20th Central Committee of the Communist Party of China called for deepening the reform of the medical and health system, and improving the mechanism for supporting the development of innovative drugs and medical devices. Recently, the Shanghai Drug Administration, Shanghai Health Commission, and Shanghai Science and Technology Commission issued the "Implementation Plan for Improving the Quality of Clinical Trials in the City to Assist in the Development and Marketing of Innovative Drug Devices", which is a positive response to this major deployment. In China, there are tens of thousands of patients like Xiaoyi. Hereditary retinopathy is a major disease with nearly 300 known pathogenic genes, accounting for 20% of the world's blind population. Due to its heritability, it is also known as the 'nightmare' of the family. "Sun Xiaodong introduced that gene therapy is an opportunity to leverage genetic eye diseases to regain sight. In order to encourage more doctors to start from clinical issues and actively engage in scientific exploration and research, Shanghai First Hospital has established the first batch of clinical research centers in Shanghai and set up an Innovation Achievement Transformation Office. Clinical doctors are closer to the scientific challenges that have not yet been overcome in clinical practice, and have a better understanding of unmet clinical needs Since 2013, Sun Xiaodong has established a joint research team for the diagnosis and treatment of hereditary eye diseases, and has been fully committed to the research of type 2 LCA gene therapy based on RPE65 gene lesions. Soon after, achievements began to emerge, but medical scientists holding patents encountered a new problem: how to truly transform scientific research results into "patient dividends"? In 2022, the Municipal Health Commission, in collaboration with the Municipal Science and Technology Commission and other departments, will formulate the "Shanghai Operation Rules for Promoting the Transformation of Scientific and Technological Achievements in Medical and Health Institutions (Trial)", and introduce supporting policies to address difficulties such as pricing and investment of achievements, cash rewards for transformation, management of state-owned assets, and pilot reforms for empowerment. In 2023, the pilot work of Shanghai's scientific and technological achievements transformation and innovation reform will be promoted accordingly. Among the pilot list of medical and health institutions in the city, the First Hospital of the city is prominently listed. To bridge the "last mile" of scientific research achievement transformation, a hospital in the city has chosen to start from the "first 100 meters" of the source of scientific research achievement transformation. Sun Xiaodong introduced that the National Eye Disease Clinical Medical Research Center of the hospital has established GMP pilot workshops, P2 laboratories, SPF level animal experimental platforms, and is equipped with experimental technology systems including metabolomics, cell biology, molecular biology, immunology, and epigenetics. The GMP pilot plant is a specialized facility used to scale up laboratory research results and conduct preliminary industrial production. It is not only a transitional production site, but also an early and critical step in the transformation of laboratory products into usable drugs Sun Xiaodong said that in the past, if medical scientists wanted to take this step, they could only passively wait for their results to be "selected" by enterprises. Now, hospitals have moved the threshold forward and encouraged doctors to complete clinical verification of the preliminary safety and potential effectiveness of research in the hospital. In this way, the market value of the results transformation is more easily highlighted and more likely to be favored by enterprises and investors. At the same time, a hospital in the city has also established an assessment system guided by clinical research. In addition to key clinical skills, patents, achievement transformation, etc. can be used for professional title promotion and assessment, further stimulating doctors' enthusiasm for scientific research and innovation. With the implementation of a package of measures, in the past five years, the scientific and technological achievements of the first hospital in the city have surged, with a total of 30 medical research achievements successfully transformed and a contract amount exceeding 600 million yuan. Among them, the single transaction conversion amount was 30 million yuan, ranking first in Shanghai. In the future, this hospital has many new deployments around pharmaceutical and medical innovation. The total area of the second phase expansion project of the South Campus of City One Hospital is about 159000 square meters, including about 40000 square meters of scientific research land. The hospital stated that it will further enhance the functions of the Clinical Translational Research Center and explore new mechanisms for the combination of innovative clinical medicine and industry university research. (New Society)