On International Rare Disease Day, Shenzhen Medical Academy of Research and Translation (SMART) and Shenzhen Bay Laboratory (SZBL) participated as co-establishing institutions at the founding ceremony of the Peking Union Medical College Hospital (PUMCH) Institute for Rare Disease Innovation and Development. During this event, SMART and SZBL signed a strategic cooperation agreement with PUMCH to establish a Joint Center for Cutting-Edge Rare Disease Research.
February 29, 2024, signified the 17th International Rare Disease Day. Recent statistics indicate that there are more than 7,000 globally acknowledged rare diseases, impacting 300 million individuals globally, with over 20 million patients suffering from rare diseases in China alone. Currently, only about 1% of rare diseases have effective treatments. The low incidence rates of individual rare diseases make drug development exceedingly difficult compared to common diseases. Furthermore, the inability to achieve the sales volume typical of common disease medications often diminishes pharmaceutical companies' enthusiasm for developing rare disease treatments.
Guided by a mission to address clinical challenges and uphold scientific responsibility, SMART and Shenzhen Bay Laboratory have actively undertaken research initiatives to confront global medical issues. Their work includes pioneering foundational research, translational studies, and new drug development for rare diseases.
To further bridge the gap between basic research and clinical application, SMART and SZBL will work with the PUMCH Institute for Rare Disease Innovation and Development under this strategic cooperation agreement to co-establish the Joint Center for Cutting-Edge Rare Disease Research. Nieng Yan, Founding President of SMART and Director of SZBL, and Shuyang Zhang, President of PUMCH, signed on behalf of the three parties.
As per the agreement, the three organizations will utilize their individual strengths in fundamental research and clinical medicine. Their goal is to synchronize with the multidisciplinary and integrative developmental requirements and trends within the rare disease sector. Their collaboration will involve setting strategic research directions in basic rare disease studies, innovating talent training programs, and jointly investing technological resources to accelerate the translation of research findings into practical applications. United, they aim to establish a location for diagnosing and treating rare diseases, a hub for researching rare conditions, a center for developing medications, and a premier zone for ensuring drug accessibility and safety. Their aim is to facilitate prompt diagnosis and treatment for patients with rare diseases and enhance their quality of life.
During the event, Professor Nieng Yan delivered a keynote address titled "From Rare Diseases to Basic Research," providing an engaging and in-depth overview of her team’s research on the structure of proteins that investigates cholesterol levels within cells.