News

A joint team composed of Professor Zhang Ge, Associate Dean (Research) of Chinese Medicine, Dr. Wang Luyao, Post-Doctoral Research Fellow of the Teaching and Research Division, along with experts from the Shanghai Sixth People's Hospital, has been awarded the First Prize of the 2023 Shanghai Medical Science and Technology Award for the project "Molecular Mechanisms and Clinical Precision Diagnosis and Treatment of Hereditary Bone Diseases." The Shanghai Medical Science and Technology Award, established by the Shanghai Medical Association in 2002, has been dedicated to foster the innovative development of and transformation of achievements in medical science and technology, and to drive the high-quality development of Shanghai’s healthcare industry.

The award-winning project collected a vast number of samples and established a diverse family resource repository of genetic bone diseases (a total of 1919 families, 4939 cases), achieving a molecular diagnostic rate of 59.1%. The clinical phenotype spectrum and pathogenic gene mutation spectrum of major types of hereditary bone diseases in the Chinese populations were delineated, providing strong support for clinical diagnosis, treatment, and research.

Osteogenesis imperfecta is a rare hereditary bone disease with no effective drugs for treatment. The team discovered that the serum sclerostin levels in patients with osteogenesis imperfecta are significantly higher than those in healthy individuals, and that a “loop3 domain” in the core region of sclerostin can serve as a molecular target for inhibiting sclerostin while preserving its cardiovascular protective function. The team further collaborated with the HKBU spin-off company, Aptacure Therapeutics Limited, to develop a nucleic acid aptamer named “Apc001” that specifically targets sclerostin loop3. This aptamer Apc001 promotes bone formation in mice with osteogenesis imperfecta, and has received orphan drug designation and pediatric rare disease designation from the US Food and Drug Administration. Currently, the team plans to submit an Investigational New Drug application for the treatment of osteogenesis imperfecta in both China and the US.

SCM extends our congratulations to the joint team for being one of the seven teams awarded this prestigious honour in 2023, and acknowledges Professor Zhang's achievements in drug discovery and transformation of research outcomes.