Researchers from the School of Chinese Medicine at Hong Kong Baptist University (HKBU) have successfully developed a novel aptamer for the treatment of osteogenesis imperfecta (OI) with the aid of artificial intelligence technology. This represents the first time that a drug developed in Hong Kong has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).
What is OI?
Also known as “brittle bone disease” it is a rare hereditary bone disease with no effective treatment to date. It affects 6 to 7 people per 100,000 worldwide. It is estimated that 700 to 800 people in Hong Kong have OI. It has been reported that inhibition of sclerostin, a protein that regulates bone formation, can improve the clinical presentation of OI reports QS WowNews.
The Research Team
The Hong Kong-based team included Professor Zhang Ge, Associate Director of HKBU’s Law Sau Fai Institute for Advancing Translational Medicine in Bone & Joint Diseases, and Professor Lyu Aiping, Director of HKBU’s Institute of Integrated Bioinformedicine and Translational Science—strategically screened and optimized a new class of molecule-nucleic acid aptamers against the sclerostin protein, which promotes bone formation in mice with OI. Moreover it doesn’t impact the cardiovascular function of sclerostin and will not increase cardiovascular risk.
What are Aptamers?
Single-stranded DNA or RNA molecules which are regarded as an alternative to antibodies. They can bind to and inhibit specific targets such as sclerostin, with their three-dimensional structures. But a random single-stranded DNA library with more than 1015 sequences, identifying the most optimal molecule for the treatment of OI, is essentially like looking for a needle in a haystack.
The team ultimately applied AI technology to further help identify the optimal molecules.
Currently the team is involved with preclinical research. They will prepare the research details in preparation to “further strengthen our technological edge and develop a more effective treatment strategy for OI in order to benefit patients and society,” reported Professor Lyu.
Professor Zhang Ge, Associate Director of HKBU’s Law Sau Fai Institute for Advancing Translational Medicine in Bone & Joint Diseases
Professor Lyu Aiping, Director of HKBU’s Institute of Integrated Bioinformedicine and Translational Science