Hong Kong Baptist University Researchers Achieve Preclinical Breakthrough for Osteogenesis Imperfecta Treatment & Receive FDA Orphan Drug Designation

Hong Kong Baptist University Researchers Achieve Preclinical Breakthrough for Osteogenesis Imperfecta Treatment & Receive FDA Orphan Drug Designation

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 prote...

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