Although the adverse effects posed by the condition are severe and often fatal (as in the case of OI type II and cardiopulmonary complications), the genetic disease is exceedingly rare: OI affects only approximately one in 15,000 to 20,000 people in the general population[1], with types I and IV being the most common[2][3].
Our team has recognised that it is quite difficult to balance the need to cure/alleviate the symptoms caused by OI in affected patients and the economical factors that influence drug production.
As there are only a relatively small number of patients that suffer from this class of diseases, it is extremely unlikely for a pharmaceutical company to turn in a profit from the drug, even if the drug is made available as a much more efficient and "cost-effective" alternative than currently-available treatments/production methods.
To facilitate the development of the ostamer for the treatment of OI patients with a low safety concern on the cardiovascular system, we have applied or and obtained Orphan Drug Designation by the US Food and Drug Administration (DRU-2019-6966).
We have also applied for and obtained the support from Hong Kong Innovation and Technology Funds (ITF).
We further collected the serum of the OI patients for determination of the binding ability between ostamer and serum sclerostin of OI patients.
We also consulted with experts like Prof. Tan Weihong who majors in aptamer and the orthopedist Prof. Chen Lin, who majors in OI, as well as our collaboration industries like WuXi Apptech, Increase Innovation Medicine (IIM), and Aptacure Therapeutics Ltd.
Hong Kong Innovation and Technology Funds (ITF): University-Industry Collaboration Project (UIM298)
To further test the potential of osteoma as a translational medicine for OI patients, we collected the serum of the selected OI patients with different gene mutations (n=2 for WNT1, n=1 for TMEM38B, n=1 for FKBP10 and n=2 for BMP1) and the serum of the healthy controls (n=6) at the same age from SHENZHEN BAOAN People’s hospital.
The sera are used for the determination of the binding affinities between ostamer and serum sclerostin of OI patients.
The serum samples utilized were under the approval of the patients and healthy cases. This study complies with the experimental guidelines of the World Medical Association and the Ethics Committee of Hospital.
Throughout the process, we ensured that we were on the right track through consulting experts from different areas:
IHP1, 2: Professor Zhang BaoXian of Increase Innovation Medicine.
Prof. Zhang Baoxian of Increase Innovation Medicine (IIM) visited our team. During the discussion between our team and President Zhang, we discussed how our agreement should be conducted in accordance with the principle of equity and free will, and common development.
IHP3, 4: Professor Tan WeiHong, academician of the Chinese Academy of Sciences, Distinguished Professor of the University of Florida, Vice President of Hunan University, and Director of the State Key Laboratory of Chemical Biosensing and Metrology (Hunan University).
In January of 2019, Professor Tan visited our team. We introduced the microfluidic aptamer screening project to Prof. Tan, and subsequently raised the adversities we faced during the operation of the system. has given invaluable advice to improve our automatic aptamer screening system based on microfluidic chips, and to refine our models.
[1] Forlino A, Marini JC (April 2016). "Osteogenesis imperfecta". Lancet. 387 (10028): 1657–1671. [2] Shapiro JR, Byers PH, Glorieux FH, Sponseller PD (2014). Osteogenesis imperfecta: a translational approach to brittle bone disease (2nd ed.). [3] Van Dijk FS, Cobben JM, Kariminejad A, Maugeri A, Nikkels PG, van Rijn RR, Pals G (December 2011). "Osteogenesis Imperfecta: A Review with Clinical Examples". Molecular Syndromology. 2 (1): 1–20.
