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OSTAMER

Tackling Osteogenesis Imperfecta Without Increasing Cardiovascular Risk

Osteogenesis imperfecta is a group of genetic disorders that results in brittle bones due to improper collagen formation.

In osteoblasts of OI patients, the Wnt signalling pathway is inhibited by sclerostin via binding to LRP5/6 co-receptors, thus lowering β-catenin concentrations, gene expression and bone formation.

Suppressing sclerostin by a sclerostin antibody has been shown to enhance bone mass and bone strength. However, in phase III clinical trials, the antibody also imposed severe cardiac ischemic events.

Ostamer, our therapeutic aptamer, tackles this development via selectively binding and inhibiting loop 3 of sclerostin. This prevents sclerostin from binding to osteoblast receptors for treatment of osteogenesis imperfecta, as well as to preserve sclerostin's secondary role as a cardiovascular regulator.

Our Ostamer has received US FDA Orphan Drug Designation in 2019 in response to successful clinical testing on OI mice.

Team:HKBU

OSTAMER