IRBM cited in UniQure publication on using HTT-lowering therapies to slow down neurodegeneration in Huntington’s Disease
UniQure has just published a study in Science Translational Medicine using a Huntingtin (HTT)-lowering therapy.…
Earlier this year, we published a paper in Molecular Pharmaceutics that highlights our work with the CHDI Foundation to develop therapeutics that substantially improve the lives of those affected by Huntington’s disease.
It is essential to understand the physiochemical properties that Huntington’s disease drug candidates need to permeate through the blood-brain barrier (BBB). In vitro BBB models that accurately reflect BBB properties will help generate important insights into the neuropharmacokinetics of a compound, accelerating the drug discovery process.
In this study, we utilized an in vitro BBB model to comprehensively evaluate the transport behavior of a series of therapeutic candidates for Huntington’s disease. Our model demonstrated a strong correlation with in vivo permeation, establishing the usefulness of in vitro model BBB models in drug discovery.
Read more here.