$1.5 Million Awarded for Oxford Research to Develop Brain-Targeted, Systemically-Administered Exosomes That Will Cross Blood-Brain Barrier to Deliver Gene-Silencing Drugs to Inhibit Expression of Mutant Huntington’s Disease Gene in Brain

Matthew Wood (photo), Ph.D., Professor of Neuroscience at the University of Oxford, has been granted an award of £1,008,110 ($1,517,155) from the UK’s Medical Research Council (MRC) for a three-year research project (September 2015-August 2018) intended to develop an systemically-administered, exosome-based, gene-silencing therapy for Huntington’s disease (HD). In the project description (see link below), it is noted that, although therapeutic compounds are being actively developed to treat HD, the delivery of these drugs into the brain is a major impediment to the successful development of these candidate drugs into effective treatments. Consequently, in the newly funded project, Dr. Wood and his Oxford team intend to develop what they term “an entirely new solution” to this major problem. Over the course of 36 months (September 2015 to August 2018), the researchers will attempt to develop a new treatment that can switch off the mutant huntingtin (HTT) gene, and that can successfully cross the blood-brain barrier (BBB) to enter the brain using small, natural sub-cellular particles called exosomes. Exosomes are fat-encapsulated, sub-cellular particles (vesicles) that are generated naturally by all cells of the body and that Dr. Wood and his team have previously exploited for the delivery of drugs into the brain by modifying the exosomes in such a way that they display small molecules on their surface that allow them to home into the brain following their injection into a vein in the body. Perfecting this systemically-administered, exosome-based, brain-targeted drug delivery approach will open the door to testing many compounds that have been proven potentially capable of reducing the levels of the mutant HTT protein that is coded for by the mutant HTT gene in HD.
Login Or Register To Read Full Story