Academic Drug Discovery: Examples in Kidney Disease and Muscular Dystrophy

Over the past twenty years, scientists in academia have placed an increased focus on translational science. Rather than competing with drug discovery organizations in pharma or biotech, efforts in academic laboratories can complement those endeavors by playing to the strengths inherent in a university setting, including working on high risk projects, taking advantage of unique expertise in specific areas, and pausing to pursue basic science questions. This Flash Talk will describe two projects that exemplify the unique opportunities as well as challenges in academic drug discovery. We have found that selective inhibition of HDAC8 has the potential to treat multiple kidney disorders including acute kidney injury (AKI) and focal segmental glomerulosclerosis (FSGS). However prototypical inhibitors are likely to be too toxic for AKI or FSGS patients. Therefore, we are focusing on HDAC8 modulators that do not act at this epigenetic target’s active site, and have generated optimized compounds that exhibit efficacy in organoid and in vivomodels.  In a second project focusing on Duchenne muscular dystrophy (DMD), we hypothesize that the protein utrophin, highly expressed during development then gradually replaced with dystrophin, can be “de-repressed” to provide functional protein that can replace the mutated dystrophin in DMD patients. Our early work in this area will be described. Both projects highlight opportunities in academic drug discovery to focus on high-risk targets and areas and to take advantage of the unique expertise in certain disease areas, opportunities that may not be as feasible in pharma or biotech environments. 00:00 Welcome and Introduction 10:20 New DH Features 12:50 Presentation 46:32 Q&A If your company doesn’t have a subscription yet, reach out here to inquire about access: https://drughunter.com/plans Recorded on June 5th 2025, as a Drug Hunter Flash Talk. Sign up to be notified of future sessions here: https://drughunter.com/flash-talk-ser... Sign up for our free newsletter here: https://drughunter.com/newsletter-sig...