Session 1: Delivering at the Molecular Level
Treating diseases of the central nervous system (CNS) remains one of the most challenging frontiers in medicine. Despite a growing understanding of neurological disease mechanisms, novel therapies have experienced a high failure rate. One of the primary reasons is poor drug penetration across the blood-brain barriers, resulting in suboptimal delivery. The need for transformative strategies to overcome these barriers is therefore urgent.
To address these challenges, Medicines Discovery Catapult (MDC) - a UK innovation centre dedicated to supporting drug discovery through collaborative initiatives and providing technology platforms - hosted a national workshop bringing together experts from academia, biotech, pharma, CROs, and patient charities. The objective was to identify challenges to CNS drug delivery and innovative technology-based solutions. The key findings from this cross-sector dialogue will be presented covering critical gaps in model predictivity, limitations of current delivery methods and therapeutic modalities, while outlining prioritised solutions and investment for the future.
Furthermore, as a research and technology organisation, MDC is strategically positioned to enable innovation in neuroscience research. Through MDC-SME partnered case studies, this talk will present two technology areas identified as promising solutions: (1) nanocarriers targeting BBB receptors for gene therapy; and (2) non-invasive focused ultrasound (FUS) for BBB opening for neurological disorders and neuro-oncology, thereby exemplifying how collaborative innovation can reshape the future of CNS therapeutics.