Pre-Conference Day

From focused ultrasound and BBB shuttles to intrathecal devices, gene therapy vectors and emerging non-invasive approaches, the field is generating more options than ever before for delivering therapeutics to the brain. Yet despite exciting preclinical progress, questions remain around long-term safety, chronic administration, operational feasibility, cost, scalability and ultimately whether these technologies can realistically be deployed at scale for large CNS indications.

This Workshop Will Gather Experts To:

• Evaluate the strengths, limitations and clinical readiness of leading CNS delivery approaches including BBB shuttles, focused ultrasound, intrathecal and implantable delivery systems, vector-based technologies and emerging non-invasive strategies
• Define the evidence required to convince pharma leadership, regulators, physicians and payers that novel brain delivery technologies can be safely and effectively implemented in routine clinical practice
• Explore the practical challenges of scaling CNS delivery platforms for chronic indications including patient burden, repeat dosing, manufacturing, site infrastructure, reimbursement and commercialization considerations
• Develop a framework for determining which delivery technologies are most appropriate for different therapeutic modalities, disease settings and target product profiles

As the number of available CNS delivery technologies continues to expand, developers face a growing challenge: determining which approach is best suited to their specific therapeutic goal. While BBB shuttles, intrathecal administration, implantable devices, focused ultrasound, gene therapy vectors and emerging non-invasive approaches each offer unique advantages, their suitability can vary dramatically depending on the target, modality, disease biology and patient population. This interactive workshop will move beyond debates around which technology is ‘best’ to explore how developers can build fit for- purpose delivery strategies that maximize clinical, operational and commercial success.

This Workshop Will Gather Experts To:

• Define how factors including target location, therapeutic modality, required biodistribution and dosing frequency should influence CNS delivery strategy selection
• Compare where systemic delivery, direct CNS administration, device-enabled approaches and BBB modulation technologies offer the greatest advantages across different disease settings
• Explore the trade-offs between efficacy, safety, patient burden, operational complexity and scalability when evaluating delivery options for chronic versus acute indications
• Develop a practical framework for matching delivery technologies to specific program goals, therapeutic modalities and target product profiles