Peter Horanyi
Senior Principal Scientist UCB
Seminars
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
- Which alternative BBB receptors (e.g. CD98hc, insulin receptor, LRP1, basigin) show the most promise beyond TfR1 for achieving sufficient brain uptake without compromizing safety or peripheral PK?
- How can we optimize receptor affinity, valency, and trafficking profiles to balance efficient transcytosis with minimal lysosomal degradation or target-mediated clearance?
- What preclinical models and translational biomarkers are most predictive for comparing receptor performance and de-risking clinical success across different shuttle platforms?
- How should developers prioritize receptor selection based on modality (e.g. antibodies, enzymes, oligonucleotides) and indication-specific needs for brain distribution and cellular targeting
- Compare direct CNS delivery and systemic administration in terms of tissue exposure and selectivity, and their impact on target-site concentration, dose, and efficacy–toxicity balance
- Assess the limitations of systemic approaches that rely on plasma pharmacokinetics as a surrogate for efficacy versus direct delivery strategies that prioritise disease-site exposure
- Evaluate adoption barriers and translational challenges for direct delivery relative to systemic modalities, including device integration and demonstration of tissue-level pharmacological advantage
- Explore how patient, clinician, and commercial perspectives shape trade-offs between invasiveness and the potential for improved efficacy and reduced toxicity through targeted delivery
