8:00 am Check-In, Coffee & Light Breakfast
8:50 am Chair’s Opening Remarks
DELIVERING ON THE PROMISE OF LOCAL CNS THERAPIES: CLINICAL EVIDENCE & OPERATIONAL HURDLES
9:00 am Advancing Neurological Therapeutics: Clinical Updates from Biogen’s Neuroscience Pipeline
Synopsis
- Highlights from key trials in Alzheimer’s disease, ALS, MS, and rare neuromuscular disorders
- Leveraging biomarkers to inform trial design and accelerate development
- Patient-centered approaches, adaptive trial designs, and lessons learned
9:30 am Panel Discussion | Advancing In Vitro Models to Better Recapitulate the Blood-Brain Barrier: Future Directions & Innovations to Ensure Translatability
Synopsis
- What are the critical gaps in current in vitro BBB models that hinder their predictive accuracy?
- How can we better integrate multiple cell types, including fibroblasts and immune components, to replicate the BBB’s immune environment?
- How can in vitro models better reflect intracellular trafficking and sorting processes critical to BBB transcytosis?
- When and how should complex models be integrated into screening workflows, and how do we balance physiological relevance with scalability?
- What roles can AI and model engineering play in building more predictive BBB platforms and accelerating novel shuttle discovery?
10:30 am Morning Break & Refreshments
Track 1: Systemic Delivery
Engineering Strategies to Expand CNS Therapeutic Reach via Receptor-Mediated Transcytosis: Optimizing Targets & Cargos
11:30 am Enhancing Alector’s Neurology Pipeline with Alector Brain Carrier (ABC) Platform
Synopsis
- Introduction to Alector ABC platform technology
- Case studies of enhancing brain uptake of multiple drug modalities using anti-TfR ABC
12:00 pm Evaluating the Potential of Alternative Receptors Beyond TfR1 to Enhance CNS Access
Synopsis
- What properties make a receptor suitable for CNS transcytosis?
- How do you evaluate receptor internalization and trafficking?
- Are we reaching the limits of TfR, or is there more to explore?
- What’s needed to validate a “novel” receptor target clinically?
- Couple single-domain antibodies to Fc regions or other antibodies to enhance pharmacokinetics and prolong half-life
Engineering Strategies to Expand CNS Therapeutic Reach via Receptor-Mediated Transcytosis: Optimizing Targets & Cargos
12:30 pm Roundtable Discussion: Achieving Brain Penetrance for Oligonucleotide Therapies: Innovations in TfR1-Mediated Delivery to Improve Efficacy & Cell Specific Targeting
Synopsis
Highly interactive breakout discussions designed to crowdsource solutions, share cross-functional insights, and explore the practical barriers to clinical adoption.
- What bioengineering strategies are improving brain penetrance?
- How are companies benchmarking success – CSF levels, parenchymal penetration, target knockdown?
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open a wider audience debate.
Track 2: Direct & Local Delivery
Advancing Local Brain Delivery: How Focused Ultrasound & CED Improve Therapeutic Biodistribution in the Brain
11:30 am Clinical Proof-of-Concept for CED: Lessons from Protein-Based & AAV Gene Therapies
Synopsis
- Clinical progress in CNS delivery of protein-based (antibodies, albumin-sized) and AAV gene therapies, with a brief overview of liposomal approaches
- Delivery challenges and strategies for effective CNS penetration and therapeutic targeting
- Regulatory considerations and pathways shaping clinical approval
12:00 pm Roundtable Discussion: Breaking the Drug–Device Deadlock: How Can We Incentivize Scalable Convection- Enhanced Delivery in CNS?
Synopsis
- What are the core barriers to integrating device strategy into drug development?
- How can we standardize user-dependent modalities like CED to de-risk clinical trials and scale reliably?
Advancing Local Brain Delivery: How Focused Ultrasound & CED Improve Therapeutic Biodistribution in the Brain
12:30 pm Enhancing Precision & Reliability of Focused Ultrasound-Mediated Blood-Brain Barrier Opening for Translation of Next-Generation CNS Therapeutics
Synopsis
- Introduce novel applications of focused ultrasound-mediated BBB opening for cellular immunotherapy, gene therapy, and radiotheranostic delivery
- Examine technical and biological variables that affect the consistency, safety, and reproducibility of focused ultra-sound-mediated BBB opening
- Consider how delivery outcomes are influenced by therapeutic cargo properties, imaging guidance, and treatment protocols, and what’s needed to support successful clinical translation
- Exploring possibilities for clinical applications of focused ultrasound-mediated delivery to the CNS
1:00 pm Lunch & Networking
Enhancing Translational Fidelity in Systemic CNS Delivery: From In Vitro Models to In Vivo Predictability
2:00 pm Addressing Cell Source & Model Complexity in In Vitro BBB Systems to Enable Physiologically Relevant Models that Accelerate CNS Delivery & Drug Screening
Synopsis
- Evaluate limitations of current cell sources and strategies toimprove BBB model fidelity
- Explore how multi-cellular enhances physiological relevance
- Highlight emerging assays and engineering tools that improvedrug delivery assessment across in vitro BBB models
2:30 pm Roundtable Discussion: Imaging, Tracers & Analytics – Uncovering Tools to Map CNS Penetration and Guide Delivery Strategy
Synopsis
Highly interactive breakout discussions designed to crowdsource solutions, share cross-functional insights, and explore the practical barriers to clinical adoption.
- Compare conventional qPCR and hybridization assays with advanced multimodal imaging, LC-MS, and radiolabelled tracer techniques
- Explore how radiolabelled probes and real-time imaging enable precise quantification of drug delivery across the blood-brain barrier and brain compartments
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open a wider audience debate.
Boosting Predictability & Validation of CNS Drug Delivery through PK/PD Innovations
2:00 pm Fireside Chat: Improving Predictability of CNS Targeting and Distribution Using Machine Learning & AI
Synopsis
- Approaches for developing predictive models (PBPK, compartmental, mechanistic) tailored to enhance CNS delivery
- The role of AI and machine learning in refining model accuracy and extrapolating from limited datasets
- How predictive modelling supports early design decisions, reduces reliance on animal studies, and informs regulatory strategies
2:30 pm Roundtable Discussion: Navigating CSF & ISF Dynamics to Optimize Local CNS Drug Distribution
Synopsis
Highly interactive breakout discussions designed to crowdsource solutions, share cross-functional insights, and explore the practical barriers to clinical adoption.
- Clarify how fluid flow patterns and exchange between CSF and ISF impact drug dispersion, retention, and target engagement
- Discuss variability in CNS fluid dynamics across species and patient populations, and implications for translational modelling
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open a wider audience debate.
3:15 pm Afternoon Break & Networking
FUNDING THE FUTURE: HOW CLINICAL VALIDATION & SCALABILITY DRIVE CNS DELIVERY INVESTMENT
3:45 pm Panel Discussion: How Clinical Confidence & Scalability Shape the Future of CNS Drug Delivery
Synopsis
- Explore how investors evaluate risk and opportunity in a space where strong science must meet real-world viability
- Examine how clinical or translational validation – from receptor engagement to dose modeling – influences investor confidence in CNS delivery platforms
- Discuss how manufacturability, scalability, and delivery route compatibility affect long-term viability and funding decisions
- Understand the importance of mechanism clarity, receptor strategy, and IP positioning in shaping perceived value and freedom to operate