Explore the Agenda
8:00 am Check-In, Coffee & Light Breakfast
8:55 am Chair’s Opening Remarks
Turning Brain Delivery Breakthroughs into Sustainable CNS Pipeline Growth
9:00 am Keynote: Why is it so much Harder to Deliver Oligonucleotides to the Brain Compared to Other Organs?
- Exploring that the rate-limiting step for oligonucleotides targeted for CNS indications is release of oligonucleotides from lysosome and brain tissue
- Evaluating novel delivery technologies across LNPs, ultrasound, viral particles and routes of administration in delivering oligonucleotides to the brain without compromising on potency, distribution, affinity or efficacy
- Reviewing the next step to optimizing structural design to ensure delivery to the cytoplasm and nucleus
9:30 am Bispecific TfR1/Target Antibody Shuttles (e.g. Anti-Aβ Trontinemab): Latest Preclinical & Clinical Insights
- Assess how bispecific constructs couple transport and therapeutic domains to enable BBB crossing while maintaining target engagement in the brain parenchyma
- Evaluate dose-dependent saturation of TfR1 transport pathways and its effect on nonlinear brain uptake
- Characterize intrabrain distribution patterns (capillary-associated vs parenchymal delivery) to assess true tissue penetration versus vascular trapping
- Compare clinical biomarker strategies (e.g. PET imaging, CSF biomarkers) for demonstrating CNS target engagement
10:00 am Fireside Chat: Building the Next Generation of CNS Pipelines Through Brain Delivery & Modality Innovation
- Determining which brain delivery technologies and therapeutic modalities warrant integration into long-term neuroscience portfolios
- Establishing evidence thresholds for advancing CNS delivery platforms from discovery-stage innovation into clinical development
- Balancing investments across internal R&D, platform partnerships, licensing opportunities, and external innovation ecosystems
- Identifying the biological, translational, and regulatory advances most likely to unlock the next wave of CNS therapeutic development
10:30 am Speed Networking
A prime chance to make the most of in-person networking and forge new connections as new companies enter, and existing ones broaden their presence within the CNS drug delivery space. Designed to maximize your introduction to numerous new individuals and serve as a catalyst for ongoing discussions during the Summit.
11:00 am Morning Break & Refreshments
Track 1: Technology & Platform Development
Designed for:
Drug delivery, platform, biologics, antibody, oligonucleotide, vector, protein engineering and chemistry teams developing the next generation of CNS delivery technologies.
Explore the engineering, chemistry and design principles underpinning brain shuttles, capsids, conjugates and device-enabled delivery systems to improve BBB transport, brain exposure and delivery efficiency.
Optimizing the Engineering & Chemistry of Antibody Brain Shuttles for BBB Penetrance
11:30 am Challenging Conventional Transferrin Receptor Design Paradigms with Enhanced Brain Delivery™ Bispecific Antibodies
- Demonstrate efficient TfR-mediated transcytosis using a high-affinity, bivalent antibody architecture that achieves robust CNS exposure despite prevailing assumptions in the field
- Present translational mouse and non-human primate data showing substantially enhanced brain penetration while preserving selective binding to pathogenic amyloid beta oligomers
- Explore a differentiated TfR binding epitope that avoids hematological liabilities (anemia) associated with earlier BBB shuttle approaches
- Share drug candidate selection, lead characterization and dose-ranging studies in rodent and non-human primates of Enhanced Brain Delivery™ therapeutics for Alzheimer’s disease
12:00 pm Nanofitins®-based BBB Shuttles for Brain Delivery of Biotherapeutics
- Nanofitins® are small affinity proteins, easy to combine with each other and various therapeutic modalities, for targeted delivery
- Nanofitins can deliver biotherapeutics into the brain, via Receptor Mediated transcytosis
- Most recent transport examples will be highlighted during this presentation
12:10 pm Session Reserved for Base Pair Biotechnologies
Track 2: Biology & Implementation
Designed for:
neuroscience, biology, pharmacology, DMPK, PKPD and translational development teams seeking to evaluate, implement and de-risk CNS delivery approaches within therapeutic programs.
Explore biodistribution, target engagement, in vitro and in vivo models, dose translation, safety assessment and disease-specific case studies to understand how delivery technologies perform in real-world development settings.
In Vitro BBB Models for Predictive CNS Drug Delivery & Translation
11:30 am Rethinking Brain Shuttle Targeting Through the Lenses of Human Receptor Variability & Dual-Targeting Strategies
- Profiling TfR, IGF1R, LRP1 and emerging BBB targets across human brain microvessels reveals conserved disease-state expression but high inter-individual variability with potential implications for patient stratification
- Understanding order-of-magnitude variability in receptor abundance and its potential impacts on binding kinetics, target engagement, and dose–exposure relationships for shuttle-enabled therapeutics
- Engineering and benchmarking dual-target brain shuttles to optimize transcytosis efficiency while balancing affinity, valency, and receptor trafficking dynamics
- Evaluating modality-specific considerations for dual shuttles including antibody and oligonucleotide payload delivery, and defining engineering principles to enhance target engagement while avoiding rapid plasma clearance, reduced BBB transport or decreased neuronal uptake
12:00 pm Engineering Glycan Targeting Strategies to Enable Non-Invasive Transport Across the Blood Brain Barrier
- Introduce the cerebrovascular glycocalyx as a previously underexplored interface regulating blood–brain barrier transport and a novel target for therapeutic delivery
- Describe the design of mucin-binding protein shuttles that exploit glycosylated structures at the BBB to enable transcytosis of antibodies and biologics into the brain
- Evaluate how glycan-targeted delivery platforms compare with conventional systemic and invasive approaches in achieving efficient brain-wide biodistribution across multiple CNS cell types
- Discuss translational implications of leveraging endogenous BBB transport biology to improve delivery of large-molecule therapeutics for neurodegenerative disease and other CNS indications
1:00 pm Lunch & Networking
Track 1: Technology & Platform Development
Engineering Antibody Shuttles Conjugated to Other Therapeutic Modalities Beyond Antibodies
1:30 pm Translating Lessons from GalNAc to the Brain Exploring How Oligonucleotide Chemistry Shapes CNS Delivery and Distribution
- Applying key learnings from the GalNAc platform to CNS-targeted delivery systems to improve brain exposure and therapeutic performance
- Understanding how oligonucleotide backbone chemistry, from negatively charged siRNAs to neutral PMOs, influences conjugate design and biodistribution
- Evaluating the impact of drug-to-antibody ratio and conjugation architecture, including 1:1, 4:1 and 8:1 formats, on delivery efficiency and CNS distribution profiles
- Leveraging chemical modifications to optimize shuttle performance, enhance target engagement and overcome barriers to effective brain delivery
2:00 pm Panel Discussion: Improving Brainshuttle Chemistry for Enhanced Brain Delivery & Distribution Through the Brain
- What are the biggest chemistry-related barriers preventing today’s brain shuttles from achieving broad and uniform distribution throughout the brain?
- How should developers balance BBB crossing efficiency against intracellular release, tissue penetration, and target engagement when designing next-generation conjugates?
- Which molecular properties are most predictive of successful translation from preclinical brain exposure data to human efficacy?
- Will future breakthroughs come primarily from improved shuttle architectures, oligonucleotide chemistry, payload engineering, or combinations of all three?
Track 2: Biology & Implementation
DMPK & Biodistribution Studies to Track & Quantify CNS Delivery
1:30 pm Advancing CNS Development Candidates Through Translational Discovery & Humanized Models to Optimize Brain Exposure
- Integrating translational discovery workflows to link target biology with PK/PD and enable progression from early discovery through to development candidate selection
- Applying humanized mouse models to generate human-relevant CNS exposure and distribution data, strengthening confidence in translational predictability
- Enhancing brain exposure through structure–property optimisation, transporter-aware design, and minimizing efflux liability during lead optimization
- Using in vivo exposure–response relationships and translational modelling to de-risk candidate selection and support robust IND-enabling strategies
2:00 pm Evaluating Drug Distribution to the Central Nervous System Via Imaging & Molecular Modalities
- A review of current methodologies used to measure drug biodistribution in the CNS, including terminal and non-terminal modalities
- Advantages/disadvantages of common modalities, including optical imaging, PET/CT, ELISA, and PCR
- Translation of methodologies across species/challenges in larger animal models, including differences in BBB permeability, CSF turnover and volume
2:30 pm Scientific Poster Session
This is an informal session to help you connect with your peers in a relaxed atmosphere to continue forging new and beneficial relationships. With an audience of CNS biologists, platform and technology experts, protein engineers, DMPK scientists, chemists and more, you will have the opportunity to display a poster presenting your own work and innovations. Don’t miss out on the chance to connect, learn, and present.
3:00 pm Afternoon Break & Refreshments
Defining What Makes Brain Delivery Platforms Fundable, Scalable & Adoptable
3:30 pm Panel Discussion: The Investment Landscape for CNS Delivery Technologies: Trends, Drivers & Funding Priorities
- Assess where capital is concentrating across CNS delivery modalities and identify the key scientific and commercial drivers behind investment decisions
- Define the target product profiles and competitive advantages that make CNS delivery platforms attractive for partnerships and acquisition
- Evaluate the timelines, capital requirements, and key inflection points for advancing CNS delivery technologies from preclinical development to clinical validation
- Examine how cost-of-goods, scalability, and risk profiles influence long-term commercial viability and investor confidence
4:15 pm Panel Discussion: Aligning Patient Experience with Next-Generation Brain Delivery for Less Burdensome, Safer & More Accessible Treatments
- Understanding patient and caregiver preferences on delivery route frequency and invasiveness to inform the design of brain shuttle and systemic platforms
- Evaluating tolerability safety perceptions and adherence challenges associated with next generation brain delivery approaches across real-world care settings
- Capturing patient perspectives on monitoring burden digital endpoints and treatment impact to guide clinically meaningful endpoint selection
- Identifying barriers to access and uptake of advanced CNS delivery technologies and defining strategies to ensure equitable and scalable implementation