8:00 am Coffee & Refreshments
8:30 am Chair’s Opening Remarks
8:40 am Exploring How Public-Private Partnerships can Enhance Progress on BBB Research
- Bernd Stowasser Head Global Public Private Partnerships & R&D Alliance Management, Strategy & Business Development, Sanofi
- Strategic overview of what is happening in the BBB field globally focusing on drug delivery for biologics
- How is the industry exploiting transport receptor and BBB models predictive of invivo exposure?
- Diving into the discovery and development of brain transport mechanisms within COMPACT and IM²PACT consortiums
- Uniting expertise from academia and industry to drive the next wave of innovation
Deciphering the Unknowns in BBB Physiology for Neurodegenerative Diseases
9:10 am Exploring the BBB as a Dynamic Interface to Elucidate Cellular & Molecular Mechanisms involved in BBB Function
- Richard Daneman Assistant Professor, Departments of Neurosciences & Pharmacology, University of California, San Diego
- Basic mechanism of BBB development and disease and how the BBB interfaces with the brain to regulate behaviour
- Elucidating how the BBB changes based on stimuli from the brain and blood
- Revealing the mechanisms that lead to dysfunction of the BBB during neurodegenerative diseases
9:40 am Panel Discussion – To What Extent are the Challenges of Crossing the BBB Disease Specific?
- In what neurodegenerative diseases is the BBB more compromised?
- How can we use this to drive therapeutics across the BBB?
- How can we explore to what extent the BBB breakdown contributes to synaptic dysfunction, neurodegeneration, and cognitive impairment?
- How does each neurodegenerative disease change the nature of the BBB itself?
- How does inflammation influence the blood-brain barriers integrity?
10:20 am Networking Break
Discovering Various Approaches to Accurately Measuring the Amount of your Biologic that has Crossed the BBB
11:00 am The Saturation Concept for Brain Penetration of Antibodies
- Axel Meyer Head of BBB and Advanced ADME at DMPK and Bioanalytical Research, AbbVie Deutschland
- RMT is a saturating process, and the dose range is limited by transport capacity of the carrier
- We validated experimentally the saturation concept in vivo using an antibody against TfR as well as different affinity variants
- As predicted by MPO modeling, at higher doses the advantage of Trojan Horse antibodies over non-targeted antibodies is progressively diminishing
11:30 am Quantitative Methodologies for Measuring Amount & Distribution of Antibodies in the Brain
- Danica Stanimirovic Director, Translational Bioscience Department, Human Health Therapeutics Research Centre, National Research Council of Canada
- How and where (which compartment within CNS) we measure antibodies impacts translational PBPK-BD
- Advantages and caveats of analytical and imaging methods for quantifying brain penetration of antibodies
- Measuring kinetics of antibodies in the brain– entry vs. elimination
- Surrogates for CNS target engagement vs. amount of Ab in the brain – what do they tell us?
12:00 pm Application of Physiologically-Based Pharmacokinetic (PBPK) Modeling to Predict Antibody Exposure in Brain
- Peter Bloomingdale Senior Scientist, Quantitative Pharmacology and Pharmacometrics, Merck & Co
- Overview of the application of PBPK modeling to understand antibody disposition in the brain and the level of target engagement
- Understanding the relationship between plasma versus brain exposure of antibodies and role of transport processes
- Case studies to highlight preclinical-to-clinical translation of antibody exposure and target engagement in the CNS
- Provide a workflow for tailoring and qualifying a PBPK/TE model depending upon the neurological disease of interest
12:30 pm Networking Lunch
Translational Studies – Finding Solutions to Bridge the Gap Between Preclinical Findings & Clinical trials
1:30 pm Panel Discussion – Driving Better Translation Across Species
- How is the binding interaction and transcytosis different across species?
- To what extent is the mouse a good model for human blood-brain barrier penetration?
- What are the main limitations in translatability between the mice studies and the human studies?
- Defining the right approach to designing preclinical studies in non-human primates
- Elucidating how the BBB is different from lower species to non-human primates to human
- How can we leverage animal studies to provide mechanistic insights?
- What is the species to species translatability? How consistent are these studies across species?
Discovering Alternative Strategies to RMT Delivery Systems
2:00 pm Exploring Neural Exosomes as a Delivery Approach to Cross the BBB
- Steven Stice Co-Founder, Chief Scientific Officer, Aruna Bio
- Harnessing the power of exosomes: realizing the potential of exosomes as delivery vehicles for a range of therapeutics, including siRNAs, ASOs and antibodies across the BBB
- Outlining what makes neural exosomes efficient BBB drug delivery tools
- Delving into the utilization of neural exosomes as both a therapeutic and a delivery vehicle to treat a range of neurodegenerative diseases
- Data to demonstrate the therapeutic benefit in multiple animal models
2:30 pm Afternoon Refreshments & Networking
3:00 pm Non-Invasive, Dual-Targeted Delivery of Non-BBB Penetrant Therapeutic Agents to CNS
- Susan Rosenbaum Founder, Chairman & Chief Executive Officer, Lauren Sciences LLC
- Technical overview of advances in how V-Smart® BBB Platform engineers micro brain targeting for improved target exposure
- Insight as to how V-Smart® is adapted for each neurodegenerative disease in V-Smart® CNS Pipeline
- Exploring advanced PK and animal model efficacy studies for V-Smart® Nanomedicines in ALS and Parkinson’s
3:30 pm Discovery & Development of Peptides Targeting Brain Mitochondria for the Treatment of Neurodegenerative Disease
- Dennis Keefe Executive Director of Discovery Biology, Stealth BioTherapeutics
- Overview of our product candidate, SBT-272, a novel peptidomimetic being developed for the treatment of neurodegenerative diseases involving mitochondrial dysfunction
- Research behind drug design to improve potency and blood-brain barrier penetration
- Evaluating early signals of efficacy in preclinical studies in ALS
- Identifying a responsive biomarker to inform ongoing preclinical studies in ALS
- Exploring future development efforts in other neurodegenerative diseases