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

Synopsis

  • 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

Synopsis

  • 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?

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

Synopsis

  • 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

4:00 pm Chair’s Closing Remarks