8:00 am Registration & Coffee

8:30 am Chair’s Opening Remarks

8:40 am High Level Review of the Recent Advances of Drug Delivery Across the BBB

  • William A. Banks Associate Chief of Staff - Research & Development | Professor, Division of Gerontology & Geriatric Medicine, Department of Medicine, VA Puget Sound | University of Washington

Synopsis

  • A critical review of the most important areas that have recently redefined the BBB and how they can be applied to the development of CNS therapeutics specifically for neurodegenerative diseases
  • Evaluating R&D focused on novel approaches to BBB delivery including the use of exosomes, antisense and biologics

Uniting the Transferrin Receptor with Brain Shuttle Technology to Get Biologics Across the BBB

9:10 am Transferrin Receptor-Mediated BBB Shuttling Antibodies Based on VNARs

Synopsis

  • Advantages of VNARs as brain shuttles
  • Mechanistic requirements for efficient brain transport
  • Wide range of payload delivery including peptides, enzymes and antibodies

9:40 am Blood-Brain Barrier Penetrating Bi-Specific Antibodies to Treat Neurodegenerative Diseases

Synopsis

  • Reviewing how anti-TfR based bi-specific antibodies in different formats showed increased brain uptake over the respective monoclonal antibody
  • Engineering and screening of scFvs for bi-specific antibody generation
  • Functional in vitro screening of TfR and non-TfR antibodies

10:10 am Speed Networking

Synopsis

This session is the ideal opportunity to meet face-to-face with the key thought leaders working in investigating the BBB. Specifically designed to connect you with new contacts from the most active companies in the field, the renowned Speed Networking session will be one of the most valuable hours you will spend at the BBB Summit

10:45 am Morning Refreshments

Expanding our Horizons & Looking Beyond the Transferrin Receptor for BBB Penetration

11:15 am Seeking Improved Targets & Compounds for Targeted Brain Delivery

  • James Gorman Brain Targeting Program, Wyss Institute for Biologically Inspired Engineering, Harvard

Synopsis

  • Abundance and enrichment of brain microvascular endothelial cell surface proteins does not reliably predict abundance and enrichment of the human homologue
  • We have initiated a hypothesis blind search for abundant and enriched human targets using human omics data
  • To select improved shuttle compounds, we have developed an antibody shuttle selection platform that combines a physiologically relevant human in vitro transcytosis assay with in vivo testing of brain PK/PD in humanized mice

11:45 am A Garden of Forking Paths: Deepening our Understanding of Intracellular Sorting & Transcytosis

Synopsis

  • More than a passenger: antibody ligands modify the cellular behavior of receptors
  • What are the processes that mediate protein transcytosis?
  • What specific molecular mechanisms regulate intracellular sorting?
  • New tools for accelerating the development of next-generation brain delivery platforms for biologics

12:15 pm ABL301, BBB-Crossing Trojan Horse Bispecific Antibody Targeting Aggregated α-Synuclein for the Treatment of Parkinson’s Disease (PD) & Grabody-B, Novel BBB Shuttle Platform

Synopsis

  • Evaluating ABL301; a bispecific ABL301 is a bispecific antibody targeting aggregated alpha-synuclein (aggregate α-Syn) with the BBB shuttle
  • Grabody-B platform utilize IGF1R as BBB shuttle and showed improved BBB penetration than monoclonal Ab in preclinical models
    • Grabody-B has been optimized by implementing Fc engineering, Affinity maturation and valency test
  • Pre-Tox Study of ABL301 in rats and monkeys showed good safety profiles

12:45 pm Networking Lunch

Driving Innovation in In Vitro Modelling

1:45 pm Pioneering a Multidisciplinary Approach to Drive the Development of Advanced Multi-Organ Human In Vitro Models

  • James J. Hickman Professor, NanoScience Technology Center, University of Central Florida

Synopsis

  • Generating a fully integrated platform to show the complete transport system from the GI tract to the interstitial space in the brain
  • Exploring extensive PKPD modelling of this system to define the pathways of transport of various drugs across the BBB, indicating what transporter proteins are present, and what the permeability’s are to different substances
  • Investigating the barrier function, not just to the BBB but also of the GI tract
  • Combining molecular, imaging and electrophysiological techniques to assess disease BBB systems, how has the BBB been affected by Alzheimer’s disease?
  • Using the PKPD system to predict clinical outcomes

2:15 pm Self-Assembled Vascular Networks as a Tool for Studying Barrier Function in the Brain

  • Roger Dale Kamm Cecil & Ida Green Distinguished Professor, Biological & Mechanical Engineering, MIT

Synopsis

  • In vitro models of the BBB can be generated from human cells with morphology and barrier function
  • BBB models can be used in conjunction with models of neurological disease such as Alzheimer’s and ALS
  • For screening purposes, these models can be cast in high throughput assays with ~40 independent experiments per plate

2:45 pm Networking Break & Poster Session

3:45 pm Panel Discussion: What is the Best Approach to Driving More Robust, Validated & Cost-friendly In Vitro Models that Predict In Vivo

Synopsis

 

  • What steps should we take to make in vitro models more robust? What steps do we need to take to hand over a model to a pharmaceutical company?
  • What are the important parameters to measure?
  • What cells are appropriate to use in our models and how can we have consistency from one cell batch to another?
  • How can we combine in vitro models with in vivo models to make a more predictive discovery platform?

4:15 pm Chair’s Closing Remarks