9:00 am Chair’s Opening Remarks

Diving into Device Based Approaches for BBB Penetration

9:10 am Opening the BBB in Parkinson´s Disease and Other Neurodegenerations: A Window into Disease Modification

  • José Obeso Professor of Neurology , HM-CINAC Fundacion Hospitales de Madrid & CEU-San Pablo University

Synopsis

  • Clinical Case Study: Using Focused Ultrasound to target the striatum in patients with Parkinson’s Disease
  • How focused ultrasound opening of the blood-brain barrier provides a unique opportunity for early intervention to treat neurodegenerative disorders
  • Use of experimental model to optimize methodology and screening for therapies
  • Exploring BBB brain delivery via viral vectors, more favourable, more invasive
  • What are the main obstacles to these techniques and how can we solve them?
  • Applicability to deliver the therapeutic agent early on
  • What have we achieved in patients to date and what’s next on the horizon?

9:40 am Learning from Oncology – Exploring Delivery of Therapeutics to CNS Tumours

  • Manmeet S. Ahluwalia Manmeet S. Ahluwalia, MD, MBA Chief of Medical Oncology, Chief Scientific Officer & Deputy Director, Miami Cancer Institute, Baptist Health South Florida

Synopsis

The why

  • There have been no meaningful new approved drugs for GBM and other BTs
  •  How lack of BBB penetrance is one of the main issues
  • However, good rationale for approved and experimental drugs/treatments for BTs but lack brain penetrance

The opportunity:

  • Brief outline of denovo chemistry, lipidation, nanoformulation, other
  •  FUS efforts in BT space
  •  Case examples with Clinical PoP for carboplatin, current landscape
  • CED efforts in BT space
  • Case study and current landscape

10:10 am Live Q&A with your Expert Speakers

  • José Obeso Professor of Neurology , HM-CINAC Fundacion Hospitales de Madrid & CEU-San Pablo University
  • Manmeet S. Ahluwalia Manmeet S. Ahluwalia, MD, MBA Chief of Medical Oncology, Chief Scientific Officer & Deputy Director, Miami Cancer Institute, Baptist Health South Florida

10:20 am Speed Networking

Assessing the Use of Nanoparticles for Brain Targeting & As Biomarkers of BBB Abnormalities

11:00 am Panel Discussion: Challenges and Opportunities in Nanoparticle Engineering for Brain Targeting – Focus on Intravenous Routes of Injection

  • Axel Meyer Principal Research Scientist I, BBB & Advanced ADME , AbbVie
  • Moein Moghimi Professor of Pharmaceutics & Nanomedicine Newcastle University, Newcastle University
  • Manuel Sanchez-Felix Senior Fellow, Novel Delivery Technologies, Novartis Institute for Biomedical Research

Synopsis

  • Can nanoparticles delivery their promise in drug delivery to the brain?
  • How much do we know about nanoparticle transcytosis across the BBB?
  • What do we need to know about nanoparticle transcytosis across the BBB?
  • Where do nanoparticles end up after transcystosis?
  • What pathological conditions in the brain should be the target of nanoparticle therapeutics?
  • The challenge with hypothesised-driven surface engineering approaches

11:30 am The Brain Delivery Trifecta: Triple-Targeted, Non-Invasive CNS Platform for Non-BBB Penetrants

Synopsis

  • Learn how V-Smart® Technology enables and engineers non-brain penetrants, from small molecules to oligonucleotides
  • Hear update on V-Smart® Platform, and its use for proprietary pharma compounds to macro-target and micro-target
  • See latest V-Smart® Pipeline pre-clinical efficacy data, for V-Smart® Nanomedicines in ALS, PD, GBM, AD

12:00 pm A Novel Solid Lipid Nanoparticle Formulation for Successful Drug Delivery Across the BBB

  • Axel Meyer Principal Research Scientist I, BBB & Advanced ADME , AbbVie

Synopsis

• Nanoparticles have been proposed as drug delivery systems for the
treatment of various diseases.
• Very often, the surfaces of nanoparticles are functionalized with
targeting ligands against specific receptors to direct them to their
therapeutic target cells or organs
• However, this targeting can be lost when nanoparticles are placed in
complex biological environments by formation of a protein corona
• Here we present a novel strategy to overcome the problem of corona formation

12:30 pm Exploring Applications of PET-MRI Nanoparticles for Multimodal Imaging of the BBB

Synopsis

  • Leveraging nanoparticles as biomarkers of BBB abnormalities: from large leakage to subtle increase of permeability
  • Focus on nanoparticles as tools to measure transient opening of the BBB and permeability of the choroid plexus
  • Discussion on nanoparticles entry routes and use for drug delivery monitoring

1:00 pm Nanoligand Carriers: A Versatile Self-Assembly Platform for the Delivery of Macromolecular Therapeutics to the Brain

  • Moein Moghimi Professor of Pharmaceutics & Nanomedicine Newcastle University, Newcastle University

Synopsis

  • Simple-by-design and plug-and-play with Nanoligand Carriers
  • Active targeting and transcytosis through two receptors
  • Fast delivery of nucleic acid medicines (siRNA, mRNA) and peptide cargoes to the brain parenchyma on intravenous injection
  • Neurons and microglia as target cells
  • Confirmed pharmacological activity on a single dose
  • Unprecedented safety

1:30 pm Live Q&A with Your Expert Speakers

  • Susan Rosenbaum Founder, Chairman, CEO, Lauren Sciences LLC
  • Emmanuelle Canet Soulas Professor of Physiology, University of Lyon, CarMeN lab, INSERM
  • Moein Moghimi Professor of Pharmaceutics & Nanomedicine Newcastle University, Newcastle University
  • Axel Meyer Principal Research Scientist I, BBB & Advanced ADME , AbbVie

1:50 pm Poster Session & Lunch

Synopsis

The Poster Session is an informal part of the conference agenda, allowing you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships. This is also your chance to showcase your work to leaders in the field!

Driving Innovation in Models of the BBB

2:30 pm Human Induced Pluripotent Stem Cell-derived Brain Endothelial Cells: Current Controversies and Progress

  • Raphael Lis Assistant Professor, Weill Cornell Medical College

Synopsis

  • iBMEC based models of the barrier have been embraced as an important standard tool in brain research
  • iBMECs were mistakenly classified as endothelial cells
  • ETS factors overexpression can rescue a vascular fate in iBMECs

3:00 pm In Vitro BBB Models for Biologic Drug Screening

Synopsis

  • Key characteristics for a good model
  • Parameters that affect RMT transcytosis of biologic drugs
  • In vitro and in vivo translation

3:30 pm Application of BBB-on-chip Models in Pre-Clinical Assessment of Glioblastoma-Targeting CAR-T Based Immunotherapies

  • Anna Jezierski Research Officer, Human Health Therapeutics Research Centre, National Research Council of Canada

Synopsis

  • Crossing the BBB is an important step in the systemic application of CAR-T therapy for the treatment of glioblastomas and other CNS malignancies
  • Development of BBB-on-Chip platforms enable visualization of CAR-T cell interaction and extravasation across the brain endothelial cell 3D lumens
  • BBB models in vitro are key to assessing CAR-T-induced BBB disruption, accompanying toxicities and cytotoxic effector function on target cells.

4:00 pm State-of-the-Art Tissue-Engineered Models of the Human Blood-Brain Barrier

  • Raleigh Linville Post-doc, Searson Group, Johns Hopkins , Institute for NanoBioTechnology

Synopsis

  • Tissue-engineered BBB models exploit advances in induced pluripotent stem cell (iPSC) and microfluidic technology
  • Tissue-engineered models recapitulate key structural and functional properties of the human blood-brain barrier
  • Tissue-engineered BBB models have key advantages compared to traditional 2D models (i.e. transwells), including physiological and stable barrier properties, unique gene/protein expression, and enriched endothelial identity
  • Tissue-engineered BBB models enable studies of brain disease and drug delivery with high spatiotemporal resolution

4:30 pm Live Q&A with Your Expert Speakers

  • Anna Jezierski Research Officer, Human Health Therapeutics Research Centre, National Research Council of Canada
  • Katherine Deng Principal Scientist, AbbVie
  • Raphael Lis Assistant Professor, Weill Cornell Medical College
  • Raleigh Linville Post-doc, Searson Group, Johns Hopkins , Institute for NanoBioTechnology
  • James Gorman Brain Targeting Program , Wyss Institute for Biologically Inspired Engineering

5:00 pm Chair’s Closing Remarks