7:20 am Registration & Breakfast
8:20 am Chair’s Opening Remarks
FROM CHIP TO MOUSE: ESTABLISHING HUMAN RELEVANT MODEL SYSTEMS TO STUDY CNS DRUG DELIVERY
8:30 am Panel Discussion: Evaluating In Vivo & In Vitro Systems to Effectively Model Transportation of Neurological & Neuro-Oncology Drugs to the CNS
Synopsis
- From microfluidics to BBB chips to organoids: which in vitro systems show greatest translational relevance?
- What must be improved in in vivo recapitulations of BBB to achieve reliable drug read outs that can be used in human trials?
- How can we achieve greater confidence in in vitro systems to replicate human BBB biology?
- How can we streamline translational dose scale up to optimize CSF dosing?
9:15 am Exploring Beyond Static Transwell Platforms to Replicate Dynamic Flow & BBB Permeability
Synopsis
- Showcasing potentials of microfluidic models to translate successful BBB-penetrant candidates to the clinic
- Stimulating shear stress to recapitulate human BBB endothelial physiology
- Exploring the practical applications of microfluidics to study receptor expression and functionality
9:45 am Speed Networking
Synopsis
An optimal chance to network with an expanding community of experts adopting novel and innovative approaches to target therapeutic candidates to the CNS. Connect with peers across discovery, preclinical, translational, and clinical neuroscience and neuro-oncology to collaboratively discuss and share ideas to advance therapeutic delivery mechanisms
10:30 am Morning Break & Refreshments
11:00 am Innovating Next Generation BBB-Penetrant AAVs for Gene Therapy
Synopsis
- Discussing innovative advancements in IV administered AAV capsids to access the CNS
- Using engineered AAVs to treat neurodegenerative disorders and beyond
11:30 am Optimizing Architecture of Brain Shuttle-Antisense Oligonucleotide Conjugates for the Treatment of Neurological Disorders in In Vivo Studies
Synopsis
- Aspects and conceptual design considerations of brain shuttle-ASO conjugates
- Analyzing the translational feasibility of this system to revolutionize potentials for clinical systemic targeting of neurological disorders
- Potential of peripheral administered CNS-targeting ASOs
12:00 pm Demonstrating Successful Blood-Brain Barrier Penetration of AAVs in Rodent Models to Optimize Gene Delivery for Neurological Disorders
Synopsis
- Leveraging in vivo screening approaches to identify Transferrin Receptor-specific antibodies to support BBB crossing of conjugated AAVs
- Utilizing specific capsid mutations to abolish natural glycan binding and minimize peripheral targeting of undesired tissues
- Comparing ICV and IV route for AAV targeting to the mouse brain
12:30 pm Lunch & Networking Break
BROADENING THE MEDICINAL CHEMISTRY TOOLBOX TO CONSIDER NOVEL CNS DELIVERY OPTIONS
1:30 pm Leveraging AI & Machine Learning to Accelerate the Development of CNS & Brain Cancer Molecules
Synopsis
- Exploring how challenges in the development of brain and CNS molecules can be met with AI and machine learning
- Developing highly accurate AI algorithms to predict the BBB permeability of any compound
- Using AI approaches to rapidly advance brain and CNS drug candidates from discovery into clinical development
2:00 pm Roundtable Discussion: Exploring Challenges & Opportunities for Advancing Delivery of Brain-Targeted Therapeutics
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas:
- Exploring new shuttle targets and analysis of their polarized expression and trafficking machinery
- How can we couple advances in medicinal chemistry to develop small molecules to treat rare diseases in the CNS?
- Which innovations prove most promising when considering new and alternative delivery routes to the brain?
2:45 pm Afternoon Break & Refreshments
MAPPING THE DELIVERY PATHWAY TO THE BRAIN: MODELLING PHARMACOLOGICAL
3:15 pm Modelling Drug Biodistribution Using PK/PD Analysis for Pediatric & Adult Glioblastoma
Synopsis
- When developing drugs for invasive brain tumors, delivery through the normal blood-brain barrier (BBB) and the blood-tumor barrier (BTB) must be addressed in any PK-PD modelling
- Moreover, delivery must be assessed in unison with drug potency and target concentrations in order to have an informed, rational decision process to bring drugs from preclinical discovery to clinical trials.
3:45 pm Leveraging Imaging Studies to Assess & Validate Drug Delivery in the Brain
Synopsis
- Visualizing drug penetration with imaging systems
- Quantify brain vs plasma concentration levels and correlate with biological activity
4:15 pm Harnessing Mass Spectrometry Techniques to Measure Drug Uptake into Brain Tumors
Synopsis
- Performing mass spectrometry across entire tumor samples to analyze chemo biodistribution and determine successful target engagement
- Exploring clinical applications of mass spectrometry for improving therapeutic uptake to desired regions of brain tumors
4:45 pm Chair’s Closing Remarks
4:55 pm Scientific Poster Session
Synopsis
This is an informal session to help you connect with your peers in a relaxed atmosphere and continue forging new and beneficial relationships. With an audience of molecular neurobiologists, CNS delivery device engineers and neuro-oncologists eager to hear the latest cutting-edge advancements, you will have the opportunity to display a poster presenting your own work. Additionally, you will have the chance to review other’s posters displaying novel approaches to administer drugs safely and efficaciously to the CNS